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AstraZeneca and Daiichi Sankyo announced on Jan. 20, 2021 that their HER2-positive metastatic breast cancer treatment, Enhertu (trastuzumab deruxtecan), has been granted conditional approval from the European Commission as a monotherapy for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more previous anti-HER2-based regimens.

The approval was based on positive results from a single-arm Phase II trial where the treatment demonstrated significant antitumor activities in patients with the disease, AstraZeneca said in a company press release. The treatment was previously approved for use in the United States under accelerated approval, and in Japan under a conditional early approval system.

“Enhertu is already transforming outcomes for patients with HER2-positive metastatic breast cancer in the US and Japan, and this approval enables us to bring the benefits of this medicine to patients in the [European Union],” said Dave Fredrickson, executive vice president, Oncology Business Unit, AstraZeneca. “We will continue to explore the potential of Enhertu in this setting, as well as in earlier lines of treatment and stages of disease, with the ambition of improving the lives of patients with HER2-targetable breast cancer.”

“This expedited review underscores the practice-changing potential of Enhertu for patients in the metastatic setting,” added Gilles Gallant, senior vice president, global head, Oncology Development, Oncology R&D, Daiichi Sankyo, in the press release. “Enhertu is the first-ever new medicine to be approved in breast cancer in Europe on the basis of Phase II single-arm data, and one of the fastest accelerated assessment procedures for an application in oncology.”

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The approval was based on positive results from a single-arm Phase II trial where the treatment demonstrated significant antitumor activities in patients with the disease.

AstraZenca and Daiichi Sankyo’s Breast Cancer Treatment Approved for Use in Europe

Loxo Oncology at Lilly, a research and development group of Eli Lilly and Company, and Merus, a Netherlands-based clinical-stage oncology company, announced on Jan. 19, 2021 that they are entering into a research collaboration and exclusive license agreement to research and develop up to three CD3-engaging T-cell redirecting bispecific antibody therapies using Merus' proprietary Biclonics platform for up to $1.6 billion.

Through the agreement, Merus will receive an upfront cash payment of $40 million, an equity investment by Lilly of $20 million, and $540 million in potential development and commercialization milestones per product, Eli Lilly said in a company press release. Merus will handle the discovery and early-stage research activities of the products, while Loxo Oncology at Lilly will lead additional research, development, and commercialization activities.

"CD3-engaging bispecific antibodies are rapidly becoming one of the most transformative immune-modulating modalities used to treat cancer. We expect these therapies will become an important component of the Loxo Oncology at Lilly biologics strategy," said Jacob Van Naarden, chief operating officer of Loxo Oncology at Lilly, in the press release. "Merus has built a differentiated platform and one that we believe can enable us to create bispecific antibody therapies with wider therapeutic indexes than those available today. We look forward to working closely with Merus to develop new potential medicines for patients with cancer."

"The collaboration with Loxo Oncology at Lilly and their world class research capabilities opens up exciting possibilities for Merus' Biclonics platform," added Bill Lundberg, MD, president and CEO at Merus, in the press release. "Our CD3 T-cell engager platform includes over 175 novel and diverse anti-CD3 common light chain antibodies across a wide range of affinities and attributes and enables functional screening of large libraries for optimal performance. We look forward to working together with Loxo Oncology at Lilly to define a new generation of medicines to treat cancer."

The companies will develop up to three CD3-engaging T-cell redirecting bispecific antibody therapies using Merus' proprietary Biclonics platform.

Loxo Oncology at Lilly and Merus Enter into Bispecific Antibody Partnership for up to $1.6 Billion

Paul S. Larson, MD, paul.larson@ucsf.edu, is professor and vice-chair of Neurological Surgery at the University of California San Francisco, chief of Neurosurgery at the San Francisco Veterans Affairs Medical Center, and surgical director of the Parkinson’s Disease Research, Education and Clinical Center.

While pharmaceutical companies spend billions of dollars developing groundbreaking gene therapies and drugs to address complex diseases of the central nervous system (CNS) (1), the method of delivery is often relegated to an afterthought. With interest and investment in this area continuing to grow at a rapid rate, it is increasingly important to maximize the therapeutic efficacy of both the drug and the delivery method.

 Neurodegenerative disorders and brain cancers are two of the most devastating human diseases, and the world’s aging population continues to increase the frequency of these disorders, making rapid and successful drug development more important than ever. Yet, CNS drug development faces extraordinary hurdles and typically takes much longer than non-CNS therapies. Gene therapy clinical trials for CNS diseases are particularly challenging and protracted due to complexities of the brain, potential side effects, the need for direct and often targeted infusion, and delivery complications. Ultimately, the impact of these life changing CNS drugs is being diminished by the “physics of infusion”. Improved delivery methods must be considered and adopted early in the drug development process, or we risk negating their potential therapeutic benefits (2).

The following discusses the current limitations of traditional drug delivery into the brain, reviews recent advancements and benefits of real time interventional MRI (iMRI)-guided gene therapies, describes some ongoing clinical trials, and provides advice for pharmaceutical companies on how to proceed with intracranial therapies and delivery.

It is well known that local delivery of therapeutic agents into the central nervous system offers advantages, including circumventing the blood-brain barrier, the potential for minimizing systemic toxicity, and the ability to achieve higher concentrations of agents at the target site (3). Intracranial delivery has been performed using traditional neurosurgical techniques and tools, including stereotactic frames and standard cannulas or catheters. These methods lack the ability to confirm accurate catheter placement. Another major challenge has been the inability to visualize the drug delivery in real-time—thus making it impossible to evaluate the accuracy and coverage of drug delivery within target stuctures (4). This traditional delivery method has become known as “blind” infusion; surgeons would infuse the prescribed amount of the drug to the target area but could not ascertain whether the drug covered the target as intended. In addition, traditional blind delivery with a simple hand injection using a syringe does not allow for infusing at a consistent flow rate, making measurement of delivery rates inaccurate and variable across procedures (4).

Gene therapy trials using traditional intracranial delivery methods began almost 15 years ago, but early results showed limited initial success (4). For example, the PRECISE trial, a large-scale Phase III trial delivering interleukin 13 (IL13)-pseudomonas exotoxin for recurrent glioblastoma, failed to show clinical benefit of survival. Post hoc analysis of the trial patients revealed that less than half of the implanted catheters were in the optimal position—in fact, only 68% of catheters were positioned in accordance with the specified protocol. Additionally, the investigators described “a fundamental lack of infusion distribution physics” as a major reason for efficacy failures. In the years since, similar outcomes have been noted in other trials (3).

Traditional surgical image guidance in the operating room is not real time, but rather dependent on co-registration to a previously-acquired MRI. In addition to the errors that co-registration can introduce with regards to device placement, conventional neuro-navigation platforms offer no means of visualizing infusions in real-time. Without live image guidance, a surgeon has no way to ensure accurate catheter placement and monitor the pattern of distribution of the infusions within the intended target (3).

Initially, surgeons believed that an infusion cannula could be placed into the center of a brain target and the injected fluid would behave in a predictable fashion, similar to an injection of fluid into a beaker of homogenous gelatinous material. The assumption was that the infusion would grow as a uniform spherical ball, increasing in size proportional to an increase in infusion volume. Surgeons now know this is not the case. For example, infusions in the putamen can result in spread of infusate outside the putamen along perivascular spaces, reflux along the inserted cannula, and, to a lesser extent, leakage into the vacated catheter tract upon its removal. Utilizing real-time imaging has enabled surgeons to observe these delivery complications and test new techniques and devices to optimize results.

The advent of real time iMRI-guided infusion has enabled larger volumes of delivered therapeutics and tactics, such as varying infusion rates and adjusting cannula depth/position to maximize coverage of the target area—and to minimize the degree of vector spread away from the intended target. This has significant implications for the success of clinical trials, as evidence suggests that adequate coverage of the target area may have a dramatic impact on patient outcomes (3).

For pharmaceutical and biologics companies, the inability to predict, visualize, and confirm infusate coverage of target regions has proven to be a major hurdle in clinical development—and a cause for therapeutic inefficacy. Without intraoperative monitoring, it has not been possible to determine how differences in surgical approaches, cannula designs, vector volumes, and dosing affect coverage within the brain (4).

One platform that allows for iMRI-guided catheter placement and real-time visualization of delivery of therapeutic agent is ClearPoint NeuroNavigation, developed by US-based medical device company, ClearPoint Neuro, which is currently enabling a range of clinical trials involving neuro-oncology, neurodegenerative disorders, rare genetic disorders, and movement disorders. It is an integrated hardware and software platform that communicates with the MRI scanner console to provide high-resolution imaging for trajectory planning, entry point selection, device placement, and infusion monitoring. The navigation system can be used in conjunction with the ClearPoint Neuro SmartFlow Cannula for precise dosage of drugs or gene therapies. The company’s cannula leverages convection enhanced delivery (CED) to apply positive pressure that allows for consistent flow rates and creates a pressure gradient at the cannula tip to push interstitial fluid out of the way, which provides better coverage and allows larger volumes of infusion. Additionally, this cannula’s single, continuous glass lumen and stepped distal tip prevents leaks, reduces reflux, minimizes priming/dead volume, and allows for precise control of infusion rate.

The introduction of iMRI-guided drug delivery uncovered many injection challenges associated with targeted infusion within the brain. Based upon the diffusion behaviors and characteristics observed during early iMRI monitoring in non-human primates, many protocols have now adopted a technique of either progressive advancement from proximal to distal for the duration of infusion, or a “pull-back” method for cell delivery where the first deposit is delivered to the most distal region. This technique of administering multiple infusions along the trajectory path was found to increase the surgeon’s ability to achieve infusions that cover the putamen or other target area as uniformly as possible, with the added benefit of providing the ability to move the cannula tip away from problematic perivascular spaces. In addition, innovations in cannula design have resulted in more reliable, efficient, and increased distribution of agents. Because image-guided systems provide real-time visualization of the infusion, they allow for intraoperative troubleshooting and real time modification to key infusion parameters, such as flow rate, positioning of the catheter, or terminating the infusion when the desired coverage has been attained (3).

This latter advancement is particularly noteworthy as it introduces the potential for 

, a game-changing capability in treating CNS disorders or brain cancer. Historically, physicians have strictly followed a standardized trial protocol that specifies uniform infusion parameters across all patients, including the dose (volume) of therapeutic delivered. Evidence suggests that many early trials administered dosages that were insufficient to achieve desired clinical outcomes. iMRI-guided surgery offers the possibility of using much higher volumes, and adjusting the volume to focus on patient specific coverage, rather than a pre-determined amount. This concept of patient specific dosing is new and exciting in its potential to improve clinical outcomes—and it is currently being applied right now in an ongoing Parkinson’s Disease (PD) trial.

Finally, these technological advancements afford an improved level of safety. iMRI provides surgeons with a significantly higher degree of confidence that the cannula is following a safe trajectory and being placed in the desired target location. This is particularly important when targeting deep structures, where safe corridors through the brain can be only 2–3 millimeters wide.

Ongoing clinical trials of iMRI-guided drug delivery

At the University of California San Francisco, Paul Larson, MD, a professor and the vice-chair of Neurological Surgery at the university, is involved in three active human clinical programs utilizing iMRI-guided gene therapy delivery. The first is an amino acid decarboxylase program (AADC), which is an enzyme replacement gene therapy strategy for PD that uses adeno associated virus (AAV) to deliver a gene for AADC. The program, sponsored by Neurocrine Biosciences and Voyager Therapeutics, is now a Phase II multi-center study. The Phase I results indicate that, as coverage of the putamen improved, there was a significant increase in PET activity and a concurrent decrease in PD medications (5).

The second is a Phase Ib investigation sponsored by Brain Neurotherapy Bio to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with earlier stage PD. Growth factors are an exciting approach as they have the potential to be a disease-modifying therapy.

The third is the world’s first intracranial gene therapy study for Huntington’s Disease (HD). Sponsored by uniQure Biopharma, it is a Phase I/II study of AMT-130 delivered to the striatum of patients with early manifest HD and is designed to establish safety and proof-of-concept. The gene product is designed to interrupt production of the aberrant Huntington protein.

Beyond these studies, iMRI-guided delivery is being used in many other ongoing or planned Phase I and Phase II clinical trials for indications, including glioblastoma, PD, Huntington’s Disease, Amyotrophic Lateral Sclerosis (ALS), and pediatric indications, including AADC deficiency, Freidreich’s Ataxia, Angelman Syndrome, and Sanfillippo Syndrome. iMRI-guided delivery with the SmartFlow cannula is already cleared for Cytarabine injection into the ventricles to treat leukemia and non-Hodgkin's lymphoma, and numerous other programs are in development involving gene therapy, cell transplants, and other therapeutics.

Although it may be premature to assign hard savings to iMRI-guided drug delivery, there are significant potential benefits from such an approach. Given the tremendous R&D costs to develop a gene therapy as well as the cost of a single dose and the infusion procedure, any increase in the likelihood of a successful procedure would translate into considerable savings for the healthcare industry at large—as well as accelerating adoption of pharmaceutical gene therapies. The potential for iMRI guidance to ensure safe and accurate placement of the catheter, administer patient-specific dosages, minimize leakage, and optimize coverage addresses many of the challenges faced by early gene therapy trials. When considering the costs of past negative phase two trials, the business case for leveraging iMRI is apparent.

The advantages of real-time MRI-guided gene therapy delivery are clear. However, one challenge for pharma companies is the number of neurosurgery centers that have implemented iMRI at their facilities. The ClearPoint NeuroNavigation system is currently installed in more than 60 of the leading neurosurgical centers across the United States, and is expanding into Europe. While the number of institutions adopting iMRI is growing, there is a need for more pharmaceutical companies and neurosurgeons to embrace this improved delivery method for intracranial therapeutics. To do otherwise is to potentially jeopardize billions of dollars in R&D investments, put study participants in harm’s way with little chance to show benefit, and run the risk of obviating cures for life-threatening diseases.

1. J.P. Fuhr, “The Possibility of Encouraging Innovation and Competition by Erwin A. Blackstone.” 

 8 (6) 1481–1493 (2018).
3. S.J. Han, et al., 

 16 (6) 635–639 (2016).
4. R.M. Richardson, et al., 

91, 1210–1218 (2020).
5. C.W. Christine, et al., 

Paul S. Larson*, MD, paul.larson@ucsf.edu, is professor and vice-chair of Neurological Surgery at the University of California San Francisco, chief of Neurosurgery at the San Francisco Veterans Affairs Medical Center, and surgical director of the Parkinson’s Disease Research, Education and Clinical Center.

*To whom all correspondence should be addressed.

Leveraging real-time MRI guidance for intracranial gene therapy administration potentially improves efficacy and outcomes.

iMRI-Guided Gene Therapy and Drug Delivery for CNS Disorders 

Thermo Fisher Scientific announced on Jan. 19, 2021 that it is acquiring Mesa Biotech, a US-based molecular diagnostic company for $450 million in cash.

Under the terms of the agreement, Thermo Fisher will pay an additional $100 million in cash if certain milestones are reached after the closing of the transaction, which is set for the first quarter of 2021, Thermo Fisher said in a company press release.

Through the acquisition, Thermo Fisher will have access to Mesa’s PCR-based rapid point-of-care testing platform for detecting infectious diseases including SARS-CoV-2, Influenza A and B, respiratory syncytial virus, and Strep A, the press release said.

"Mesa Biotech's innovative platform will enable us to accelerate the availability of reliable and accurate advanced molecular diagnostics at the point of care. Since the start of the pandemic, Thermo Fisher has acted quickly to provide support to the scientists and healthcare professionals at the frontlines of combating COVID-19," said Mark Stevenson, executive vice-president and chief operating officer of Thermo Fisher Scientific, in the press release. "The addition of Mesa Biotech's easy-to-use, rapid PCR-based test is highly complementary to our existing offering and will further help us meet the continuing demand for COVID-related testing while we work to rapidly scale and develop point-of care tests for other infectious diseases in the future."

Through the acquisition, Thermo Fisher will have access to Mesa’s PCR-based rapid point-of-care testing platform for detecting infectious diseases.

Thermo Fisher to Acquire Mesa Biotech for $450 Million

Leukocare, a German biotechnology company, announced on Jan. 11, 2021 that it has officially launched its development laboratories and offices in Milford, MA.

According to a company press release, the new US offices will give the company the ability to support its US-based customers on site while continuing to collaborate with its global team. The offices and laboratories are located on Rentschler Biopharma´s Milford premises, where the companies will continue to carry out their global strategic alliance that was established in 2017.

“I am delighted to announce the opening of Leukocare´s US site today,” said Michael Scholl, CEO at Leukocare, in the press release. “The company´s outstanding development over the last few years led to our desire of establishing a presence in the largest pharmaceuticals market worldwide. This strategic step enables us to be even closer to both our US and global customers and to better understand and serve their needs. Furthermore, the existing infrastructure of Rentschler in Milford—that we will partially make use of—allows us to start operations without any delay and, thus, makes Milford the perfect choice for our newly established US site. We therefore will continue to pursue our strategy of pushing boundaries of biopharmaceutical product development by combining innovative biostatistics, artificial intelligence, and vast formulation expertise with first-class analytics.”

The new US offices will give the company the ability to support its US-based customers on site while continuing to collaborate with its global team.

Leukocare Opens Development Laboratories and Offices in Massachusetts 

Jill Wechsler is Pharmaceutical Technology's Washington Editor, jillwechsler7@gmail.com.

Although there was much analysis and anticipation over the past year about the difficulties and challenges in distributing millions of doses of new vaccines across the nation, the process so far has been much less effective than anticipated. Since FDA authorization of two innovative mRNA vaccines to combat COVID-19 in late 2020, 30 million doses have been distributed, but only 4 million people were vaccinated in December 2020, and just 13 million have received shots so far.

While the Trump administration’s Operation Warp Speed (OWS) deserves kudos for supporting the accelerated development and production of safe and effective anti-COVID vaccines in record time, its plan for distributing and administering the preventives has been marred by political infighting and conflicting messages. State health officials have complained loudly of the lack of resources to support a massive new vaccination program and on limited information from Washington on the timing and size of anticipated vaccine distributions. The initial roll-out to health care workers and nursing home residents were tricky to schedule and arrange, and federal vaccine prioritization guidelines proved cumbersome. Many states are moving on to include more elderly populations, acknowledging that efforts to target certain groups such as nursing homes, teachers, and other front-line workers have been confusing and wasteful.

A main problem was the administration’s delay in authorizing the Centers for Disease Control and Prevention (CDC) to provide states with clear guidelines for updating existing local procedures for distributing vaccines that critical “last mile” to clinics and health centers, a challenge familiar to supply-chain experts. Uncertainty about vaccine shipments and supplies, at a time when hospitals were dealing with surges in COVID-19 infections and deaths, prompted medical centers to cancel in-house vaccination activities. And there still is confusion about whether clinics should hold back supplies for second doses or administer all on hand.

Although vaccine manufacturing has been ramping up, and supply has not been blamed for current distribution failures, there remains concern about unanticipated production problems. In November 2020, Pfizer rattled OWS by disclosing that it would provide only 50 million doses in 2020, instead of the 100 million doses initially expected. And just last week, Pfizer announced plans to reduce vaccine supplies in Europe as it moved to renovate production processes at its Belgium facility. At one point in December, Moderna had to discard 400,000 vaccine doses due a filtration issue during production, although the company maintained that it would still produce its promised doses. Pfizer agreed recently to increase vaccine deliveries in the United States by another 100 million doses in 2021, but that is linked to access to certain supplies and raw materials needed for production. There is great hope that Johnson & Johnson will gain emergency authorization soon for its one-shot COVID vaccine, which would greatly bolster supplies of a product much easier to deliver, store, and administer.

Meanwhile, research is underway on strategies for extending the current vaccine supply. These include studies on whether smaller or less frequent doses of the Moderna vaccine provide sufficient protection. There’s a search for a certain kind of syringe that is able to extract an additional sixth dose from Pfizer five-dose vials. There also has been talk of delaying or dropping the second doses for current vaccines, but FDA officials oppose such an untested move.

Congress finally approved a $900 billion relief package in late December, which provides an additional $8 billion for state vaccine distribution and administration, and that is now beginning to trickle down to local operations. However, efforts by the Biden transition team to form a new vaccine distribution program were hampered by limited communication and information sharing by the Trump administration and OWS. In fact, current vaccine policy was further confused by the recent announcement by Health and Human Services (HHS) secretary Alex Azar that OWS would ship to states thousands of vaccine doses held in warehouses to provide second shots for patients, only to learn that there was little supply on hold to do this.

Meanwhile, President-elect Joe Biden released a

new initiative for ramping up vaccine distributio

n to meet the goal of administering 100 million doses to Americans in the next 100 days. The plan calls for mobilizing the National Guard and the Federal Emergency Management Agency to establish federal vaccination centers, while also working with pharmacies, community health centers, and mobile vaccination clinics to reach broader populations. There’s a promise of more support and resources for states and plans to ensure that all entities know well in advance and understand vaccine allocations and timing of deliveries, including an assurance of second-dose availability, in keeping with current FDA recommendations.

The plan acknowledges the need for added access to critical vaccine components, such as vials, syringes, stoppers, and needles, and may leverage the Defense Production Act to fill any gaps. And there’s support for boosting vaccine finish/fill and packaging operations, as well as for augmenting required cold chain systems needed for effective vaccine distribution and storage. The new administration furthermore backs efforts to “maximize the manufacture of vaccine,” but is vague on how to do this.

In looking to put a new stamp on its federal vaccine initiative, Biden named former FDA commissioner David Kessler to replace OWS leader Moncef Slaoui in directing continued development and production of vaccines and therapies to combat the pandemic. Kessler is best known for expanding FDA regulation of tobacco, establishing FDA user fees, and revising regulatory policies in the face of the AIDS epidemic. There is concern that he has little experience in vaccine research or distribution and has been away from federal health policy for the past 20 years. But now he will be key to the success of the new administration’s promise to vaccinate 1 million people a day to gain control of the pandemic and restore the nation’s health and economic well-being.

Since FDA authorization of two innovative mRNA vaccines to combat COVID-19 in late 2020, 30 million doses have been distributed, but only 4 million people were vaccinated in December 2020, and just 13 million have received shots so far. 

Vaccine Rollout Even Worse than Expected

CPhI Worldwide, organized by Informa Markets, announced on Jan. 15, 2021 that the CPhI event, which was originally scheduled for Aug. 31–Sept. 2, 2021 at the Fiera Milano in Milan, Italy, will be rescheduled for Nov. 9–11, 2021 at the same venue. The decision was made based on the hope that COVID-19 vaccine programs are fully advanced and that international travel is permitted.

“At CPhI Worldwide, we understand the importance of meeting face-to-face with your peers and potential business partners,” the CPhI Worldwide Team said in a press release. “The new dates will also hopefully ensure COVID-19 vaccination programs are well advanced, creating more certainty around international travel and ensuring the event is as successful as possible, delivering you the same opportunities and the experience you’ve come to expect from CPhI Worldwide.

The CPhI event, which was originally scheduled for Aug. 31–Sept. 2, 2021 at the Fiera Milano in Milan, Italy, will be rescheduled for Nov. 9–11, 2021 at the same venue.

CPhI Worldwide Moves to November 2021

Fujifilm and The Massachusetts Center for Advanced Biological Innovation and Manufacturing announced on Jan. 14, 2021 that they are investing $76 million into a new manufacturing and innovation center in Watertown, MA.

The 40,000-ft.2 facility will feature eight cleanrooms with configurations for the production of cell and viral vector products, Fujifilm said in a company press release. The site will all also provide quality control, lab, office, and convening space for academia, industries, and hospitals.

Operations at the center will begin in early 2022.

“Fujifilm is pleased to partner with leading academia, industry, and teaching hospitals, to explore the application of genetically modified cell therapies, and bring new treatment options to the market,” said Takatoshi Ishikawa, senior executive vice-president and chief life science officer, Fujifilm, in the press release. “This collaboration supports our larger healthcare strategy to utilize the combined strength of the Fujifilm Group’s products and services to offer personalized life-altering treatment options to clinicians and their patients.”

The 40,000-ft.2 facility will feature eight cleanrooms with configurations for the production of cell and viral vector products.

Fujifilm to Establish New Manufacturing and Innovation Center in Massachusetts 

Thermo Fisher Scientific announced on Jan. 15, 2021 that it has completed its acquisition of Henogen, Novasep’s viral vector manufacturing business in Belgium, for EUR 725 million (US$875 million) in cash.

Henogen will be integrated into Thermo Fisher’s Pharma Services business within its Laboratory Products and Services segment, Thermo Fisher said in a company press release. The new business offers contract manufacturing services for vaccines and therapies to biotechnology companies and features 7000 m2 of clinical and commercial manufacturing capacity.

“Novasep’s viral vector business is an excellent strategic fit as Thermo Fisher continues to expand its capabilities for cell and gene vaccines and therapies globally,” said Michel Lagarde, executive vice-president of Thermo Fisher, in the press release. “The addition of their manufacturing capabilities in Europe complements our four development and manufacturing sites in North America. In addition, they bring an incredibly talented team with more than two decades of experience across a broad range of viral vectors. The combination will benefit our global customers seeking support and capacity in the region as well as European customers bringing new medicines to patients inside and outside of Europe.”

“Thermo Fisher is an outstanding partner for our viral vector business and its future development,” added Michel Spagnol, president and CEO of Novasep, in the press release. “Our diverse customer base will benefit from the combination of our viral vector services capabilities with the scale and capabilities of Thermo Fisher. Our talented employees will bring deep expertise to an organization that shares our commitment to providing ground-breaking new medicines to patients.”

Henogen will be integrated into Thermo Fisher’s Pharma Services business within its Laboratory Products and Services segment.

Thermo Fisher Completes $875-Million Acquisition of Novasep’s Viral Vector Manufacturing Business

Roche confirmed on Jan. 12, 2021 that the US Department of Health and Human Services (HHS) and the Department of Defense (DOD) have agreed to purchase additional supply of Regeneron Pharmaceuticals’ casirivimab and imdevimab antibody cocktail for use in non-hospitalized COVID-19 patients. The agreement is part of Operation Warp Speed.

Under this new agreement, the US government will purchase up to 1.25 million finished doses of the antibody cocktail by June 30, 2021. With Regeneron already supplying doses to treat approximately 300,000 people, the new agreement brings the total potential purchase to more than 1.5 million doses in the United States.

The efficacy and safety of casirivimab and imdevimab continue to be evaluated in clinical trials for the treatment of COVID-19 in certain hospitalized and non-hospitalized patients. The trials include an open-label trial (RECOVERY) of hospitalized patients in the United Kingdom and a trial for the prevention of COVID-19 in household contacts of infected individuals. In addition, lower doses of casirivimab and imdevimab are being studied with the aim of increasing supply and offering the therapy to more patients. To date, nearly 15,000 people have participated in clinical trials for this antibody cocktail, Roche said in a company press release.

Roche is collaborating with Regeneron to increase global supply of the cocktail and aims to have more than 2 million treatment doses available annually. Regeneron is responsible for the development and distribution of the treatment in the US, and Roche is primarily responsible for the development and distribution outside the US; the company is actively working with governments outside of the US on potential supply agreements. If approved, the companies will support access in low- and lower-middle-income countries through drug donations that will be made in partnership with public health organizations.

The US government said it will provide the additional doses from this new agreement at no cost to patients, though healthcare facilities may charge fees related to administration. The government will also continue to coordinate allocation of the antibody cocktail to state and territorial health departments.

“We are doing all we can to lessen the impact of the ongoing COVID-19 pandemic, and we remain committed to collaborating with Regeneron to increase supply of their antibody cocktail,” said Bill Anderson, CEO of Roche Pharmaceuticals, in the company press release. "Casirivimab and imdevimab will be critically important to help address the pandemic if approved, and we will continue to work with regulators and governments across the globe to bring the medicine to as many people as possible.”

This new agreement covers 1.25 million additional doses of the antibody cocktail containing casirivimab and imdevimab for the treatment of COVID-19.

US Government to Purchase Additional Doses of Regeneron’s COVID-19 Antibody Cocktail

On Jan. 12, 2021, Boehringer Ingelheim and Enara Bio, a science-led company targeting the T-cell/cancer-cell interface for the development of cancer immunotherapies, announced that they have entered into a strategic collaboration and licensing agreement in which they will research and develop novel targeted cancer immunotherapies. The collaboration will leverage Enara Bio’s Dark Antigen discovery platform.

Under the agreement, Boehringer Ingelheim has the option to license Dark Antigens discovered and validated by Enara Bio and will be responsible for all non-clinical and clinical development. Boehringer Ingelheim will also be responsible for the commercialization of associated cancer immunotherapies, including therapeutic vaccines and T-cell redirecting biologics. Meanwhile, Enara Bio will retain rights to use any discovered antigens in cell therapy-based products and is eligible to receive an upfront payment, together with research/preclinical milestones and licensing fees for each tumor type that is explored. Enara Bio is also eligible to receive more than EUR 876 million (US$1.06 billion) in clinical, regulatory and commercial milestones, in addition to royalties on future product sales.

Under the collaboration, Boehringer Ingelheim will combine its approach to developing cancer therapeutics with Enara Bio’s expertise in identifying cancer antigens, with the aim being to provide potential new therapies for difficult-to-treat lung and gastrointestinal cancers.

Dark Antigens represent a new class of cancer-associated antigens. These antigens are derived from genomic dark matter, or the portion of the human genome that is normally not expressed as protein, the companies explained in a press release. Dark Antigen-encoding sequences, which are usually silenced in healthy cells but are activated and presented on tumor cells, are associated with specific cancer types. In addition, these sequences are shared across patients, according to the companies. Because Dark Antigens are not typically visible to the immune system, they represent a large potential repertoire of novel antigens that can be developed as targets for new immunotherapies, the companies stated in the press release.

Through the collaboration, Enara Bio’s proprietary Dark Antigen Platform Technology (EDAPT) will be used to discover and validate novel Dark Antigens in up to three tumor types in the lung and gastrointestinal cancer space. The discovery of shared antigens could lead to the development of vaccines that can have an impact on a broader group of cancer patients.

“We are excited to partner with Enara Bio as part of our mission to bring transformative new treatments to cancer patients,” said Jonathon Sedgwick, PhD, senior vice-president and global head, Cancer Immunology & Immune Modulation Research, Boehringer Ingelheim, in the press release. “We are advancing a unique pipeline of cancer cell-directed agents, immuno-oncology therapies, and intelligent combination approaches to help combat cancer. Enara Bio’s unique discovery platform offers a novel and highly differentiated approach that will allow us to look beyond the known proteome to identify and characterize Dark Antigens to support the development of T-[c]ell [r]eceptor (TCR)-directed immunotherapies and therapeutic vaccines. We believe this is a highly innovative and promising approach to the development of the next wave of cancer immunotherapies.”

“We are extremely pleased to enter this strategic licensing agreement, our first major deal leveraging our pioneering Dark Antigen discovery and validation capabilities,” added Kevin Pojasek, president and CEO of Enara Bio, in the press release. “Boehringer Ingelheim is an innovation-led company dedicated to producing breakthrough treatments with a significant focus on cancer. We are excited to build this relationship and are encouraged that Boehringer Ingelheim shares our view of the potential of Dark Antigens to be a source of important and unconventional targets for novel cancer immunotherapies. This agreement provides strong validation for our science and our approach to exploiting the cancer-associated antigenic repertoire derived from genomic dark matter and we look forward to a productive collaboration.”

The collaboration will focus on up to three types of lung and gastrointestinal cancers.

Boehringer Ingelheim and Enara Bio Partner on Cancer Immunotherapies

Pfizer announced on Jan. 12, 2021 that, during the second half of 2020, it invested a total of $120 million in four clinical-stage biotech companies as part of the Pfizer Breakthrough Growth Initiative (PBGI). Through this initiative, Pfizer is investing up to $500 million in biotechnology companies in an effort to provide funding and access to the company’s scientific expertise. The aim is to help ensure the continuity of promising clinical development programs of potential future strategic interest to Pfizer.

$10 million in Vancouver, BC-based ESSA Pharma, a clinical-stage pharmaceutical company focused on developing novel and proprietary therapies for the treatment of patients with prostate cancer.

$25 million in Cambridge, MA-based Trillium Therapeutics, a clinical-stage immuno-oncology company focused on developing innovative therapies for the treatment of cancer. Jeff Settleman, senior vice-president and chief scientific officer of Pfizer’s Oncology Research & Development Group, was also named to Trillium’s scientific advisory board.

$25 million in Cambridge, MA-based Vedanta Biosciences, a privately held clinical-stage company focused on developing a new category of therapies for immune-mediated diseases. The company’s work is based on a rationally defined consortia of human microbiome-derived bacteria. Michael Vincent, senior vice-president and chief scientific officer of Pfizer’s Inflammation & Immunology Research Unit, will join Vedanta’s scientific advisory board.

$60 million in Bedford, MA-based Homology Medicines, a clinical-stage genetic medicines company specializing in treatments for rare genetic diseases with significant unmet medical needs. Seng Cheng, senior vice-president and chief scientific officer of Pfizer’s Rare Disease Research Unit, has joined Homology’s scientific advisory board. He will handle matters related to Homology’s gene therapy and gene editing programs for phenylketonuria (PKU).

“Pfizer has a long history of collaborating across the healthcare ecosystem with the shared goal of turning great science into innovative new medicines,” said Debbie Baron, senior vice-president of Business Development, Pfizer, in a company press release. “Our investments in Homology, Vedanta, Trillium, and ESSA reflect our commitment to find new and creative ways to leverage Pfizer’s resources to deliver breakthroughs to patients.”

The first four investments of the new program will support clinical-stage biotechnology companies while new investments will include companies focused on oncology, rare disease, and inflammation and immunology.

Pfizer Invests $120 Million in Biotechnology Innovation

On Jan. 14, 2021, Precision Medicine Group (PMG) announced that it has acquired Project Farma, a patient-focused bioengineering services firm focused on supporting life science innovators in the manufacturing and scale-up of advanced therapies. The acquisition significantly expands PMG’s expertise in cell and gene therapy.

Through the acquisition, Project Farma will be part of Precision for Medicine, PMG’s research and development services arm. Project Farma has expertise in planning and implementing complex biomanufacturing strategies and facilities for clients, ranging from start-up biotechnology companies to global biopharmaceutical companies and academic medical centers, PMG said in a company press release. Project Farma has led more than a dozen manufacturing facility builds with capital investments greater than $1 billion.

“In today’s highly competitive and scrutinized advanced therapies marketplace, ensuring a safe, scalable, and high-quality manufacturing capability can be the difference between success and failure. Project Farma are the experts at defining and developing the optimal manufacturing strategy and leading implementation of a customized solution. This essential capability is now expanded and leveraged by the comprehensive cell and gene therapy services available through Precision ADVANCE,” said Precision for Medicine’s President, Chad Clark, in the company press release.

“Since 2013, Precision has supported over 70% of [FDA]-approved cell and gene therapies and is now even better positioned to support the hundreds of therapeutic new entrants coming to market. With our acquisition of Project Farma, Precision is the only life-science services company with true end-to-end capabilities in cell and gene therapy,” said Mark Clein, CEO of Precision Medicine Group, in the press release.

The acquisition significantly expands Precision Medicine Group’s cell and gene therapy expertise.

Precision Medicine Group Acquires Project Farma

FDA announced on Jan. 13, 2021 that it had published a report summarizing the work to date of the COVID-19 Pandemic Recovery and Preparedness Plan (PREPP) initiative, which was launched in August 2020 to strengthen the Agency’s response to the current pandemic and to future public health emergencies.

As part of the initiative, FDA asked an independent, non-government organization to review its pandemic response, record its accomplishments and activities, and pinpoint future opportunities for consideration, an FDA press release said. The organization interviewed FDA leaders, organized listening sessions with external stakeholder groups, and partnered with subject matter experts on the FDA staff to generate a 

"We have a responsibility to learn from our actions to focus on what we can—and must—do better for the future,” FDA said in the press release. “Our experience so far has taught us that transparency and continuous improvement are the essential drivers of a successful response."

A report on the FDA PREPP initiative’s work in 2020 aims to strengthen the Agency’s response to future public health emergencies. 

FDA Publishes COVID-19 Pandemic Recovery and Preparedness Plan Summary Report 

Hovione, a pharmaceutical spray drying company headquartered in Portugal, announced on Jan. 12, 2021 that it has launched ASD-HIPROS, a proprietary screening service for spray dried dispersions.

According to a company press release, the service uses Hovione’s Intelligent PROprietary Screening methodology to screen for combinations of polymers, drug loads, surfactants, and solvents with an advanced computational tool, which produces scale-independent representative samples of the formulations. The samples are then assessed based on performance and stability.

“ASD-HIPROS is a multiple-step screening service that was perfected in the last 15 years and is able to provide an accurate assessment of spray dried dispersion, in less than two months and requiring as little as [five grams] of API,” said Dr. Filipe Gaspar, Hovione’s Chief Technology Officer, in the press release. “Our accumulated experience and expertise in spray drying were used for the development of this platform. It offers a rational formulation definition using a combination of 

 computational modelling and high throughput formulation testing, maximizing the chances of identifying a winning formulation based on outputs obtained from spray drying prototypes.”

The service uses Hovione’s Intelligent PROprietary Screening methodology to screen for combinations of polymers, drug loads, surfactants, and solvents with an advanced computational tool.

Hovione Launches Screening Service for Spray Dried Dispersions

Three New Jersey teens are aiming to aid the pharmaceutical supply chain, one unused medication at a time.

The Altrui Foundation is a non-profit organization dedicated to redirecting unused medications to communities in need. Middletown, NJ residents and founders of the organization, Sourish Jasti, a freshman at the Wharton School of the University of Pennsylvania, Shreya Kavuru, a senior at St. Paul’s School in New Hampshire, and Rahul Kavuru, a junior at St. Paul’s School, formulated the plan in March of 2020 while spending time together before the COVID-19 pandemic began.

“We were having this conversation about how there’s a big problem in the supply chain where billions of unused medications are going to waste each year and pharmacies are spending about $1 to $3 per pound to discard these medications,” Shreya Kavuru says. “At the same time, there are millions of uninsured patients all over the world who are going without the medications they need. We identified this problem and started thinking about how we can play a role in this supply chain.”

The students got to work and decided to make The Altrui Foundation a center point between the manufacturers of the unused medications and the charitable organizations that seek them.

“Charitable organizations request manufacturers’ [medications] whenever there is something wrong. So, if a hurricane hit a certain part of the world, and they needed certain medications, they would reach out to whatever manufacturers that they had connections to,” Jasti says. “Sometimes they’d have strong relationships, other times it would just be cold calling. So, it was a long, messy, process and it was a hassle on both sides.”

According to Jasti, when using The Altrui Foundation, manufacturers provide an inventory list, and the charitable organizations compile medication requests and send them to the foundation. The foundation then serves as a middle ground between the two by transferring the unused medications to the charitable organizations, eliminating the uncertain back and forth between the two parties.

“We’re basically able to complete an inventory list of short-dated products by working with multiple people,” Jasti says. “We keep the sanctity of the direct relationship between the two, so we never touch the product, and we don’t have to go through the regulatory hurdles and the tax deductions at the end of the year. We’re handling the data behind it and our value proposition is a platform to facilitate both sides.”

As of December 2020, the team has shipped more than 12 million medications for Rising Pharmaceuticals, Ingenus Pharmaceuticals, Aurobindo, Camber Pharmaceuticals, Hetero, and Ascent Pharmaceuticals, and the number is growing rapidly. Jasti credits the foundation’s success to its dedicated team.

“We have people on the operations side working with excel sheets, and people working on the outreach with manufacturers and organizations. We also have some really talented people who are in photography and handle our social media,” he says. “We’re trying to give opportunities to people who want to get themselves involved. We could be doing a lot of this with fewer people, but we think that people bring in their own perspectives and their own ideas and it helps to make a really collaborative environment.”

Overall, the students want The Altrui Foundation to serve as a gateway for new initiatives in the pharmaceutical space.

“We’re able to solve two problems in society, which is, one, to allow patients to have an accessible outlet to medications and, two, to help the environment not suffer as many environmentally unfriendly, harmful effects,” Rahul Kavuru says. “This foundation can open the door for different initiatives, and this is just the beginning, so hopefully, with this project, we can create different initiatives that can really help the world and tackle its most pressing needs.”

The Altrui Foundation is a non-profit organization that serves as a center point between manufacturers of unused medications and the charitable organizations that seek them.

New Jersey Students Launch Foundation to Transfer Unused Medications to Communities in Need

As the Biden administration readies to take over the reigns of government, top administration officials continue to rush through new rules and decisions that appear to challenge the independence and effective operations of FDA. One new requirement could revise oversight and review for prescription drugs and certain medical devices. This follows adoption of a broader new rule to “sunset” many established regulations and programs in the Department of Health and Human Services (HHS) unless they can justify their continuation. And in another area, the administration has tried to push through a new plan blocking FDA oversight of genetically modified animals despite strong objections from agency leaders.

The task of dealing with these issues may fall to Janet Woodcock, who appears poised to serve as Acting FDA Commissioner until the Biden administration selects and gains confirmation of a new leader for the agency. Woodcock was recently named medical advisor to Commissioner Stephen Hahn and has been director of the Center for Drug Evaluation and Research (CDER) for many years but was detailed to head up development of anti-COVID-19 therapeutics under Operation Warp Speed.

A sign of FDA’s difficult position is the recent White House move to block Hahn from selecting a replacement for departing Chief Counsel Stacy Cline Amin. Just as the commissioner named experienced attorney Mark Raza to fill the job during the administration’s final days, HHS announced that political appointee James Lawrence would become FDA’s chief counsel, where he could continue the administration’s deregulatory campaign.

A late surprise from the Trump administration is the plan for FDA to publish new information on drug approval timelines and to explain and justify when reviews exceed 180 days. Touted as a “transparency” measure, the new policy instructs FDA to provide data on each approved application for new drugs and generics stating dates filed and approved and number of days over the statutory 180-day timeframe. CDER currently has user fee review goals of 10-months for most new drugs and generics and six months for priority therapies, but change would require the agency to collect and report data on review time frames using different metrics. The larger fear is that this policy could lead to more onerous requirements; at a minimum it’s seen as a meaningless distraction at a time when all hands are overloaded with initiatives to address the pandemic.

A related HHS policy would waive FDA premarket review for certain medical devices. This extends the current review exemption under the COVID-19 health emergency for items such as gloves, personal protective equipment, ventilators, and infusion pumps. Not a critical change, but it is regarded as another challenge to FDA internal policy making.

In addition, the battle over FDA authority to regulate laboratory developed tests (LDTs) continues to create problems. A contentious issue for several years, HHS revoked FDA’s authority over such tests in August in response to pressure from private labs for reduced regulation. However, in November, HHS reinstated FDA’s authority to issue Emergency Use Authorizations (EUAs) for lab tests in order to provide companies with liability protection for erroneous results during the pandemic. By then, however, FDA staff claimed a lack of resources to handle this activity, prompting a shift to outsourcing the EUA reviews.

The most contentious challenge to FDA’s authority, though, involves a late move by the Trump administration to shift oversight of genetically modified animals to the Department of Agriculture, a move backed by industry working in this area. Commissioner Hahn evidently refused to sign an HHS memorandum of understanding authorizing the change, claiming that it required further analysis of the legal and health repercussions.

On another front, FDA staff has had to accept administration efforts to limit utilization of prescription drugs to induce an abortion. In March 2020, FDA was able to relax in-person dispensing requirements for many drugs, including certain controlled substances to reduce the risk of spreading COVID-19. However, the Trump administration left in place the specific rule requiring women to make in-person visits to a clinic or doctor’s office to obtain mifepristone, one of two drugs required for a medication abortion. Even though a US District Court judge suspended the in-person pick-up rule during the health emergency, the administration continued to fight for its reinstatement and recently gained a ruling to that effect from the Supreme Court.

Many of these recently issued regulations and policies can be rescinded or dropped by the Biden administration under established transition practices. However, these efforts by the Trump administration to limit FDA’s authority have rekindled calls for FDA to become an independent regulatory agency, separate from HHS.

As he prepared to exit the agency amidst all the political infighting, Commissioner Hahn joined with Anand Shah, deputy commissioner for medical and scientific affairs, in issuing a 

report outlining FDA’s important achievements 

in combatting the “devastation and turmoil” wrecked by the COVID-19 pandemic. The report highlights the agency’s accomplishments and offers recommendations for continuing to address the still-evolving public health emergency. To improve FDA’s emergency response and ability to sustain innovation, it calls for strengthening the EUA process, government partnerships, innovation in clinical trial conduct, and supply chain surveillance, among many other strategies. Hahn and Shah emphasize that FDA decisions are based on rigorous science and aim to protect and promote the health and safety of American patients.

As the Biden administration readies to take over the reigns of government, top administration officials continue to rush through new rules and decisions that appear to challenge the independence and effective operations of FDA. 

FDA Struggles to Fend Off Further Attacks from Trump Administration

Cytiva announced on Jan. 14, 2021 that it has completed a KUBio installation for Lonza in the form of a high throughput modular biomanufacturing facility in Guangzhou Biopark, China.

 facility will serve as a process development and manufacturing site for Chinese companies developing medicines, as well as multinationals with manufacturing requirements in China, Cytiva said in a company press release. The site will feature 6500 m

 of lab space and one KUBio modular facility, which will accelerate antibody development services and manufacturing.

“The Cytiva team showed great agility and speed in delivering this important site,” said Stefan Stoffel, chief operating officer, Lonza, in the press release. “With the KUBio and FlexFactory single-use platform now ready for business, Lonza will be able to respond to the great demand for innovative therapies in the current environment, now and in the future.”

“In a rapidly-growing industry with increasing needs for mAbs [monoclonal antibodies] and other key biopharmaceuticals, this facility demonstrates the strength of combining modular manufacturing with single-use technologies,” added Olivier Loeillot, vice-president, Cytiva, in the press release. “Together, the KUBio and FlexFactory speed up delivery of innovative therapeutics to patients who need them.”

The 17,000 m2 facility will serve as a process development and manufacturing site for Chinese companies developing medicines as well as multinationals with manufacturing requirements in China.

Cytiva Delivers Chinese Biomanufacturing Facility to Lonza

CMIC, a Japanese contract resource organization (CRO), announced on Jan. 12, 2021 that it has joined the Decentralized Trials and Research Alliance (DTRA), an organization aiming to accelerate the adoption of decentralized clinical trials, as a founding company.

By joining as a founding company, CMIC will become a part of the DTRA Leadership Council and will begin advancing decentralized trials in Japan by establishing its connection with organizations and companies in the United States, CMIC said in a company press release.

“By joining the Leadership Council, CMIC will be able to study real-world examples of decentralized clinical trials that are led in the US, and network with US organizations,” said Toru Fujieda, president of CMIC, in the press release. “Through this, CMIC will be able to formulate realistic and efficient studies within the Japanese clinical trial environment and propose solutions for challenges that arise during a trial. As a member of DTRA, we will leverage the latest global digital health technology to the fullest and contribute to smooth decentralized trial conduct in Japan. Furthermore, as the largest Japanese CRO with a broad Asian footprint, we will bring our decentralized trial adoption experiences, trends, and needs of the fast-growing Asian market into DTRA, in order to facilitate the development of future global standards.”

CMIC will work to advance decentralized trials in Japan.

CMIC Joins DTRA as a Founding Company

Catalent announced on Jan. 13, 2021 that it will acquire the manufacturing and packaging operations of Acorda Therapeutics, a US-based biotechnology company.

Through the agreement, Catalent will have access to Acorda’s 90,000-ft2 Chelsea, MA facility, which will serve as a global center of excellence in the Catalent network for spray-dried dispersion and dry powder encapsulation and packaging, Catalent said in a company press release. Additionally, Catalent will handle the commercial manufacture of Acorda’s INBRIJA, a prescription medicine used to treat hypomobility or off episodes in adults with Parkinson’s disease treated with regular carbidopa/levodopa medicine.

"Catalent is a premier partner to companies working on multiple forms of inhaled drug delivery, from early-stage development to commercial-scale supply," said Jonathan Arnold, president of Oral and Specialty Delivery at Catalent, in the press release. “This acquisition complements our existing US-based capabilities in metered-dose and nasal inhalation by adding an experienced team of people and a premier facility enabling us to provide customers with extended capabilities for commercial-scale filling and packaging of dry powder inhalers.”

Acorda’s Chelsea, MA facility will serve as a global center of excellence in the Catalent network for spray-dried dispersion and dry powder encapsulation and packaging.

Catalent to Acquire Acorda Therapeutics’ Manufacturing and Packaging Operations

Lonza and Capricor Therapeutics, a US-based clinical-stage biotechnology company, announced on Jan. 12, 2020 that they are entering into an agreement for the development of CAP-1002, Capricor's clinical cell therapy that uses allogeneic cardiosphere-derived cells technology for the treatment of Duchenne Muscular Dystrophy. The therapy is also in a clinical trial to treat complications arising from COVID-19.

Under the terms of the agreement, operations will be initiated at Lonza’s Houston, TX Center of Excellence where Lonza will handle process development activities for late-clinical and commercial-scale GMP manufacturing of the cell therapy, Lonza said in a company press release. Additionally, the collaboration will work to expand Capricor’s manufacturing capacity for possible late-stage clinical trials and commercialization.

“As we continue to expand our manufacturing efforts for CAP-1002, our lead cell therapy product candidate, this collaboration with Lonza provides us with a partner which has world-class expertise in technology transfer and an established track record of commercializing biologics,” said Linda Marbán, CEO, Capricor Therapeutics, in the press release. “We are excited because this is an important step in our ability to potentially bring CAP-1002 closer to commercialization and allows us to bring this important therapy to patients with Duchenne Muscular Dystrophy as quickly as possible, if approved.”

“Capricor’s lead candidate CAP-1002 is demonstrating efficacy in late-stage clinical studies to significantly benefit patients,” added Alberto Santagostino, senior vice president, head of Cell and Gene Technologies, Lonza, in the press release. “We will leverage our process development expertise and industrial manufacturing capabilities to enable Capricor to scale this therapy and make it available to patients globally, once approved for commercialization.”

Capricor also received FDA acceptance of an investigational new drug application for a phase II clinical trial of CAP-1002 in patients with COVID-19, in August 2020.

Under the terms of the agreement, operations will be initiated at Lonza’s Houston, TX Center of Excellence where Lonza will handle process development activities for late-clinical and commercial-scale GMP manufacturing of the cell therapy.

Lonza to Partner with Capricor Therapeutics for the Development of Duchenne Muscular Dystrophy Cell Therapy 

Sanofi announced on Jan. 11, 2021 that it is acquiring Kymab, a UK-based clinical-stage biopharmaceutical company, for a total of $1.4 billion.

Under the terms of the agreement, Sanofi will provide an upfront payment of $1.1 billion and will provide an additional $350 million if certain milestones are achieved, Sanofi said in a company press release.

The acquisition will give Sanofi full global rights to KY1005, Kymab’s fully human monoclonal antibody that binds to OX40-Ligand, giving it the potential to treat a variety of immune-mediated diseases and inflammatory disorders, according to the press release.

“The Kymab acquisition adds KY1005 to our dynamic pipeline, a potential first-in-class treatment for a range of immune and inflammatory diseases. The novel mechanism of action may provide treatment for patients with suboptimal responses to available therapies,” said Paul Hudson, Sanofi CEO, in the press release. “We understand from our ongoing work in debilitating immunological diseases how critical it is to find the right treatment for each patient. We look forward to rapidly developing this investigational medicine.”

“The agreement is a testament to the commitment, drive, and expertise of the entire Kymab team and we are pleased to receive this endorsement from Sanofi,” added Simon Sturge, CEO, Kymab, in the press release. “With its significant global resources, we believe Sanofi is the perfect partner to progress Kymab’s pipeline of products and the merger will expedite the time it takes for our novel therapies to get to patients.”

The acquisition will give Sanofi full global rights to KY1005, Kymab’s fully human monoclonal antibody that binds to OX40-Ligand, giving it the potential to treat a variety of immune-mediated diseases and inflammatory disorders. 

Sanofi to Acquire Kymab for $1.4 Billion

Baxter BioPharma Solutions and Novavax announced on Jan. 11, 2021 that they have entered into a sterile manufacturing agreement for Novavax’s COVID-19 vaccine candidate, NVX-CoV2373.

Under the terms of the agreement, Baxter will provide commercial-scale manufacturing services at its Halle/Westfalen, Germany, facility to prepare the vaccine candidate for distribution in the United Kingdom and European markets, Baxter said in a company press release. NVX-CoV2373 is currently undergoing Phase III trials and has not yet been authorized or approved for use.

“The quest to develop vaccines for COVID-19 has reinforced the opportunity for industry partners to work together and contribute their unique capabilities and expertise for the benefit of all,” said Marie Keeley, vice-president, Baxter BioPharma Solutions, in the press release. “We welcome the opportunity to work with an innovative company like Novavax and look forward to helping bring their vaccine candidate to the market.”

“Our priority is to bring a safe, effective COVID-19 vaccine to people around the world,” added Rick Crowley, executive vice-president, chief operations officer, Novavax, in the press release. “Partners like Baxter BioPharma Solutions are enabling Novavax to quickly establish a commercial supply chain network to ensure access for global populations, and ultimately help bring about an end to the global COVID-19 pandemic.”

Baxter will provide commercial-scale manufacturing services at its Halle/Westfalen, Germany facility to prepare Novavax’s COVID-19 vaccine candidate for distribution in the United Kingdom and European markets.

Baxter and Novavax Enter into COVID-19 Vaccine Sterile Manufacturing Agreement

 providing sponsors of human gene therapy (GT) products for neurodegenerative diseases information on product development, preclinical testing, and clinical trial design. Marketing approval pathways are also discussed in the document. Neurodegenerative diseases are disorders where the central nervous system or peripheral nervous system degenerates.

Specifically, the guidance addresses chemistry, manufacturing, and controls (CMC) considerations and states aspects of GT products for neurodegenerative diseases may have particular CMC considerations. The guidance emphasizes product design should be based on current scientific knowledge and GT products should be designed to reduce inflammatory immune responses. Considerations for drug product purity, identity, and strength are also emphasized.

The guidance also discusses aspects of preclinical studies, clinical trials, expedited programs, and communications with FDA.

The draft guidance document offers developers of these treatments information regarding product development, preclinical testing, and clinical trial design.

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