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Vial and syringe with a vaccine

Clover Biopharmaceuticals, a clinical-stage firm developing a Covid-19 vaccine with potential manufacturing and distribution advantages over other vaccine technologies, has raised $230 million as it prepares to advance its lead candidate into pivotal testing.

Chengdu, China-based Clover said Tuesday that it expects to start a global Phase 2/3 clinical trial for its vaccine candidate, SCB-2019, in the first half of this year. The company added that it has already started planning for the production of potentially hundreds of millions of vaccine doses.

The Clover Covid-19 vaccine is protein-based. SARS-CoV-2, the virus that causes Covid-19 infection, is an enveloped RNA virus—the outer coating is dotted with spike proteins that bind to a receptor on the host cell. These spikes are trimeric, meaning they’re formed by three proteins.

Using its Trimer-Tag technology, Clover developed a trimeric spike protein that resembles the one found on the outer envelope of the novel coronavirus. The vaccine uses an adjuvant, an ingredient that boosts immune response, supplied by Dynavax Technologies.

As a protein-based vaccine similar to many of the vaccines developed for influenza, shingles, and hepatitis B, Clover said production can use manufacturing processes that are already well established. The company added that this production can be rapidly scaled up to large quantities.

Another advantage of the Clover vaccines are the temperature requirements. The company said its vaccines and adjuvant should be stable for long periods at refrigerator temperatures of 2 to 8 degrees Celsius. At room temperature, Clover has said its vaccines are stable for at least two months. Those temperature and storage requirements stand in contrast to messenger RNA vaccines, which must be distributed frozen and stored at temperatures well below what medical-grade freezers can achieve. Last week, Pfizer and BioNTech asked the FDA to approve a change in the storage temperatures permitted once vaccines reach a vaccination site.

The new financing follows publication in The Lancet earlier this month of peer-reviewed results from an early-stage test of two Clover Covid-19 vaccine candidates. The 150-patient study showed that the vaccines were well tolerated and safe. Both vaccines also induced neutralizing antibodies at levels comparable to or higher than those found in the blood of those who have recovered from Covid-19.

Clover said that its research includes vaccines that could address multiple variants of the novel coronavirus. In addition to supporting its Covid-19 vaccines, Clover said the new capital will support plans to advance multiple programs into human testing later this year. Other vaccines in the Clover pipeline include programs for rabies and influenza. The company also said it plans to expand its manufacturing and capabilities.

GL Ventures and Temasek both led the Series C financing. Oceeanpine Capital, OrbiMed, and Delos Capital also invested. Clover said it has raised more than $400 million in the past year.

Clover also has financial support from the Coalition for Epidemic Preparedness Innovations (CEPI), which has committed to finance development of the company’s Covid-19 vaccine candidate up through licensure with a total investment of $328 million. Some of that cash will fund the global Phase 2/3 study. If the Clover vaccine is shown to be safe and effective, it would be distributed through Covid-19 Vaccines Global Access, or COVAX, the World Health Organization’s initiative to ensure equitable vaccine access throughout the world.

Public domain photo by Flickr user Alachua County

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Pfizer and BioNTech are asking the FDA to approve a change that would permit storage of their Covid-19 vaccine within a temperature range found in medical-grade freezers.

To be clear, this proposed change doesn’t eliminate the need for ultra-cold temperatures as the vaccine moves throughout the supply chain—temperatures that require specialized equipment. Those storage requirements are a barrier to its distribution because not all facilities have equipment that can achieve the required temperatures. What Pfizer and BioNTech are proposing is that when the vaccine reaches its destination, such as a hospital or pharmacy, it could be stored for up to two weeks at (relatively) warmer freezer temperatures.

The FDA granted emergency use authorization to the messenger RNA vaccine, named Comirnaty, last December. According to the product’s label, the vaccine must be stored in ultra-cold temperatures between minus 112 degrees and minus 76 degrees Fahrenheit (minus 80 degrees and minus 60 degrees Celsius). At those temperatures, the vaccine can last up to six months. Pfizer ships the vaccines in specially designed containers that can be refilled with dry ice every five days. Those containers can serve as temporary storage for up to 30 days.

The vaccine can be stored at refrigerator temperatures for up to five days. Pfizer and BioNTech are asking the FDA to approve an additional option to store the vaccine at minus 13 degrees to 5 degrees Fahrenheit (minus 25 degrees to minus 15 degrees Celsius) for two weeks. That range would put it closer to the storage requirements for the mRNA vaccine from Moderna. The temperature range Pfizer and BioNTech are proposing would be in addition to the five days at refrigerator temperatures that is currently permitted under the authorization.

“If approved, this new storage option would offer pharmacies and vaccination centers greater flexibility in how they manage their vaccine supply,” Pfizer CEO Albert Bourla said in a prepared statement.

Pfizer and BioNTech are proposing the additional temperature option based on new data about their vaccine’s stability. The data span from the earliest clinical trials up to batches currently in production.

Messenger RNA vaccines are a new technology that employ a snippet of genetic material from the novel coronavirus. This mRNA serve as a blueprint that a cell’s protein-making machinery use make the characteristic spike protein found on the surface of the novel coronavirus. That protein is what triggers an immune response and confers immunity.

Though mRNA vaccines are administered at room temperature, they must be kept at ultra-cold temperatures in the supply chain because mRNA is fragile. The extremely cold temperatures keep the components of the vaccine from breaking down. As mRNA companies study the storage data they have for vaccines, they are getting a better understanding of how long these vaccines can last at certain temperatures. Last November, Moderna released data that it said supported storage of its vaccine at refrigerator temperatures for up to 30 days.

Temperature requirements can be a barrier to the distribution of the vaccine to rural areas, which may not have facilities with appropriate freezers. In that regard, the Johnson & Johnson Covid-19 vaccine is seen as offering an advantage. In addition to requiring only a single shot (both authorized mRNA vaccines require two), the J&J vaccine can be stored at standard refrigerator temperatures. An FDA advisory committee is scheduled to hold a hearing about that vaccine on Feb. 26.

Photo by BioNTech

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Johnson & Johnson has formally asked the FDA to allow emergency use of its Covid-19 vaccine as the company seeks to add a third option to the U.S. lineup of vaccines for the novel coronavirus.

The application comes a week after J&J released preliminary data showing 66% overall efficacy in a Phase 3 clinical trial. Those results fall short of the efficacy marks of messenger RNA (mRNA) vaccines from Moderna and Pfizer. But the J&J jab offers advantages compared to those vaccines.

Unlike mRNA vaccines, which are given as two doses weeks apart, J&J’s vaccine is a single shot. Also, the J&J vaccine can be kept at refrigerator temperatures. Both the Moderna and Pfizer vaccines must be kept frozen—Pfizer’s at ultra-cold temperatures. They’re then thawed and temporarily stored at refrigerator temperatures before dosing.

J&J’s vaccine storage requirements are the same as those for most vaccines, making it an easier fit vaccine distribution channels already in place. The company said that if authorized, its vaccine will ship using the same cold chain technologies it uses for transporting other medicines. Of the vaccine candidates that have advanced to late-stage testing, J&J’s is the only one given as a single dose.

Before the FDA decides whether to grant emergency use authorization to the J&J vaccine, the pharma giant’s candidate must be evaluated by an independent advisory committee to the agency that will evaluate the clinical data and discuss the efficacy and safety risks of the shot. The vaccines from both Moderna and Pfizer went through the same step. The J&J meeting is scheduled for Feb. 26.

J&J’s vaccine, developed by the company’s Janssen division, employs a version of the virus that causes the common cold. That virus is modified so it does not cause illness. It’s used to deliver to cells a snippet of the genetic code for the spike protein, which is prominent on the surface of the novel coronavirus. The cells of the body read that genetic material and make copies of the spike protein. The immune system responds to those copies by making antibodies that protect against Covid-19.

AdVac is the same platform Janssen used to develop an Ebola vaccine that was approved by the FDA in 2019. The technology is also the foundation of experimental Zika, RSV, and HIV vaccines. J&J said that the safety profile observed with its Covid-19 vaccine was consistent with other experimental vaccines based on AdVac.

J&J evaluated its Covid-19 vaccine in a Phase 3 study enrolling 43,783 patients. The main goal was to show protection from moderate to severe disease. There were geographic differences in efficacy rates. The vaccine candidate was most protective in the U.S., where efficacy was 72%, the company said. In Latin America, efficacy was 66%; in South Africa, it was 57% effective. While those marks fell short of the efficacy rates demonstrated by the Moderna and Pfizer vaccines, cross trial comparisons are tricky. Also, the J&J studies were done when more variants of the novel coronavirus were circulating compared to when the Moderna and Pfizer vaccines were tested.

Paul Stoffels, J&J’s chief scientific officer, said in a prepared statement that the company has vaccines ready to ship immediately upon receiving emergency authorization.

“With our submission to the FDA and our ongoing reviews with other health authorities around the world, we are working with great urgency to make our investigational vaccine available to the public as quickly as possible,” he said.

In addition to the submission to the FDA, J&J is seeking similar authorizations from health agencies in other countries. The company said an application to the European Medicines Agency will be submitted in coming weeks.

Photo: Teka77, Getty Images

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Voyager Therapeutics is losing Neurocrine Biosciences as a research partner on an experimental Parkinson’s disease treatment, the latest in a string of setbacks for the biotech’s efforts to develop gene therapies addressing neurological disorders.

Cambridge, Massachusetts-based Voyager disclosed late Tuesday that Neurocrine provided a termination notice on the Parkinson’s candidate NBlb-1817, which is currently in mid-stage clinical testing. The decision follows the FDA’s December decision to place a clinical hold on that program due to safety concerns.

Termination of the partnership on the Parkinson’s gene therapy will be effective Aug. 2. The collaboration agreement requires Neurocrine to provide 180 days written notice of a termination. The San Diego biotech acknowledged providing that notice in its own regulatory filing. Three other programs covered by the agreement, one for Friedreich’s ataxia and two others in the discovery stage, are not affected by Tuesday’s decision and will continue, Voyager said.

The alliance began in 2019 when San Diego-based Neurocrine pledged $165 million in cash and stock to Voyager. Of the four programs covered by the pact, the Parkinson’s candidate was the most advanced. According to the deal terms, Neurocrine was responsible for funding Phase 2 clinical development. After the study produced data, Voyager held the option to split the rights to the gene therapy with Neurocrine, sharing in further development costs. Alternatively, Voyager could grant its partner full global rights in exchange for milestone payments pegged to sales.

Prior to the Covid-19 pandemic, Neurocrine anticipated advancing the Parkinson’s gene therapy to a pivotal study in the second half of 2020, the company said in its annual report. The pandemic and the clinical hold stalled that timeline.

Voyager uses engineered viruses to deliver gene therapies to the brain. Parkinson’s is characterized by a lack of dopamine, a brain chemical that’s key to controlling muscle movement. Standard treatment includes prescriptions of levodopa, which is converted by a brain enzyme into dopamine.

As Parkinson’s progresses, a patient has less of that key enzyme in parts of the brain where it is needed to convert levodopa to dopamine, Voyager states in its filings. The Parkinson’s candidate is designed to deliver a gene directly into the neurons where dopamine receptors are located, providing the instructions the brain needs to make the key enzyme.

The Parkinson’s gene therapy is administered via a direct injection into the brain. For its amyotrophic lateral sclerosis and Friedreich’s ataxia programs, the company is exploring other approaches, including spinal or intravenous injections.

In its announcement of the end of the partnership in Parkinson’s, Voyager said Neurocine based its decision on a review of its portfolio and the prioritization of other programs in its pipeline. No mention was made about the safety of the Parkinson’s gene therapy. When Voyager disclosed the clinical hold in December, it said that a Neurocrine safety report noted MRI abnormalities in some study participants. It also said the independent board responsible for monitoring the safety of study participants requested a pause on dosing of patients until it could review additional data.

Until the alliance on NBIb-1817 is officially terminated in August, Neurocrine is the company of record for the clinical program. Voyager said that last month, the FDA informed Neurocrine of the information needed to respond to the clinical hold. In addition to an assessment of how the therapy may have contributed to the adverse findings, Voyager said the agency wants a mitigation plan to manage them along with supportive data to justify that the benefit of the therapy outweighs its risks.

Voyager said it will support Neurocrine on any imaging or clinical assessments requested by data safety monitors of the study, as well as any information sought by the FDA. The company added that it is evaluating the financial effect the termination will have on the company and the future of the Parkinson’s program.

The end of the partnership on the Parkinson’s gene therapy marks the second time a company has passed up the opportunity to advance that program. Sanofi was Voyager’s first partner on the experimental therapy, committing $100 million up front in 2015 for rights to several gene therapies for brain disorders. At the time of the deal, the Parkinson’s gene therapy was in early-stage testing. In 2017, Sanofi returned the therapy to Voyager after the biotech declined to amend the original deal to grant the pharma giant a share of the U.S. rights to the program.

The loss of Neurocrine as a partner in Parkinson’s comes a little more than six months after an alliance with AbbVie ended. That partnership focused on developing gene therapies for Alzheimer’s and Parkinson’s. AbbVie terminated the alliance before either program reached Phase 1 testing, at which point Voyager would have become eligible for additional payments from the North Chicago, Illinois-based pharma company.

Photo: SIphotography, Getty Images

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A major hurdle to quick and efficient vaccine distribution could be the proliferation of “pharmacy deserts” across the country.

There are 56,802 local and chain pharmacies, which evens out to about 1.83 pharmacies per 10,000 people on average, according to a new report from GoodRx. But these facilities are unevenly distributed leaving many residents in “pharmacy deserts.” These are areas where people have to drive a long distance to reach the closest pharmacy or areas with too many people sharing the same pharmacy.

Pharmacy deserts may have dire implications for the Covid-19 vaccine rollout, according to a report from GoodRx, which tracks U.S. prescription drug prices and provides free discount coupons. Since pharmacies are playing a key role in administering the vaccine, living in a pharmacy desert may prevent Americans from getting vaccinated in a timely manner.

Two vaccines have been approved in the U.S., one developed by Pfizer and BioNTech, and the other by Moderna. Both are two-dose vaccines. So far, the rollout has been characterized by missteps and is moving slowly, with only 17 million vaccine doses administered out of nearly 38 million distributed as of Thursday, according to data from the Centers for Disease Prevention and Control. But President Joe Biden has promised to speed up the process — vowing to administer 100 million Covid-19 vaccine shots in the first 100 days of his administration.

GoodRx examined pharmacy deserts using information from the National Council for Prescription Drug Program’s DataQ and the GoodRx databases. Researchers also used data from the American Community Survey to calculate population per county.

Both rural and urban areas can have pharmacy deserts, according to the report.

Pharmacy deserts in rural counties are more likely to be characterized by a lack of pharmacy resources. About 177 U.S. counties have zero pharmacies, leaving 635,000 residents with no choice but to drive a long distance to access one.

On the other hand, pharmacy density is a bigger issue for Urban Americans, the report shows. For example, there are 10 million people living in Los Angeles County in California, but there are only 1.61 pharmacies per 10,000 residents. Cook County in Illinois, which has 5.2 million residents, has only 1.50 pharmacies per 10,000 residents. Both fall below the national average of 1.83 pharmacies per 10,000 residents.

Many pharmacy deserts are also vaccination deserts, the report states.

For instance, assuming the promised 100 million vaccines are distributed, only 11.2% of the population in San Mateo County, California, will receive both doses of the Covid-19 vaccine, researchers estimate, as the county only has 1.06 pharmacies per 10,000 residents.

Other similar vaccine deserts include Shelby County, Tennessee, El Paso County, Texas and Pinal County, Arizona.

The researchers conclude that administering the Covid-19 vaccine through pharmacies alone “is not going to cut it.” Additional mass vaccination sites are going to be needed as well as support services in more rural counties, like transportation or in-home vaccinations.

Biden’s administration has already heeded the call for more vaccination sites. The president’s vaccine distribution plan, unveiled in a speech last week, includes plans to mobilize the Federal Emergency Management Agency and the National Guard to ramp up distribution, according to STAT News.

Photo: LarisaBozhikova, Getty Images

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The Covid-19 pandemic may fade but some of the changes it has wrought in the life sciences may – or should – endure, according to two prominent pharma CEOs who spoke during a session Friday at the J.P. Morgan Healthcare Conference.

For starters, the pandemic has shown how quickly companies and regulators can bring products to market, with tests, vaccines and therapies being studied and approved at record speeds, according to the CEOs, Paul Hudson of Sanofi and Tom Polen of BD.

Companies also have collaborated in ways they would not have contemplated previously. Hudson cited Sanofi’s work with GSK to develop a Covid-19 vaccine. The partnership might once have taken a year to come together but took only 12 days, Hudson said.

“So many times, it was a race against the virus, not a race against each other,” said Hudson, who became Paris-based Sanofi’s CEO in fall 2019 after leaving Novartis.

Regulators have moved at similar speeds, delivering answers in minutes rather than months, Hudson said.

The fast pace stems from the pressure of Covid-19, which has killed two million people around the world and is continuing to spread and mutate. But the sense of urgency could be applied in the future to tackle other diseases, like childhood cancers.

“I think the industry is at its best when it’s purpose-driven with a singular focus,” Hudson said during the JPM session, which was moderated by managing directors at Boston Consulting Group.

Large pharma companies, meanwhile, showed that they could pivot and innovate quickly, despite stereotypes to the contrary. Polen cited BD’s effort to develop a rapid-antigen test for Covid-19. It went from launch to monthly production of 10 million units within six months, said Polen. It normally would take three years.

The speed was a function of setting a clear mandate and a deadline but also doing things differently inside the company, Polen said. While the test was a life science project, for example, BD plucked talent from across the organization to lead the charge.

Another key was empowering and supporting employees so that they were not afraid of taking risks. Early on, the team found a promising antibody, one that is now used in the test, Polen said. The normal progression would be to order a small batch, wait to see if it works and then start ordering batches for commercial production. BD executives assured employees they were OK with ordering the commercial batches up front despite the risk and cost.

“That ended up being a tremendous part of the success equation,” Polen said

The pandemic was not the only force reshaping the industry in 2020. Hudson and Polen discussed the lasting impact they hope to see from the racial justice protests that followed the death last year of George Floyd, an African American, at the hands of Minneapolis police officers.

“This year brought a whole new and very much-needed focus on this issue for us as BD,” said Polen, who became CEO of the Franklin Lakes, New Jersey-based company in January 2020.

The company’s leaders have worked to create an environment where employees feel comfortable broaching difficult topics around race, Polen said. “That was a big change this year.”

Harris noted the need to improve diversity among patients in clinical trials but also among the investigators.

For 2021, Harris and Polen expect a sharper competition for talent in the life sciences.

“I think it’s going to escalate after we get out of Covid,” Polen said. But some of the workplace changes wrought by the pandemic could prove useful in the competition. Employees that once might have had to move for a promotion or a new job now can stay where they are and work remotely, an option that could help dual-income families.

Polen and Harris also discussed the growing role in health care for big tech companies like Amazon, Apple and Google. The CEOs painted the tech giants as potential partners rather than rivals. Not only can tech companies help bring products to patients, they also tools to transform pharma’s internal operations.

The challenge ahead lies in preserving the energy, purpose and speed that animated the industry in 2020, Hudson added. “People can quickly fall back into the old routines.”

Photo: Dmitrii_Guzhanin, Getty Images

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A second vaccine for Covid-19 has won the blessing of U.S. regulators.

The U.S. Food and Drug Administration on Friday approved for emergency use a vaccine developed by Moderna, a biotech company based in Cambridge, Massachusetts.

The green light comes a week after similar approval was given to a vaccine from New York-based Pfizer and its German partner, BioNTech. That first vaccine is already being deployed on the front lines of a pandemic currently claiming more than 3,000 lives a day in the U.S. and stretching the country’s health care resources to a breaking point.

The approval of Moderna’s vaccine means it, too, will soon join the fight against Covid-19. The U.S. has committed to buying 200 million doses of the vaccine, which is administered in two shots, according to the company. Hospitals are expecting deliveries as soon as next week.

“With the availability of two vaccines now for the prevention of Covid-19, the FDA has taken another crucial step in the fight against this global pandemic that is causing vast numbers of hospitalizations and deaths in the United States each day,” FDA Commissioner Dr. Stephen M. Hahn said in a statement.

The initial shipment from Moderna is expected to total 5.9 million doses, more than double the initial 2.9 million doses of the Pfizer/BioNTech vaccine.

Further, the Moderna vaccine may pose less of a logistical challenge. Unlike the Pfizer/BioNTech product, it does not need to be stored at extremely low temperatures, enabling it to go more places. 

Moderna plans to make 20 million doses available by the end of December.

“We remain focused on scaling up manufacturing to help us protect as many people as we can from this terrible disease,” Moderna CEO Stephane Bancel said in a statement. He said the vaccine, while developed quickly, is based on a decade of research.

The Moderna and Pfizer/BioNTech vaccines are based on a similar platform: messenger RNA, which helps human cells create their own antibodies to the Covid-19 virus. Developed in record time, both vaccines are around 95% effective but generate some temporary side effects, including pain at the injection site, fever, joint pain and headaches. Both also require two doses.

More severe reactions are possible as the vaccines are given to a larger population. Two health care workers in Alaska reportedly developed allergic reactions to the Pfizer vaccine, with one requiring hospitalization. This was also observed in the U.K. 

The Pfizer/BioNTech vaccine is approved for use in people 16 and over, though members of an FDA advisory panel questioned whether there was enough evidence to support use in those under 18. The Moderna vaccine is approved for those 18 and over.

The age difference led to different outcomes for the vaccines when they came before an FDA advisory panel comprised of outside experts. Panelists were voting on whether the vaccines’ benefits outweighed the risks. The Pfizer/BioNTech vaccine was cleared on a 17 to four vote, with one abstention, while the Moderna vaccine was cleared unanimously, also with one abstention. The abstention reflected concern about the broad scope of the question up for approval

“I’m very uncomfortable with the language,” said Michael Kurilla, an infectious disease expert and director of the clinical innovation division at the National Center for Advancing Translation Sciences, a part of the National Institutes of Health.

“I think in the midst of a pandemic and with limited vaccine supply available, a blanket statement for individuals 18 years and older is just too broad,” Kurilla said at the end of the advisory panel meeting on Thursday. “I’m not convinced that for all of those age groups, the benefits do actually outweigh the risks and I would prefer to see it more targeted toward people at high risk of serious and life-threatening Covid disease.”

Another critical question facing the vaccine makers is what to do about people taking part in clinical trials, some of which are expected to last into 2022. The trials are comparing the results in those given the actual vaccine against those given a placebo. Participants are not supposed to know which one they received.

But on a website about their vaccine, Pfizer and BioNTech said they are allowing people who got a placebo in their trial to get the actual vaccine. The offer is limited initially to healthcare workers and nursing-home residents, two groups that have been prioritized for vaccination in the U.S. But it will eventually extend to others. There are nearly 44,000 participants in the Pfizer/BioNTech trial.

Moderna also has a plan for the so-called unblinding of its trial participants, of which there are more than 30,000.

The next vaccine candidates are expected to come before regulators early next year. One candidate is being developed by AstraZeneca and Oxford University. Another is from Johnson & Johnson, which said this week it expects to ask for emergency use authorization in February.

Photo: Michael Burrell, Getty Images

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A San Diego-based biopharma company has raised $200 million to advance work on its genetics-based therapies, which target cancer, cardiovascular disease and other conditions.

The investment represents the biggest yet for Ambrx Inc., which has several cancer therapies in its clinical pipeline.,

“The conclusion of this financing and the strong data emerging from our ongoing clinical programs, as well as our deep preclinical pipeline of proprietary drug candidates, positions the company for rapid growth,” said Dr. Fang Tien, the company’s CEO and chairman, in a statement.

Ambrx relies on proprietary technology using what it describes as an expanded genetic code to develop precision biologics, which can include antibody drug conjugates, bispecifics and targeted immuno-oncology therapies.

Investors in the $200 million round include Fidelity Management & Research Co., Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management, as well as funds and accounts managed by BlackRock.

Ambrx also has struck partnerships with larger drug companies.

In January, it said it was collaborating with Sino Biopharma to develop and commercialize two drug candidates for cancer. Terms of the deal were not disclosed but it provided Ambrx with an upfront payment for its work to develop the two drugs. Sino Biopharma will handle the application process for the drugs as investigational therapies in the U.S. and China.

The Ambrx pipeline, meanwhile, includes therapies in various clinical stages developed in partnership with the likes of Bristol-Myers Squibb, BeiGene and Astellas.

Ambrx also has worked with Elanco to bring multiple animal health therapies to market.

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Dementia or brain damage and injury as a mental health and neurology medical symbol with a thinking human organ made of crumpled paper torn in pieces as a creative concept for alzheimer disease.

In a meeting Friday, outside advisers to the Food and Drug Administration took a dim view of studies supporting what could be the first new drug for Alzheimer’s disease in nearly 20 years.

Over more than six hours, an 11-member advisory committee of outside experts raised numerous concerns about the data underpinning aducanumab, a drug developed in a partnership between Cambridge, Massachusetts-based Biogen and Japan-based Eisai.

In a final vote, 10 members of the advisory committee panel said the studies so far did not support the drug’s effectiveness, though FDA had argued otherwise in a presentation the panel. The 11th member registered an “uncertain” vote.  Though the vote is nonbinding, as is customary, FDA will consider the panel’s recommendations as the agency reviews the drug’s application for approval.

In a statement sent to MedCity News, Biogen’s CEO thanked patients and others for speaking at the FDA meeting. 

“Biogen thanks the many patients and advocates who shared their personal thoughts and experience at today’s Advisory Committee meeting, reflecting the significant unmet need for a treatment for Alzheimer’s,” said Michel Vounatsos said. “We appreciated the opportunity to share our data with the Advisory Committee, and we will continue to work with the FDA as it completes its review of our application.” 

Aducanumab  aims to slow the build-up of amyloid beta plaque in the brain, a key feature of Alzheimer’s disease. Previous therapies with the same goal have largely proven futile.

The drug from Biogen and Eisai appeared headed for the same dustbin in spring 2019. The companies halted a pair of clinical trials after it was determined they were likely to fail. The trials involved patients with mild cognitive impairment and dementia due to Alzheimer’s.

But the drug gained new life in October 2019 when Biogen unveiled data from the trials showing the drug appeared to work when taken in higher doses. 

Patients and caregivers acknowledged the drug may not be perfect. But they said it represented hope for people suffering from Alzheimer’s and a spur for continued research in the field.

One speaker – Dr. Stephen Salloway, an Alzheimer’s researcher and professor at the Warren Alpert Medical School of Brown University in Rhode Island – compared aducanumab to AZT, an early treatment for HIV/AIDS that eventually gave way to more effective drugs.

“We are at a critical juncture in Alzheimer’s disease,” Salloway told the panel, adding that patients and their families don’t have time to wait another four or five years for more clinical studies.

Other speakers said they had taken part in clinical trials for aducanumab and that it had helped them.

FDA staff also took a positive view of the drug. In materials prepared ahead of the meeting Friday, the agency described the study involving higher doses as being “highly persuasive and capable of providing the primary contribution to a demonstration of substantial evidence of effectiveness of aducanumab.”

Scientists on the FDA advisory panel were largely unconvinced. They recognized the urgent need for new treatments for the fatal disease, which afflicts 5.8 million Americans and has no cure. But they were concerned about the precedent if they approved a drug based on studies they deemed inconclusive at best.

“I think there’s a huge danger to approving something that turns out not to be effective. I think that danger is much, much greater,” said panel member Dr. Joel S. Perlmutter, a neurology professor at the Washington University School of Medicine in St. Louis.

Photo: wildpixel, Getty Images

Update: This story has been updated with comment from Biogen 

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