Latest news on patient death in n-of-1 CRISPR trial; Good news for RSV; Vaccine R&D revolution; and more
05 Nov 2022
VaccineGene TherapyAntibodyGeneric DrugCell Therapy
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This is the rare week where Endpoints News is making news. If you haven’t heard, Drew Armstrong will be joining us from Bloomberg to become our new executive editor. We are thrilled to have him on board. Read founder and editor John Carroll’s note about our big announcement — and what that means for Endpoints.
Patient death in n-of-1 CRISPR trial
Almost two months after the nonprofit Cure Rare Disease received FDA clearance to administer its first-in-human CRISPR therapeutic for Duchenne muscular dystrophy, the nonprofit announced that the one patient in the trial, Terry Horgan, the brother of the nonprofit’s founder and CEO, Rich Horgan, passed away. Researchers are still trying to figure out what exactly happened with the single shot CRISPR therapy, the nonprofit said in a statement, and that process may take up to four months.
Good news for RSV
After years of efforts, companies working on the prevention of respiratory syncytial virus infections had a streak of good news to report. In a first, Pfizer said Phase III results show it can protect infants from RSV by vaccinating their mothers during pregnancy. GSK, on the other hand, scored priority review for the use of its vaccine among older adults. Then to close out the week, the EU officially approved nirsevimab, the antibody jointly developed by Sanofi and AstraZeneca, to prevent RSV disease in a broad group of infants.
Vaccine R&D revolution
The historic campaign to vaccinate the world against Covid-19 produced its first shot in 326 days. But can we do even better next time around? As the world attempts to strengthen vaccine R&D infrastructure to go even faster and more equitably distribute vaccines, with many congregating around CEPI’s moonshot goal of delivering a vaccine within 100 days, scientific, regulatory and manufacturing challenges persist. Here’s my deep dive and accompanying panel.
IRA weighs on pharma hivemind
President Joe Biden’s Inflation Reduction Act, which will open the door to Medicare drug price negotiation, is continuing to shape decision-making across the industry. Eli Lilly laid blame on the new law as the reason it scrapped a $40 million cancer drug — a BCL2 inhibitorBCL2 inhibitor — licensed from Fosun Pharma. Specifically, the company cited how the law changes the dynamics of small molecule R&D. Days later, Alkermes announced it would siphon off its burgeoning oncology business, in order to capitalize on biologics research. And while Amgen didn’t shift any directions yet, it warned of “material adverse effect” on sales.
Rubius implodes
Just weeks after Rubius, a one-time Flagship star, looked to restructure around a second try at making red blood cells a drug tech of the future, the biotech put out word that it’s axing the bulk of the remaining staff and looking for a sale or merger. With shares trading slightly north of 30 cents apiece, it won’t take much to take over the crumbling structure that once commanded a market valuation in the billions. Up for grabs: preclinical primate data and a drug — dubbed RTX-250 — marketed as an avenue to activate dendritic cells.
New mRNA player enters the fray
Australian pharma giant CSL is making a late bid for mRNA glory, and it lined up one of the also-ran US Covid-19 biotechs touting its own particular tech twist to partner with. CSL Seqirus, the big vaccines arm of the global player, shelled out $200 million in cash and put up another $4.3 billion in milestones — including a hefty $1.3 billion for development goals — to ally itself with Arcturus Therapeutics. Together they’ll work on Covid-19, flu, pandemic prep and three unnamed respiratory infectious diseases.
From private equity to venture
Just how much has private equity come to love biopharma during these stressful post-boom times and battered valuations? JP Morgan made a lavish entry into life sciences venture investing, tapping Alexion co-founder and ex-OrbiMed investor Stephen Squinto to lead a new group that promises to play advisor as well as financier to the companies they back. Life Sciences Private Capital is taking the field to go after “early and growth stage healthcare companies” with a special focus on drugs and tech.
Forget about IPO, let’s go for Series C
Elicio Therapeutics, an oncology and Covid-19 biotech led by a former Flagship Pioneering venture partner, is close to wrapping up its $40 million Series C after a bear market shut the doors to a planned IPO, Kyle LaHucik reports. The Boston biotech had sought to list on Nasdaq as $ELTX, with a draft registration statement filed in April 2021.
More backers for tRNA play
Leslie Williams’ plans to climb Mount Everest almost got in the way of her new tRNA biotech, but after Covid-19 disrupted those trips, she had time to go full throttle on securing the IP from the University of Iowa, hiring a core team and putting hC Bioscience together. Already backed by Takeda and ARCH, hC got more investors on board in a Series A extension that’s giving it another $16 million.
While Novo Nordisk plots a comeback for the obesity drug Wegovy, two early-stage programs in obesity and cardiovascular disease are on the chopping block. Due to portfolio considerations, the Danish drugmaker will be scrapping a Phase I obesity program dubbed LA-GDF15 and a PCSK9 inhibitorPCSK9 inhibitor that just completed a Phase II trial in patients with or at risk of developing atherosclerotic cardiovascular disease.
Thanks to higher-than-expected sales of Mounjaro (tirzepatide), Eli Lilly reported better-than-expected Q3 sales of $6.94 billion. As it pushes forward with plans to make the drug a megablockbuster, the company also discussed plans to double manufacturing capacity by the end of 2023 to meet “unprecedented” demand. It is, however, trimming its year-end guidance.
Regeneron revealed a 15% drop in revenue compared to Q3 last year, pulling in $2.94 billion after its Covid-19 antibody cocktail got pulled from the market. It also quietly disclosed that it’s cutting two drugs from its pipeline: a multi-antibody therapy, known as REGN1908-1909, to treat allergies in cats — and the Teva and Mitsubishi Tanabe-partnered NGF antibody fasinumab.
Pfizer suffered its first quarterly revenue dip since its BioNTech-partnered Covid vaccine was authorized in 2020 — and expects patent expiries to cost the company around $17 billion between 2025 and 2030. But CEO Albert Bourla emphasized “It’s all about what’s next,” as it outlined plans for bringing new drugs to the market as well as scoping out deals.
A decade after the crew that would become AbbVie teamed up with the French biotech Inventiva to find small molecule drugs that target RORγ for autoimmune diseases, AbbVie is winding down the resulting program, which is in Phase IIb for psoriasis, due to new findings observed in a preclinical chronic toxicology study.
Moderna’s stock plunged in premarket trading as CEO Stéphane Bancel reported a drop in Covid sales and lowered his full-year sales expectations due to delivery delays, short-term supply shortages and a “very complex third quarter from a manufacturing standpoint.” But the mRNA player reassured investors that it’s in a much better place now.
GSK touted record sales of shingles vaccine Shingrix, which contributed to total revenue of $8.9 billion. CEO Emma Walmsley made it “categorically clear” her team will be prioritizing dealmaking going forward. At the same time, tens of thousands of Zantac cases — with accusations that the heartburn drug caused cancer — are still waiting to be heard.
New approved indications and massive year-over-year sales growth have Daiichi Sankyo eyeing a year-end pop in sales for its AstraZeneca-partnered cancer drug Enhertu. The Japanese pharma upped its forecast for Enhertu this year by more than $400 million, projecting it could haul in about $1.3 billion.
R&D
J&J hit the brakes on one of its partner’s early-stage programs, halting enrollment in ESSA Pharma’s Phase I trial combining ESSA’s experimental drug with approved prostate cancer drugs from J&J. The biotech chalked the decision up to “operational recruitment challenges” and, in a follow-up, said it will explore testing the combo in a different population.
GSK is wrapping up two large clinical trials on a new antibiotic for uncomplicated urinary tract infections early, setting it up for an FDA filing next year. An independent review group determined that the non-inferiority trials testing GSK’s gepotidacin versus nitrofurantoin already met their primary endpoints in an interim analysis, a major win for the antibiotic space.
Senescent cells have been at the heart of Unity Biotechnology for years, and after previous stumbles, the anti-aging biotech says it likely found a light at the end of the tunnel: a pivotal study of its only clinical-stage drug is now on tap for September 2023 after a Phase II trial in diabetic macular edema proved a single injection to be durable out to 24 weeks.
Almost exactly three years after a data update at SITC underwhelmed, NextCure is calling it. The Yale spinout will stop working on its lead program, known as NC318, after observing no responses in a Phase II monotherapy portion. However, it will still support Yale’s investigator-initiated trial testing the drug in combo with Merck’s Keytruda in patients with lung cancer.
Radiotherapy maker Actinium made headway on a pivotal readout for CD45-targeting lead candidate Iomab-B, which beat placebo when given to patients with relapsed or refractory acute myeloid leukemia before they undergo bone marrow transplants. The endpoint of the study was durable, complete remission of at least six months after initial remission.
Abeona Therapeutics is trumpeting a data drop for the rare “butterfly skin” disease, with Phase III results showing its EB-101 healed wounds by at least 50% at a significantly greater margin than a placebo over six months, and significantly reduced patients’ pain associated with the wounds. Simultaneously, Abeona announced it had secured a $35 million private placement.
Not even two weeks after watching its stock nosedive, Tricida is looking at all of its strategic alternatives and options to reduce its operating expenses — usually a precursor to layoffs, a sale, or both. Ever since the FDA rejected its kidney drug, the biotech has banked its hopes on a comeback trial. But the high-stakes wager proved disappointing.
Exscientia is looking at breaking out of small molecule R&D. The UK AI player announced it will make a foray into biologics, more specifically antibodies, looking to implement precision engineering and utilize in silico screening. To that end, it will be adding new building space to its headquarters to house the new laboratory.
#CPHI22
The time is upon us as the manufacturing world descends on Frankfurt for CPHI. This year’s conference plans to be filled to the brim with the top companies and experts from around the world of pharmaceutical manufacturing. Reporting live on the ground, my colleague Tyler Patchen sat down with a conference exec ahead of the meeting to find out what was in store.
Samsung Biologics, the CDMO arm of the South Korean conglomerate, is committed to boosting its presence both at home and abroad. Just as it kicks off operations at its latest manufacturing plant, the company spoke at CPHI about a new campus in the works, for which the company had already dropped $324 million to purchase the land.
What challenges do companies that transport biopharma material encounter on the ground when they talk about supply chain issues? UPS Healthcare execs weighed in on how the pandemic woke the logistics world up to gaps in cold chain delivery, and what they are most concerned about as deliveries at ultra-low temperatures are becoming the norm.
CPHI’s annual report, which polled 400 industry execs from around the world, suggests that a majority sees a moderate outlook for manufacturers and biotechs. However, executives are extremely bullish on mRNA technologies, with it getting well north of 60% of the vote as a tech that will see the most preclinical investment next year. Another area that execs seem to be excited about is psychedelic-based therapeutics.
Tyler also previewed several top themes that were likely to shape conversations at the confab, from supply chain woes (exacerbated by rising energy costs and inflation) to growth and potential bottleneck of cell and gene therapies to the specific challenges faced by contract manufacturers. He also took a look back at the biggest manufacturing projects so far in 2022.
CELL/GENE TX
As million-plus-dollar gene therapies appear set to become the norm rather than the exception, the Boston-based drug pricing watchdog ICER is coming on board — in some cases. Its latest report finds that CSL Behring’s potential gene therapy etranacogene dezaparvovec for hemophilia B could be considered fairly priced at around $3 million.
As cell therapy player Allogene launched a study for its candidate targeting large B-cell lymphoma, its joint venture, Allogene Overland Biopharm put the finishing touches on a new CAR-T manufacturing facility in Shanghai just under a year after starting construction — a feat the company calls “truly remarkable” considering Shanghai was in lockdown for a big part of it.
Texas’ Instil Bio hit pause on enrollment in clinical trials because “a recent decrease in the rate of successful manufacturing” meant it was unable to dose some patients with its lead tumor infiltrating lymphocyte (TIL) therapy. The biotech is looking into the issues and reviewing its manufacturing processes before resuming the trials in melanoma and other solid tumors.
CHINA
For F-star, the once high-flying UK biotech helmed by Eliot Forster, the $161 million all-cash acquisition by China’s Sino Biopharm was a last resort as it grappled with a brutal public market and dwindling cash reserves. But even that deal is running into a snag. Nasdaq-listed F-star and its buyer are once again extending the deal to allow CFIUS more time to review.
Following an inspection of GSK’s contract manufacturer in Poland that identified questionable practices, China decided to ban the company from participating in its centralized drug procurement program through April 29, 2024. GSK noted that it only supplies one drug, the urinary retention drug Avodart (dutasteride), through this program.
In late July, Sangamo’s former R&D chief Adrian Woolfson launched his own gene therapy outfit. Replay’s primary platform was an HSV vector which, as a hub-and-spoke biotech, it said it was building four different companies around. Now, the first of those four companies is out of the bag. Named Eudora and led by a quartet of academics, it will go after three eye diseases.
A year and a half after diving headfirst into the psychedelic colloquially known as “toad venom,” RA Capital is back for more. The VC firm is leading a $60 million into Lusaris Therapeutics, whose main focus is looking at the psychedelic 5-MeO-DMT, a compound naturally derived from plants and the Colorado river toad, to go after treatment-resistant depression.
The latest startup to enter the microbiome world is keen to serve more than just drug R&D. Concerto Biosciences, which came out with a $23 million Series A, hopes that with the “rulebook of microbial ecology” it’s building with a screening platform, it can treat atopic dermatitis, protect crops and get rid of bad odor in those sweaty armpits.
A new biotech incubated at Samsara BioCapital just dropped with a basket of assets, including two clinical-stage ones, from Gilead, Shionogi, Phenex Pharmaceuticals, Astellas’ Mitobridge and Yale. With a Gilead vet at the helm, OrsoBio was originally branded The Liver Company and is looking to treat severe metabolic disorders, such as NASH and type 2 diabetes.
Remember that secretive ARCH biotech that licensed two antibodies from MorphoSys in June? HI-Bio, short for Human Immunology Biosciences, is now back with a $120 million financing round, as well as more details about itself. Capitalizing on new science, the biotech has a focus on autoimmune and immune-mediated diseases.
New Galapagos CEO Paul Stoffels — best known for his turn as the longtime CSO at J&J — has come up with a three-pronged strategy to dig a way out of the big hole the biotech finds itself in. And it calls for taking out the budget axe and chopping 200 jobs so they can put more money into deals. The biotech will also shutter work in fibrosis and kidney diseases.
California-based Avanir Pharmaceuticals filed a WARN notice in late October detailing 109 layoffs in California at its headquarters in Aliso Viejo, on the south side of Los Angeles. This comes after Otsuka’s American subsidiary announced that it would be integrating AvanirAvanir into its business, effective starting 2023. Otsuka acquired AvanirAvanir back in 2015 for $3.5 billion.
Surface Oncology is letting go of 20% of its workforce as the biotech prunes its pipeline and narrows the scope of its lead drug. The cancer drug developer is looking for external partners on its CD39 asset, dubbed SRF617, after a strategic review, which the company said would extend the runway into the second quarter of 2024.
In September, iBio paid a very affordable price to gain access to an AI-based platform and new therapeutic candidates from the startup RubrYc as the Texas CDMO looks to dive further into drug discoveries. In its latest move, iBio is now jumping headfirst into drug discovery and the AI approach — while getting rid of its CDMO arm and cutting 60% of its workforce.
As it turned out, the main reason Artiva chose to withdraw its IPO had to do with a different big plan it wanted to share — one that couldn’t be done under the constraints of having an S-1 on file with the SEC: a joint development, manufacturing and commercialization pact with Affimed to combine the biotechs’ therapies into a treatment for CD30-positive lymphomaCD30-positive lymphoma patients.
Bayer is punting an Ionis Pharmaceuticals-partnered drug back to the biotech, giving up on a program on which it had spent $240 million. It had grabbed Ionis-developed fesomersen, an antisense designed to prevent blood clots, as part of a 2015 licensing deal, but it decided to focus on an in-house blood thinner, asundexian, instead.
Now that its cabozantinib franchise has nestled its way into blockbuster status, Exelixis is replenishing the pipeline with some early-stage bets. The 28-year-old out biotech shelled a total of $100 million in cash to grab a preclinical antibody targeting SIRPα and a Phase I peptide-drug conjugate from Sairopa and Cybrexa, respectively.
Always on the lookout for the next thing to buy, Thermo Fisher Scientific settled on an oncology diagnostic player for its latest acquisition. The life sciences conglomerate is shelling out $2.6 billion to acquire The Binding Site Group from a group of private equity investors, integrating a suite of diagnostic assays and instruments for blood cancers and immune system disorders into its specialty diagnostics business.
FINANCING
Skipping an IPO, Emalex Biosciences instead secured $250 million in Series D funds to bankroll a Phase III clinical trial in children, adolescents and adults who have Tourette syndrome. The Chicago biotech plans to kick off the trial this quarter and will target 90 sites across the US, Canada and Europe with a 220-patient trial.
Artiva Biotherapeutics quietly withdrew its IPO a year and a half after first filing its paperwork, with a pitch centered on its NK cell therapies. Boasting a collaboration with Merck, Artiva got an IND clearance for its second oncology asset just recently. Two other companies have similarly backed out of plans to go public just this week.
In January, Alterome launched from stealth — but remained quite stealthy. Armed with an additional $35 million, it is now shedding just a little of that secrecy. The biotech said it raised the Series A extension to fund a third program arising from its strategy to go after “undruggable 3.0” with targeted therapies.
Seeking money to get its first microbiome-based candidate into a clinical trial in Australia, Intrinsic Medicine couldn’t succeed down the IPO route during a rough sweeping of the biotech market this year, so the Seattle upstart is instead going with a blank check. The Seattle company will combine with Phoenix Biotech Acquisition, a SPAC vehicle that looked at more than 90 companies before signing on the dotted line with Intrinsic.
Despite setbacks on its own candidate as well as cooling enthusiasm across the field, ALX Oncology is still keen to push forward its CD47 drug. And it’s getting a loan of up to $100 million from Oxford Finance and Silicon Valley Bank to fund multiple trials, from gastric and head and neck cancer to urothelial and breast cancers.
CORONAVIRUS
In the fight against Covid-19, AbbVie’s approved products and investigational assets have run into multiple hurdles. The latest is a Phase I axe, quietly revealed on the federal clinical trials database in recent days. The Chicago-area Big Pharma cited “re-evaluation of risk vs benefit (based on animal data)” in an update to the Phase I trial of an oral drug dubbed ABBV-990.
Penny stock biotech Rigel Pharmaceuticals reported a close miss for its sole approved drug in a Phase III Covid-19 trial. Its tyrosine kinase inhibitor, fostamatinib, failed to meaningfully reduce days on oxygen for patients without respiratory failure and who had certain “high-risk prognostic” factors. Nonetheless, the biotech said it’s encouraged by the results and plans to talk them through with its partner at the Department of Defense.
“Where’s Juan?” a soccer player asks while nervously eyeing the goal. “Covid, again,” a teammate responds while holding up a text message. The referee blows the whistle. That’s the introduction to one of HHS’ latest Covid-19 ads designed to convince those who are disproportionately affected by the pandemic to get the updated vaccine boosters.
LAW
AbbVie is putting up a fight against generic versions of its endometriosis drug Orilissa. It filed suit against more than a dozen competitors, including Sandoz and Teva, asserting patents — the latest of which expires in 2036 — and claiming that abbreviated NDAs filed by the rival drugmakers have caused or will lead to “foreseeable harm and injury” to AbbVie.
Teva execs confirmed that a settlement was reached in New York resolving claims about its role in the US opioid epidemic. By signing on to the national agreement, the state will get $523 million from the generics giant, ending years of litigation against opioid manufacturers and distributors. “And it’s done in the way that New York basically signs on to the national agreement, which has a certain value. And then they also get what you call a trial bump because they have a verdict against us,” CEO Kåre Schultz told investors and analysts.
Top pharmacy chains are looking to bundle the bulk of their opioid-related litigation into a major settlement. All told, CVS Health said it agreed to pay more than $5 billion to states, tribes and other groups over the next decade to resolve claims, Walgreens would pay $4.8 billion in remediation payments over 15 years and Walmart will likely pay another $3 billion.
Since at least mid-August, President Joe Biden’s rallying cry around beating Big Pharma on the issue of drug pricing has been the highlight of stump speeches, serving as the centerpiece of more than a dozen speeches, according to an Endpoints News tally of the transcripts. It is, however, unclear to what extent this accomplishment will translate into votes in the midterms.
Bristol Myers Squibb unveiled a surprise victory for its potential $4 billion-per-year drug Reblozyl. In a Phase III trial, the drug spurred a “statistically significant and clinically meaningful improvement” in red blood cell transfusion independence, or how long a patient with anemia goes without a transfusion.
With drug price negotiations in the books, Rep. Katie Porter (D-CA) is onto the next challenge. Her office released a new report on skyrocketing drug launch prices, particularly in oncology, which she calls for Congress to address with the momentum from the passage of the Inflation Reduction Act.
MARKETINGRX
Almost half of all adults with type 1 diabetes say they felt like quitting sports as a kid because of their health condition – and one in five ended up doing so. Now Dexcom wants to encourage the next generation to stick with sports, so it’s lined up a first-ever name, image, likeness (NIL) college athlete program to showcase 14 players living with type 1 diabetes. The athletes all use Dexcom’s continuous glucose monitoring devices. Through the campaign, Dexcom is also breaking ground in the pharma and medtech industries for national NIL sponsorship deals.
Novo Nordisk expects the supply of its obesity med Wegovy to finally stabilize by the end of this year — and with that, a “broad commercial re-launch is expected to commence next year.” The company had halted promotional efforts in the US after high demand, compounded by manufacturing issues, led to a shortage of the drug.
Despite years of public health messaging and awareness advertising, people over the age of 50 still aren’t getting the shingles shots — or even widely understanding the risks. So GSK is doubling down on advertising, this time with a direct message about the dormant virus that causes shingles, with two new TV ads, one in English and another in Spanish, warning this group to “wake up” to their shingles risk.
Astellas’ fezolinetant isn’t due for an FDA decision until February in vasomotor symptoms associated with menopause — but the pharma company isn’t waiting until then to educate women about what’s causing their hot flashes. Its latest TV ad spots seek to spread the word about VMS and offer more educational resources.
As Merck looks to expand its cash cow Keytruda into earlier lines of cancer, the company’s marketing team is encouraging patients to meet scientists halfway. Its campaign, “Focus on Your Lungs,” debuted to help spread the word about annual lung cancer screening. The new campaign focuses on who should be getting tested — and on addressing the stigma often associated with lung cancer.
East coast biotech and pharma companies once again took the top four spots on Science’s list of top 20 industry employers this year, headed by Insmed for the second year in a row. Alnylam, Regeneron and Incyte followed, with the Swiss Syngenta Group ranking fifth, according to Science. The companies were chosen by more than 6,200 survey respondents.
Merz Aesthetics is going even younger with its anti-wrinkle campaign. The Raleigh, NC-based company tapped 31-year-old singer-songwriter, actress and choreographer Teyana Taylor as the latest brand partner for its Botox rival Xeomin. The partnership is part of Merz’s broader “Beauty on Your Terms” campaign, which enlisted 33-year-old Joe Jonas back in August.
We got a relatively brief MarketingRx roundup this week, but it’s no less interesting: Moderna created a custom clue for “Jeopardy” last week in a new TV ad; Bristol Myers Squibb Foundation and Gilead Sciences posted its next class of awardees in a training program to increase diversity across clinical trials; AIDS advocacy group wrapped a campaign against Gilead with local Sunday print ad; and more.
FDA+
Amicus added to its series of painful pratfalls as the biotech repeatedly trips its way toward what executive chairman John Crowley still sees as a sure layup at the FDA. The agency yet again delayed an approval decision needed for AT-GAA — Amicus’ treatment for Pompe disease — after regulators were unable to complete a manufacturing inspection in China.
The number of pharma sponsors taking advantage of the FDA’s expedited programs has skyrocketed in recent years, including the use of multiple designations to hurry a new compound along. Those increases are only expected to continue, according to FDA-authored research published in JAMA that reviewed 581 novel drug indication approvals from 2008 to 2021.
Since the Covid pandemic began in 2020, the FDA has shuttered its White Oak, MD, doors to outside companies not only seeking input on their developing assets, but for adcomm meetings too. And the agency is still not ready to go back, although it did update how it defines in-person meetings to include Zoom meetings with cameras on.
Slowly but surely the FDA is cracking down on the vast, illegal online sale of drugs made possible by behemoths like Amazon. In a warning letter, the FDA told Amazon CEO Andy Jassy that the company sold and distributed drugs with undeclared active ingredients — including those of an NSAID pain drug — that were marketed as dietary supplements.
Apellis had secured a priority review for its geographic atrophy program, but it turns out the FDA wants to wait a little bit longer before making a decision. Regulators pushed back their decision date for pegcetacoplan from Nov. 26 to sometime next February, to consider newly submitted two-year efficacy data from a pair of Phase III studies — which qualify as a major amendment.
As the FDA pushes to more broadly standardize the way it collects safety data to better detect concerning signals with pharmaceuticals, biopharma companies are pushing back on why there isn’t a more international approach given the global nature of the industry. The comments follow a one-day public meeting in September focused on two new FDA documents.
After a rapid rise in the use of expanded access during the pandemic, the FDA released an updated Q&A on the program. This latest revision comes as the agency says it’s received fresh questions on how to implement some of the new regulatory requirements. In the document, the agency clarifies when sponsors’ expanded access policies need to be made public and when (on the rare occasion) it might reject an expanded access request.
MANUFACTURING
The FDA is ready for participants to apply to a pilot program that will kick off on April 1, 2023, around chemistry, manufacturing and controls (CMC) readiness. The pilot project will help expedite the CMC development of products that may have faster development timelines, through increased communication between the FDA and sponsors.
New warning letters from the FDA outline quality issues for Fort Worth, TX-based Sovereign Pharmaceuticals and India-based generics giant Lupin. For Sovereign, regulators said it “failed to validate the manufacturing process” for an undisclosed tablet, while Lupin’s problem was it had not established written procedures for cleaning equipment for manufacturing APIs.
This week in Manufacturing roundup: The CDMO arm of the Terumo Corporation is looking to branch out of Japan and offer its services in other nations; Sanofi’s API spinoff, EUROAPI, will augment a production site in Frankfurt, Germany, with an €18 million investment; The AAV manufacturer Avirmax CMC put the finishing touches on a new facility in the San Francisco Bay Area city of Hayward, CA; and more.
DON’T MISS
In a difficult year for biotechs, a startup out of Jeff Aronin’s incubator Paragon Biosciences had a midnight burial, Endpoints News has learned. Skyline Biosciences was built around an attempt to treat oral pre-cancerous lesions, but it “did not meet the milestones necessary to warrant additional development resources,” according to Paragon, which characterized Skyline as a “pre-proof of concept stage company.”
AstraZeneca is still trying to figure out how some of its confidential patient data ended up on GitHub, a software developer’s platform, but the company said the data have now been taken down. The incident seemingly happened due to a user error. Some of the data were related to AstraZeneca’s prescription assistance program, which offers discounts to those in need.
With a record number of biotechs now trading below cash amid a brutal public market downturn, many have sought ways to extend the cash runway. In the case of Sensei Biotherapeutics, though, a billionaire investor believes the right thing to do would be to reduce the cash runway. Writing in an open letter, he called on the company to return money to shareholders.
Treatment-resistant depression may have finally met its match (for some) as new Phase II results published in the New England Journal of Medicine showed the feasibility of a single dose of psilocybin in helping patients with treatment-resistant major depression for up to 12 weeks. An accompanying editorial called the findings “both intriguing and sobering.”
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