Shares of Fennec Pharmaceuticals stock were cut almost in half early Monday as the company said manufacturing issues caused another FDA rejection of its reformulated version of sodium thiosulfate, which is intended to help kids who lose hearing due to chemo treatment.
The biotech had resubmitted an NDA for the drug to treat platinum-based, chemo-related ototoxicity in young children earlier this year. The first NDA was denied by the FDA last year, with the agency citing manufacturing issues with the biotech’s supplier.
Again this morning, according to Fennec, the FDA identified unspecified deficiencies following a pre-approval inspection of the manufacturing facility that Fennec planned to use for the drug, to be known commercially as Pedmark. Once the official CRL is received, Fennec says it plans on requesting a follow-up meeting with the FDA to discuss next steps on a second resubmission.
At the time of the company’s first CRL on Pedmark, CEO Rosty Raykov told investors there were no clinical safety or efficacy problems and an additional trial was not required, but when pushed on the Form 483 the biotech received and what issues had to be resolved, Raykov demurred from the issue. That time, Fennec’s stock price fell more than 35%.
Pedmark received fast track designation from the FDA back in 2018 after two Phase III studies looked at the survival and reduction of ototoxicity in five pediatric cancers that typically require intensive treatment. Currently, there are currently no approved drugs that prevent ototoxicity, or hearing loss, in platinum-based chemotherapies. And patients who end up losing their hearing must typically opt for cochlear implants.
While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.
Tillman Gerngross (Adagio)
Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.
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Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.
In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.
As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.
Róbert Wessman, Alvogen CEO
Alvogen is handing off two of its subsidiaries to CEO Róbert Wessman and his healthcare investment firm Aztiq — who has now tied the two together in a massive biobucks deal.
In an alliance with Thailand’s PTT Group subsidiary Innobic, the two companies signed an agreement last week to buy a 100% stake in Alvogen Emerging Market Holdings Limited (AEMH) for $475 million from Alvogen, buying out shareholders such as CVC Capital Partners and Singapore’s Temasek. And now, the group is the majority shareholder of Alvogen’s former Asian subsidiary Lotus Pharmaceuticals and the only shareholder of Alvogen Malta, the owner of B2B pharma Adalvo.
Dutch VC Forbion is hopping on the ever-lengthening SPAC train.
To be led by Jasper Bos, who joined Forbion Growth as a general partner back in May just after the fund closed at $428 million, Forbion European Acquisition will target late-stage opportunities in the life sciences industry in Europe to merge with and bring onto Nasdaq.
Cyril Lesser, senior controller at Forbion, will be the CFO while Bos serves as CEO.
Jeff Albers, Blueprint Medicines CEO
J&J’s Rybrevant scored the first approval back in May for a small group of lung cancer patients with a rare EGFR mutation. Despite a swarm of other biopharma companies angling for a piece of that market, Blueprint Medicines is betting nearly $500 million on a candidate it thinks will stand out.
Blueprint is putting down $250 million in cash and another $215 million in biobucks for Lengo Therapeutics and its preclinical non-small cell lung cancer program LNG-451. Though it hasn’t been tested in humans, Blueprint says the candidate was ‘highly brain-penetrant’ in preclinical trials, and has the potential to inhibit all common EGFR exon 20 insertion variants — which are found in just 2% to 3% of NSCLC patients.
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Amid burgeoning efforts to create a new type of cell therapy out of regulatory T cells — whether by channeling or blocking their immunosuppressive power — Quell Therapeutics wants to shoot for a first.
If everything goes well, the Syncona-backed biotech will be in the clinic early next year, marking what it calls the historic feat of dosing a patient with a CAR-Treg with multiple edited genes.
Rogerio Vivaldi, Sigilon CEO (Sigilon via website)
Back in July, the FDA placed a clinical hold on the Bob Langer and Flagship-backed biotech Sigilon Therapeutics for its lead program to treat hemophilia A. On Monday, Sigilon reported what caused the pause.
After a patient in the three-person Phase I/II study reported a serious adverse event, Sigilon discovered the spheres used to deliver the cell therapy had fibrosed, the biotech announced Monday. As a result, the treatment contained within the spheres was no longer viable after the patient developed inhibitors to Factor VIII.
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