Ferring Pharmaceuticals A/S has notched another U.S. FDA approval, this time for a bladder cancer treatment, Adstiladrin (nadofaragene firadenovec). The non-replicating adenovirus vector-based gene therapy’s approval comes only weeks after the FDA’s Nov. 30 approval of the privately held company’s Rebyota (fecal microbiota, live), the first fecal microbiota treatment in the U.S. Adstiladrin is another landmark, as the first FDA-approved gene therapy to treat high-risk, non-muscle-invasive bladder cancer. Saint-Prex, Switzerland-based Ferring said it anticipates the product becoming commercially available in the U.S. in the second half of 2023.
Bryostatin-1 has failed another Alzheimer’s disease (AD) study. Synaptogenix Inc. released top-line data showing the protein kinase C activator missed the primary endpoint in the company’s phase II study in treating advanced AD.
When potentially dangerous high liver enzymes showed up in two asymptomatic participants during Third Harmonic Bio Inc.’s ongoing phase Ib study of the KIT inhibitor THB-001, the company decided to discontinue the clinical trial.
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
Wave Life Sciences Ltd. is partnering again. The company is getting $170 million up front – $120 million in cash and a $50 million equity investment – in a new partnership with GSK plc to advance oligonucleotides in unnamed targets. The agreement, which has a four-year research term, allows for GSK to advance up to eight programs using Wave Life’s platform and for Wave Life to advance three, or more if GSK approves, of its own collaboration programs.
The successful suitor for Horizon Therapeutics plc has been revealed as Amgen Inc., which will buy the Dublin-based company for $27.8 billion in cash in what is by far the year’s biggest acquisition. The deal brings successful biologics into Amgen’s portfolio that include Tepezza (teprotumumab), a monoclonal antibody targeting insulin-like growth factor 1 for treating thyroid eye disease, Krystexxa (pegloticase), a pegylated uricase enzyme for treating chronic refractory gout, and Uplizna (inebilizumab), a monoclonal antibody targeting insulin-like growth factor 1 for treating thyroid eye disease.
Erasca Inc. agreed to give Novartis AG $20 million cash up front and company shares (NASDAQ:ERAS) worth $80 million for an exclusive global license to naporafenib, a pan-RAF inhibitor for treating RAS/MAPK pathway-driven tumors, Erasca’s sweet spot. Erasca CEO Jonathan Lim told investors Dec. 9 that the therapy is complementary to the company’s portfolio, which includes 11 development programs targeting the pathway.
Top-line primary data from week 48 of Eiger Biopharmaceuticals Inc.'s phase III D-LIVR study of lonafarnib, a prenylation inhibitor for treating chronic hepatitis delta virus, also known as hepatitis D, hit statistical significance vs. a placebo in the composite primary endpoint.
Replimune Group Inc. has found consistent treatment efficacy in its pivotal phase II Ignyte melanoma study. The interim data strongly bolstered the stock (NASDAQ:REPL), which ended Dec. 7 with a 22% increase to $23.09. The new results for the combination treatment show what Replimune called clinically meaningful durable activity across the range of anti-PD1 failed cutaneous melanoma presentations, including in patients with moderate to high tumor burden, with 85% of responses ongoing.
In a massive deal that is one of the year’s biggest, Akeso Inc. signed a collaboration and license deal with Summit Therapeutics Inc. to out-license its bispecific antibody, ivonescimab (AK-112), for development and commercialization in the U.S., Canada, Europe and Japan.
