Now in its 10th year, Clarivate’s newly released Drugs to Watch 2023 report primarily features treatments targeted to a particular biomarker, signaling the advance and increasing potential of personalized medicines. The 15 drugs on the list are anticipated to either register sales of more than $1 billion by 2027 or be clinical game changers. Nine are predicted to hit $1 billion annual sales each in China by 2030.
Once a rarity, billion-dollar deals are now coming fast and furious. Now three have appeared in only a few days. Gene therapy developer Voyager Therapeutics Inc. and Neurocrine Biosciences Inc. continued a longstanding partnership by agreeing to develop treatments for neurological diseases and for three new programs with rare CNS targets. Cytomx Therapeutics Inc. and Moderna Inc. entered a deal worth a potential $1.24 billion to Cytomx for generating and developing treatments in oncology and non-oncology. In the third deal, Royalty Pharma plc acquired an interest in Ionis Pharmaceuticals Inc.'s royalty in Biogen Inc.'s Spinraza (nusinersen) and Novartis AG's pelacarsen for up to $1.125 billion.
Fate Therapeutics Inc. is making big changes. The company told Janssen Biotech Inc. it was not interested in continuing the collaboration and option agreement cut in 2020 that could have brought Fate up to $3 billion. Fate also is halting four solid tumor natural killer (NK) cell programs to concentrate on two of its NK cell programs and two T-cell programs, all while reducing headcount to 220 employees in the first quarter of 2023.
The eyes certainly have it. There are plenty of ocular gene therapies being studied in a crowded field and now there is a new one as Perceive Biotherapeutics Inc. has closed on a $78 million series B financing.
Phathom Pharmaceuticals Inc.’s shares sank 31% on news that the U.S. FDA will not take action on the company’s NDA for oral small-molecule potassium-competitive acid blocker vonoprazan by the Jan. 11 PDUFA date.
Eli Lilly and Co., through its wholly owned subsidiary Prevail Therapeutics Inc., is delving more deeply into gene therapies. In a new collaboration, Prevail will use privately held Capsida Biotherapeutics Inc.’s adeno-associated virus engineering platform, coupling the tech with Prevail’s gene therapies to target causes of CNS diseases.
New research published in BMJ Open claims there are inconsistencies in Amgen Inc.’s phase III Fourier study for cholesterol lowering. Cardiac deaths were numerically higher in those taking the PCSK9 inhibitor Repatha (evolocumab) combined with a statin than those in the placebo group, the research noted.
After working through a complete response letter (CRL) brought about by COVID-19 from the U.S. FDA in June 2021, Mediwound Ltd. has received the agency’s approval for Nexobrid (anacaulase-bcdb) to treat severe burns in adults.
Little more than a month after the U.S. FDA approved the first gene therapy for adults with hemophilia B, Uniqure NV’s Hemgenix, strong phase III data have come from Pfizer Inc. The Pfizer results show fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX) gene for treating adult men with moderately severe to severe hemophilia B, hit the primary endpoint in the phase III Benegene-2 study. The one-time therapy is designed to allow those living with hemophilia B to be able to produce FIX instead of receiving regular, ongoing doses of exogenous FIX.
Two weeks ahead of its Jan. 6, 2023, PDUFA date, the U.S. FDA has approved the bispecific Lunsumio (mosunetuzumab) to treat adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. It’s the first bispecific antibody approved for treating any type of non-Hodgkin lymphoma (NHL). FL is the most common slow-growing form of NHL.
