Stoke Therapeutics Inc.’s stock tumbled 32% after the company reported phase I/IIa data of its antisense oligonucleotide, STK-001, in Dravet syndrome, showing it reduced convulsive seizures in children most consistently at the highest dose, with benefits increasing as time progressed.
While med-tech financings in the second quarter (Q2) rose by 24% over the first quarter (Q1) – an historically low Q1 – the overall funding landscape in 2023 has struggled to keep up with other years, with IPOs in particular hitting rock bottom. A total of $5.8 billion was raised in Q2 through 128 transactions. Combined with Q1, the amount for the first half of 2023 is $10.5 billion through 255 transactions. This is an overall drop in value during the first six months of 31% compared with the same timeframe in 2022. It also is down from 2017 – the next lowest year – by 22%.
Venture firm Flagship Pioneering Inc. and Pfizer Inc. are each investing $50 million in a pact that could result in the development of 10 programs, each worth a potential $700 million in milestones. Altogether, if all products are successfully commercialized, the deal could be worth $7 billion, with milestone money going to Flagship and some of its 45 bioplatform companies.
There are at least two ways to look at biopharma deals and M&As in 2023. Through one vantage point, the volume of both, and the value of M&As, are at their lowest levels in at least five years – and deal values have declined by 8% compared with 2022. From another perspective, however, M&As appear to be picking up, and those same declining deal values in the first half of 2023 represent the third highest amount since 2017.
Two biopharma companies entered the public markets on July 14, with Apogee Therapeutics Inc. pricing a $300 million IPO, the second largest U.S. debut this year, and Sagimet Biosciences Inc. raising $85 million. Apogee, of San Francisco, and Waltham, Mass., is advancing APG-777 and APG-808, which are in development for atopic dermatitis (AD) and chronic obstructive pulmonary disease, while San Mateo, Calif.-based Sagimet’s lead candidate is the FASN inhibitor denifanstat for nonalcoholic steatohepatitis.
Taking aim at the elusive place where stubborn cancer cells multiply, Crossbow Therapeutics Inc. has launched with $80 million in funds through a series A round. The Cambridge, Mass.-based company plans to advance its novel therapies, which mimic T-cell receptors and target peptide-loaded major histocompatibility complexes on cancer cells. If all goes well, the first product will be in the clinic in 2025.
Although biopharma investors pulled back in 2022 – largely a response to freely flowing funds the previous two years during COVID-19 surges – they are cautiously returning to the sector, as the industry has raised $32.8 billion in the first half of 2023, a 23.6% jump over the same period last year.
Becoming the first gene therapy approved for hemophilia A, Roctavian (valoctocogene roxaparvovec) finally received the U.S. FDA’s blessing on June 29, after developer Biomarin Pharmaceutical Inc. spent nearly three years working to address issues raised in a 2020 complete response letter. The approval came a day prior to the June 30 PDUFA date.
Although the highest dose of Bioxcel Therapeutics Inc.’s BXCL-501 hit the primary endpoint in the phase III Tranquility II trial for acute agitation in Alzheimer’s disease patients, shares fell dramatically on June 29 by 64% when investors learned the company received an FDA warning letter over infractions at a trial site.
Adults with generalized myasthenia gravis (gMG) have yet another therapeutic option, this time from UCB SA, with the U.S. FDA’s approval of orphan drug Rystiggo (rozanolixizumab-noli), a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor.
