In one of the larger biopharma IPOs in 2023, Cargo Therapeutics Inc. pulled in $281.3 million on Nov. 10, selling 18.75 million shares at $15 each, the low end of its price range. The market debut comes just eight months after the San Mateo, Calif.-based company raised $200 million in an oversubscribed series A round.
A year-and-a-half after Eli Lilly and Co.’s Mounjaro (tirzepatide) gained U.S. FDA approval for adults with type 2 diabetes, the GLP-1 and GIP dual agonist was cleared for chronic weight management in adults who are obese or overweight and who also have one related condition.
Paxmedica Inc.’s shares (NASDAQ:PXMD) surged by 179% in early trading Nov. 7 after reporting the publication of phase II autism spectrum disorder results for PAX-101 (suramin) intravenous infusions. Suramin, an anti-trypanosomal and anti-purinergic agent introduced in 1923 for East African sleeping sickness, showed a statistically significant improvement at the 10-mg/kg dose in a secondary endpoint of Clinical Global Impressions – Improvement scale vs. placebo.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use, Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
The public markets have hammered biopharma companies conducting IPOs in the past four years, with the number of debuts dwindling and the amounts raised falling dramatically as well. But one of the more telling measures is price performance, which is significantly down for all IPOs completed since 2020. The stock prices of 13 firms that entered the U.S. markets in 2023 are currently at a decline of 26.8% on average, with only two companies trading above their offering price.
With one of the higher series A rounds for 2023, the newly launched Aiolos Bio Inc. pulled in $245 million to advance its lead product, AIO-001, an anti-thymic stromal lymphopoietin monoclonal antibody set to enter a phase II trial for moderate to severe asthma.
While biopharma dealmaking remains active, a strong third quarter (Q3) was not enough to bring it to the same level seen during each of the last three years, although values are coming close. At the same time, M&As appear to be rising above 2022, but even with the increase, they still lag behind other years. If the Pfizer Inc./Seagen Inc. merger, worth $43 billion, closes before the end of 2023, for example, M&As will still not come close to the overall value seen in 2019 and 2020.
Royalty monetization is a financing tactic that is becoming increasingly popular during challenging times, and PTC Therapeutics Inc. is the latest firm to leverage a marketed drug to pay off debt and fuel its development pipeline. The South Plainfield, N.J.-based company agreed to sell up to $1.5 billion of its Evrysdi (risdiplam) royalty stream to Royalty Pharma plc, of New York. Evrysdi is a survival motor neuron 2 RNA splicing modifier approved by the U.S. FDA in 2020 to treat spinal muscular atrophy.
While biopharma dealmaking remains active, a strong third quarter (Q3) was not enough to bring it to the same level seen during each of the last three years, although values are coming close. At the same time, M&As appear to be rising above 2022, but even with the increase, they still lag behind other years. If the Pfizer Inc./Seagen Inc. merger, worth $43 billion, closes before the end of 2023, for example, M&As will still not come close to the overall value seen in 2019 and 2020.
