Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $23.69, or 319%, to $31.11 after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy. Apitegromab met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded. Subjects were given apitegromab, a binder of the inactive form of myostatin or current standard of care (either Spinraza [nusinersen, Biogen Inc.] or Evrysdi [risdiplam, Roche AG]) and turned up the strong results at week 52. Scholar Rock plans to submit a BLA as well as an EU marketing authorization application in the first quarter of next year.
Astrazeneca picks up early stage Lp(a) disruptor in $2B CSPC deal
Astrazeneca plc is adding a preclinical-stage candidate to its cardiovascular pipeline via a potentially $2 billion licensing agreement with CSPC Pharmaceutical Group Ltd., which includes a $100 million up-front payment for rights to YS-2302018, an oral Lp(a) disruptor. Elevated levels of Lp(a), or lipoprotein (a), have been identified as a risk factor for cardiovascular diseases, and Astrazeneca said it intends to develop YS-2302018 against a range of indications, both alone and in combination regimens that could include its PCSK9 inhibitor, AZD-0780.
Nobel Prize in Physiology or Medicine for discovering microRNA
Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its discoverers – Victor Ambros and Gary Ruvkun – the 2024 Nobel Prize in Physiology or Medicine. “This year’s prize is definitely a physiology prize. It helps our basic understanding of … how cells differentiate and become specialized,” Gunilla Karlsson Hedestam commented. Karlsson Hedestam is a member of the Royal Swedish Academy of Sciences and a professor at the Karolinska Institutet, where she directs the group for the research of the genetic basis for B- and T-cell recognition and function.
PBM private labels: Good or bad for US biosimilar market?
A growing foray of pharmacy benefit managers’ (PBMs) private labels into the U.S. biosimilar space is intensifying concerns about the antitrust aspects of PBMs’ vertical integration that has them serving as price negotiator, formulary setter, payer, group purchasing organization, pharmacy, provider and now drug "manufacturer." When CVS Caremark President David Joyner testified at a House Oversight hearing a few months ago about how CVS’ private label adalimumab biosimilar had significantly impacted the Humira competition, none of the committee members raised antitrust issues. But last week, Senate Finance Committee Chair Ron Wyden (D-Ore.) urged the FTC to expand its ongoing investigation into PBM practices, including the emergence of private label biosimilar agreements, saying, “It is important we keep a watchful eye on the evolving tactics of PBM megafirms as they proceed with owning more and more of the prescription drug supply chain.”
Judo throws effort into renal siRNA; $100M banked so far
Judo Bio Inc. made its debut by rolling out findings during the Oligonucleotide Therapeutics Society (OTS) annual meeting in Montreal that showed the value of using megalin receptors for intracellular delivery of ligand-small interfering RNA (siRNA) therapeutics to the kidney as a way of reducing expression of the targeted genes. OTS attendees learned of Judo’s STRIKE (Selectively Targeting RNA Into KidnEy) platform, designed to develop and discover ligand-siRNA conjugates that harness the natural endogenous process of receptor-mediated endocytosis for uptake of oligonucleotide therapeutics to specific kidney cell populations. The company raised $100 million in series A plus seed money in November 2021.
BioWorld Insider podcast: Gene and cell therapies will propel innovation
Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in the newest episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.” Zieler also shared his thoughts on creating sustainable health care in aging societies, the innovation cycle gap between the U.S. and Europe, and how collaboration between business and government is critical in forging ahead in this wide-ranging discussion.
Novo Nordisk to launch Wegovy in South Korea mid-October
Danish pharma giant Novo Nordisk A/S is set to launch its blockbuster glucagon-like peptide-1 therapy, Wegovy (semaglutide), in South Korea’s growing obesity therapeutics market next week, a company official confirmed to BioWorld. While stating that Wegovy “will be made available in Korea mid-October 2024,” the Bagsværd, Denmark-headquartered company did not unveil any pricing or reimbursement plans, emphasizing only that “the price of Wegovy reflects its clinically proven benefits for weight loss, as well as the innovation it represents for the treatment of obesity – besides the differences in convenience, efficacy and additional cardiometabolic outcomes.” Wegovy’s launch comes more than a year after Korea’s Ministry of Food and Drug Safety approved the semaglutide prefilled pen formulation.