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Home » Newsletters » BioWorld

BioWorld

Oct. 7, 2024

View Archived Issues
Child in wheelchair with parent, doctor

Scholar Rock’s phase III Sapphire gleams; bright new bid in SMA

Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, 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 (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded. Read More

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. It’s an impressive figure for such an early program, but the Cambridge, U.K.-based pharma hailed the small molecule’s potential against a range of indications, both alone and in combination regimens that could include PCSK9 inhibitor AZD-0780. Read More
Illustration of Victor Ambros and Gary Ruvkun

Two win Nobel for microRNA work

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 “for the discovery of microRNA and its role in post-transcriptional gene regulation.” Read More

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." Read More
siRNA

Judo throws effort into renal siRNA; $100M banked so far

Judo Bio Inc. emerged from stealth mode and rolled out data showing 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. Read More

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.” Read More

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. Read More

ICYMI: Week in review, Sept. 30-Oct. 4, 2024

A quick look back at top stories. Read More

Biggest gainers and losers for Sept. 30-Oct. 4, 2024

The top 10 biopharma stock gainers and losers for the week. Read More

Appointments and advancements for Oct. 7, 2024

New hires and promotions in the biopharma industry, including: Galapagos, Inozyme, Mosaic, Myricx, Oak Hill. Read More

Financings for Oct. 7, 2024

Biopharmas raising money in public or private financings, including: Editas, Humacyte, Onconetix, Sirnaomics, Sonnet Biotherapeutics, Vertex. Read More

In the clinic for Oct. 7, 2024

Clinical updates, including trial initiations, enrollment status and data readouts and publications: Biodexa, Relief, Ultragenyx. Read More

Other news to note for Oct. 7, 2024

Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Relay. Read More

Regulatory actions for Oct. 7, 2024

Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Bristol Myers Squibb, Telix. Read More

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