Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA approves first-in-class drug to treat narcolepsy
Takeda Pharmaceutical received FDA approval for its narcolepsy type 1 drug, Orzeyful, on Wednesday. The oral drug targets the root cause of narcolepsy — a lack of the neuropeptide orexin in the brain — by activating orexin receptor 2. The approval was based on two large Phase 3 trials showing that the drug led to statistically significant improvements in daytime sleepiness, cataplexy, and health-related quality of life. “The FDA approval of ORZEYFUL marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment,” said Julie Kim, President and CEO of Takeda in the press release. The approval follows years of developing narcolepsy drugs at Takeda, with the company stopping a clinical program back in 2021 due to liver toxicity issues. – Allison Whitten
AstraZeneca and Bristol Myers weigh $400 billion merger
Reported merger discussions between AstraZeneca and Bristol Myers Squibb have now ended, bringing a potential deal that could have created one of the world’s largest pharmaceutical companies to an early close. The reported talks attracted significant attention because of their unprecedented scale, although Reuters subsequently reported that a senior source denied the companies had been engaged in merger discussions. AstraZeneca’s shares fell more than eight percent following the initial reports, wiping roughly $22 billion from the company’s market value, as investors questioned the strategic rationale for combining two major pharmaceutical companies. While the talks appear to have gone no further, the episode highlights the growing pressure on large drugmakers to strengthen their pipelines and diversify ahead of patent expiries, while also raising questions about how much further consolidation the sector can absorb. – Bree Foster
LifeMine raises $263M for organ transplantation drug
On Thursday, LifeMine announced that they had raised $263 million to support the clinical development of LIFE-001, a novel calcineurin activation inhibitor to prevent organ transplant rejection. The drug is a long-acting injectable with the aim of avoiding the immunophilin-dependent organ damage caused by all previous calcineuron inhibitors. So far, the ongoing Phase 1 trial in over 120 adults has shown an improved profile compared to prior versions. “More than 1.6 million people worldwide are living with a transplanted organ, yet physicians continue to rely on calcineurin inhibitors that have remained largely unchanged for more than three decades despite their well-recognized toxicities,” said Gregory Verdine, President and CEO of LifeMine in the press release. – Allison Whitten
Aurora Therapeutics drops lead CRISPR program
Aurora Therapeutics has abandoned its lead CRISPR program in phenylketonuria (PKU) and reduced its workforce just seven months after emerging from stealth with $16 million in seed financing, according to reporting by STAT News. Just a month after Aurora launched, Beam Therapeutics announced its own base-editing program for PKU, and in June the FDA accepted its investigational new drug application. Beam has considerably greater resources than Aurora, including an established base-editing platform and existing FDA relationships. Aurora's rapid retreat suggests that targeting a rare disease alone is no longer sufficient to differentiate new entrants, with startups increasingly needing proprietary delivery technologies, novel editing chemistries, or unique biological insights to compete against better-resourced incumbents. Additionally, while regulators have signalled greater flexibility for individualized genetic medicines, there are high barriers for early-stage gene therapy companies as they still need to meet the costly requirements of clinical-grade manufacturing, safety testing, and regulatory documentation. – Bree Foster
Pfizer targets another $2.5 billion in cost savings
Pfizer has expanded its multibillion-dollar cost-cutting program, aiming to deliver an additional $2.5 billion in savings by 2029 as it continues to offset declining COVID-19 product sales and pressure across parts of its broader portfolio. The revised restructuring plan, first launched in 2023, is now expected to generate a total of $6.7 billion in savings, including $1.5 billion from manufacturing efficiencies alongside productivity improvements across commercial, research and development, and corporate functions. The announcement came as Pfizer reported second-quarter revenue of $15 billion, up one percent year-on-year, driven by five percent growth in its non-COVID medicines and stronger-than-expected sales of products including Eliquis, Ibrance, and Vyndamax. However, the company continues to face headwinds from falling sales across several oncology products, including Seagen-acquired therapies Adcetris and Tivdak, and recently recorded a $3.8 billion impairment charge following disappointing clinical data for an experimental Seagen cancer drug, alongside a $525 million write-down related to the withdrawal of the sickle cell therapy Oxbryta. – Bree Foster
Moderna’s mRNA flu shot gets nod from FDA
The first mRNA flu vaccine, mFlusiva, from Moderna was approved on Wednesday to prevent the flu in adults 50 years and older. The approval is based on Phase 3 trials showing that mFlusiva had a relative vaccine efficacy of 26.6 percent compared to standard flu vaccines in preventing the flu. However, the approval comes after a tumultuous recent history whereby the FDA under Marty Makary first refused to review the application before doing an about-face a week later and moving forward with the review. “Flu remains a significant public health challenge, and mFLUSIVA provides an important new option for America's seniors. This approval also reflects the ongoing potential of our mRNA platform to help address important public health challenges through continued scientific innovation,” said Stéphane Bancel, CEO of Moderna in the press release. – Allison Whitten
