Remynd NV has turned in positive phase IIa data for a new target in Alzheimer’s disease, showing that restoring calcium homeostasis in neurons reduces levels of pathological tau proteins, increases dopamine levels and has a positive effect on cognition in symptomatic patients. The company said the results of the placebo-controlled, doubled-blind trial testing REM-127, published Sept. 12, demonstrate that binding septin filaments in the cell cortex restores the normal functioning of calcium ion channels that have been disrupted by pathological tau. “It’s the first time that a septin modulator with this mechanism of action is tested in patients,” said Gerard Griffioen, chief scientific officer of Remynd.
Avidity’s offering follows reversal of Duchenne disease progression
As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures. Avidity priced its upsized underwritten public offering of 15 million shares of its common stock of $40 each on Sept. 11. Avidity stock (NASDAQ:RNA) was up 9% at midday on Sept. 12 at $44.74 per share.
US chaos positions China as more dependable leader
China was already making strides to lead the biotechnology industry in many key areas such as cell therapies and AI, but the chaotic nature of the Trump administration and the turmoil in the U.S. has catapulted China’s status as a more “dependable” partner, presenters said during the Bio Hong Kong conference, Sept. 10 to 13. “The rise of China as a biotechnology leader is reshaping the global innovation landscape,” said Paul Chan, financial secretary of Hong Kong. “The mainland is now the world’s second-largest pharma market and accounts for 30% of global innovative drug assets up from 10% in 2019.”
Fibrogen leads drug developer stock gains on China sale, FDA update
Drug developer stocks posted a mixed performance across July and August, with 23 companies on the BioWorld Drug Developers Index (BDDI) gaining ground and seven losing value. Fibrogen Inc. led the gainers with a 126% surge over the two months, boosted by the approval of the sale of its China subsidiary to Astrazeneca plc and, earlier in August, reaching agreement with the U.S. FDA on the design of the pivotal phase III trial for roxadustat.
Neuronal lipid metabolism plays role in ABCA7 Alzheimer’s risk
Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4. Previous work has shown that some of the effects of ABCA7 disruption are due to changes in microglial activation. Now, investigators at the Massachusetts Institute of Technology (MIT) have gained new insights into the mechanisms that link loss of function of ABCA7 to dysfunction in neurons as well.
Biopharma deals soar to $185B through August while M&A activity slows
Biopharma dealmaking remained robust through August 2025, with total disclosed deal value reaching $185.28 billion for the first eight months of the year, the highest January-to-August total in BioWorld’s records, and up nearly 36% over the same period in 2024. Meanwhile, biopharma M&A activity remains subdued in 2025, with $46.5 billion in M&A deal value announced through August, the lowest total for the period in seven years.
Health Canada vows to streamline operations for device and drug makers
Health Canada has posted a report on reduction of red tape for regulation of a wide swath of health care products, but the most important provision may be a proposed expansion of mutual recognition agreements. One example of this is a mutual recognition of other regulators’ inspections of device and drug manufacturing facilities, a seemingly marginal but nonetheless important win for companies in the life sciences.
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Bausch, Bioxodes, Cantargia, Capsida, Citius, Corstasis, Crown, Denovo, Design, Dewpoint, Durect, Ferring, Genvivo, Gri, Immunobrain, Inflammasome, J&J, Maze, Merck, Nacuity, Otsuka, Revolution, Sanofi, Silexion, Takeda, Vaxart, Yoltech