Multiple Alzheimer's Treatments Progress, Neumora Leadership Changes & AAV/CRISPR Tx Lead the Deals Space.

Unsurprisingly, as we wrap up AAIC, this week's headlines are dominated by exciting news in the Alzheimer's and broader neurodegeneration space. A deeper dive of the results announced in Amsterdam (alongside trends we're seeing and market analysis) will be published over the next few weeks. This Thursday however, we'll also recognise the fantastic news that has made headlines in broader CNS & Rare Disease.

Clinical Trials

• On Monday, 礼来 presented results from their donanemab TRAILBLAZER-ALZ 2 study at AAIC. Although ARIA is still a safety concern, results showed strong clinical improvement vs. placebo. This improvement was particularly marked in patients with low/medium tau, indicating the importance of early treatment. A decision by the FDA on approval is expected by end of 2023.
• Alnylam Pharmaceuticals announced on Monday that its Alzheimer's RNAi therapy, ALN-APP, has shown positive (interim) PhI results. A single dose of the amyloid-precursor targeting therapeutic reduces the protein by 65% at 6 months.

Leadership

• Neumora announces new President, CEO, and Board Member, Henry Gosebruch . Joining from AbbVie, Gosebruch reunites with ex-colleague & current Neumora CMO, Michael Gold . On the same day, the biotech also announced it would be initiating PhIII trials for navacaprant in major depressive disorder.

Deals

• argenx raises $1.1 billion through the sale of over 2 million shares in a global stock offering. The biotech recently announced positive PhII data for its chronic inflammatory dyemyelinating polyneuropathy (CIDP) asset, Vyvgart Hytrulo. The subcutaneous antibody fragment therapeutic has met its primary endpoint and shown to cut risk of relapse by 61% (vs placebo) alongside 67% of patients showing evidence of clinical improvement.
• Acumen Pharmaceuticals, Inc. announces upsized public offering, priced at $130 million. The AD biotech, led by a suite of Lilly veterans, also announced positive topline data from FIH INTERCEPT-AD PhI study on Sunday. The anti-amyloid antibody, ACU193, met both its primary and secondary objectives, demonstrating proof of mechanism.
• Neurogene Inc. and Neoleukin Therapeutics announce merger agreement. The resulting company, Neurogene, Inc., (NGNE on Nasdaq) will drive Neurogene's pipeline, focused on rare neurological diseases and genetic medicines. Neurogene has recently raised $95 million in private financing, resulting in combined company cash of around $200 million.
• Prevail Therapeutics and Sangamo Therapeutics, Inc. announce gene therapy partnership. Sangamo's AAV capsids (egineered via their SIFTER platform) have shown high efficacy in animal models for delivering gene therapy into CSF for CNS disorders. Prevail will evaluate Sangamo's technology with the option to license the capsids in the future for neurological targets.
• Sanofi and Scribe Therapeutics announce partnership to bolster the French pharma's non-viral gene therapy capabilities. With a $40 million upfront payment, Scribe's CRISPR technology will be licensed to Sanofi to develop gene therapies initially focused on sickle cell disease.

It seems that most partnerships are still focused on gene therapy technology as we try and find the best methods for delivery. Stay tuned next week for a deeper analysis into all seen at AAIC, including the wins for biopharma & patients alike, as well as the obstacles we are most likely to face.