NeuroPulse Wk 44: Acumen’s Alzheimer’s Antibody Cleared Plaque and Missed on Cognition in 542 Patients
Acumen’s sabirnetug missed its primary endpoint at both doses in 542 people with early Alzheimer’s disease despite significant plaque reduction, and Acumen has stopped developing it. BMS handed back Prothena’s PRX019 after Phase 1, China approved Bayer’s asundexian on a 26% cut in ischaemic stroke, and Canada’s drug agency drafted a yes on Spinraza for adults. Rapport raised about $380 million and Aspen priced a $140.6 million IPO.
on iADRS
on asundexian
after RAP-219
OrphanPulse
Acumen’s sabirnetug cleared plaque and missed on cognition in 542 patients, and BMS handed back an $80 million neurodegeneration antibody in the same week
Acumen Pharmaceuticals reported on 30 October that ALTITUDE-AD, a placebo-controlled Phase 2 of sabirnetug (ACU193) in 542 people with mild cognitive impairment or mild dementia due to Alzheimer’s disease, missed its primary endpoint at both doses. Neither 35 mg/kg nor 50 mg/kg, infused every four weeks, separated from placebo on iADRS at 18 months, and CDR-SB and the other key secondary endpoints were not significant. Amyloid PET fell significantly at both doses at weeks 36 and 76.
Acumen has stopped developing sabirnetug, including the open-label extension, and is reviewing strategic options; BioSpace reported the shares fell about 80%. A day earlier, Bristol Myers Squibb told BioSpace it would not advance PRX019 (BMS-986495), a neurodegeneration antibody it licensed from Prothena in May 2024 for $80 million upfront, after reviewing Phase 1 data in healthy adults. The licence ends on 28 January 2027, and Prothena forgoes up to $617.5 million in milestones and any royalties, PharmaShots reported.
The plaque signal did not carry over to cognition. Sabirnetug was designed to bind soluble amyloid-beta oligomers, yet the topline reports plaque reduction without clinical benefit. Acumen gave no effect sizes, confidence intervals or p-values for any endpoint, so the size of the miss is not public.
- ARIA-E still occurred. About 10% of treated participants had it, with no split by dose or against placebo.
- Fluid biomarkers come after the decision. Acumen expects them in the coming weeks, with the programme already stopped.
- BMS is keeping its tau bet with Prothena. Moponetug (BMS-986446), an anti-tau antibody with Fast Track designation, is in Phase 2 with a readout expected in the first half of 2027, BioSpace reported.
- Approved amyloid antibodies keep growing. Lilly reported Kisunla sales of $193 million in Q3, up 175%, on 29 October.
China approved Bayer’s asundexian, the first factor XIa inhibitor, on a 26% cut in ischaemic stroke, and FDA has had it under priority review since May
As our 27 October Brief reported, Bayer announced on 26 October that China’s NMPA had approved asundexian as Sunharbo, 50 mg once daily, to reduce the risk of stroke in adults after a non-cardioembolic ischaemic stroke or high-risk transient ischaemic attack. In OCEANIC-STROKE, 12,327 patients in 37 countries took asundexian or placebo on top of antiplatelet therapy. Ischaemic stroke occurred in 6.2% against 8.4%, a 26% relative reduction, and disabling or fatal stroke in 2.1% against 3.0%, with no increase in major bleeding.
Asundexian now sets the bar for add-on stroke prevention. It lost to apixaban in atrial fibrillation three years ago, so the class has narrowed to use on top of antiplatelets, where milvexian must beat a 26% reduction with no rise in major bleeding.
- FDA is next. The application has been under priority review since May 2026, and Japan’s review is ongoing.
- Milvexian reads out in 2026. LIBREXIA-STROKE and LIBREXIA-AF are expected this year, after the acute coronary syndrome trial was stopped in November 2025.
- Price is the launch test. Sunharbo adds a branded daily drug to generic antiplatelets, and China’s national reimbursement negotiation is the next step.
Canada’s drug agency drafted a yes on Spinraza for adults with SMA, four years after it told provinces not to pay
As our 27 October Brief reported, Biogen Canada announced on 26 October that Canada’s Drug Agency had issued a draft recommendation to reimburse Spinraza (nusinersen) with conditions, for adults with type 2 and 3 SMA and for patients with two, three or four SMN2 copies. In July 2022 the committee said not to reimburse it in adults, finding no randomised evidence in treatment-naive adults. The review began in March 2026 under the agency’s pilot of reimbursement reassessments, feedback closed on 29 October and the final recommendation is due on 1 December.
The draft changes no coverage yet. The conditions are not public, and any price condition will surface in the final recommendation and the pan-Canadian Pharmaceutical Alliance negotiation that follows. Provinces then decide whether to fund.
- The 2022 price was C$118,000 a vial, or C$354,000 a year in maintenance and about C$708,000 in the first year, by the Brief’s estimate.
- Four SMN2 copies are new. The 2022 draft did not include them.
- Quebec already pays. It has reimbursed Spinraza for all SMA types and ages since 2018.
Egetis launched Emcitate in the US at about $950,000 a year, the first treatment for MCT8 deficiency
Egetis Therapeutics has launched Emcitate (tiratricol) in the US for peripheral thyrotoxicosis in adults and children with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, pharmaphorum reported on 28 October. FDA approved it in September, and Egetis launched earlier than it had predicted. The US list price is about $950,000 a year; in Germany, where it launched after EU approval, the list price is about €10,000 a month before discounts.
The US list price is about $950,000 a year against about €120,000 at German list price. Analysts put the eligible population at 5,000 to 10,000 across the US and Europe combined, so payers on both sides will see the gap.
- A priority review voucher is for sale. FDA awarded one with the approval, and Egetis hopes to sell it before the end of 2026; recent comparable deals were valued at $150 million to $220 million.
- Analysts see $300 million to $500 million a year in potential sales for Egetis’s first product.
- Support runs through Egetis RareLink, its patient programme with PANTHERx Rare Pharmacy.
Grünenthal’s Qutenza beat a low-dose patch on post-surgical nerve pain over 12 weeks, but not at week 12 alone
Grünenthal presented the Phase III RISE trial as late-breaking posters at the IASP 2026 World Congress on Pain in Bangkok, announced on 28 October. RISE randomised 409 adults with moderate to severe neuropathic pain 6 to 60 months after surgery to a single 60-minute application of the Qutenza 179 mg patch (8% capsaicin) or a 0.04% capsaicin control patch. Qutenza cut average 24-hour pain significantly more than control averaged over weeks 2 to 12, one of two primary endpoints; at week 12 alone, the difference favoured Qutenza but was not significant.
The split primary leaves the size of the benefit open. Grünenthal reported no effect sizes or p-values for either primary endpoint, and the release does not mention a US filing for post-surgical pain.
- Retreatment held the effect. In the extension, with up to four retreatments, Qutenza beat control over weeks 2 to 42.
- No new safety signals, consistent with earlier Qutenza trials.
- The US label is narrower than Europe’s. Qutenza is approved in the US for postherpetic neuralgia and diabetic peripheral neuropathy of the feet, and in the EU for peripheral neuropathic pain in adults.
Idorsia will develop daridorexant in autism on an exploratory signal from a paediatric insomnia trial
Idorsia said on 26 October that it will start a development programme in autism spectrum disorder for daridorexant, the dual orexin receptor antagonist it markets as Quviviq for adult insomnia. The decision follows a Phase 2 in 165 children and adolescents with insomnia, more than half of whom had diagnosed or subthreshold autism or ADHD. After two weeks on 50 mg, participants with autism or subthreshold autism improved on the PROMIS Pediatric Sleep-Related Impairment questionnaire, with the largest effects on affect and behaviour, cognition and daily activities.
The autism finding is an exploratory subgroup. Idorsia gave no subgroup size or statistics, and the measure is a sleep-related impairment scale rather than a core autism endpoint. Programme details are due in the first half of 2027, after talks with regulators.
- The insomnia result is firmer. Total sleep time on day 1 rose with dose (p=0.0185), by 50 minutes on 50 mg against 20 on placebo.
- No drug is approved for the core features of autism, said Carmen Schröder of Idorsia’s autism advisory board.
- A paediatric insomnia Phase 3 starts in the second half of 2027, and Idorsia is developing a dedicated formulation for the autism programme.
Teva paid $57.5 million upfront in a bankruptcy sale for BioXcel’s at-home agitation film, which FDA is reviewing
Teva Pharmaceuticals said on 27 October that it had acquired worldwide rights to BXCL501, a dexmedetomidine sublingual film, by buying BioXcel Therapeutics’ neuroscience assets in a court-supervised Section 363 sale. Teva pays $57.5 million upfront and up to $67.5 million in contingent payments tied to approval timing and sales milestones. BXCL501 is under FDA review, as a supplemental application to Igalmi, for at-home treatment of acute agitation in adults with schizophrenia or bipolar I or II disorder; Igalmi itself is approved for use in hospital.
Teva bought a filed asset at a distressed price. The upfront is less than half of the potential $125 million, and part of the contingent money is time-based and tied to when approval comes.
- The setting is the prize. Acute agitation affects 25% to 40% of patients with these conditions, according to Teva’s market research.
- No PDUFA date was given for the at-home application.
- More detail may come on 11 November, when Teva holds its R&D Day in New York.
Rapport raised about $380 million at a 24% discount, eight days after its epilepsy drug narrowly beat placebo in bipolar mania
Rapport Therapeutics priced underwritten offerings on 28 October: about $175 million of common stock at $21 a share, $25 million of pre-funded warrants and $150 million of 2.25% convertible notes due 2032, convertible at about $26.78. A $30 million private placement at $21 brings the base raise to about $380 million, or up to $432.5 million with options. The price was about 24% below the prior close, and shares fell 22% before the open, TIKR reported.
The money covers both Phase 3 programmes. FOCUS 1 and FOCUS 2 in focal onset seizures are enrolling, and Rapport plans two Phase 3 trials in bipolar disorder from the second half of 2027. Rapport says the cash runs to the end of 2029.
- The bipolar signal was modest. RAP-219 beat placebo by 3.1 YMRS points, at the low end of the three to four points Stifel calls competitive.
- Conversion would add about 5.6 million shares, on top of roughly 11 million new shares, TIKR estimates.
- Cash at 30 June was $436.1 million, so the raise nearly doubles it before fees.
Aspen priced a $140.6 million IPO at the bottom of its range for a Parkinson’s cell therapy made from patients’ own skin
Aspen Neuroscience priced 9,375,000 shares at $15 on 29 October, the bottom of a $15 to $17 range, for gross proceeds of $140.6 million, and began trading on Nasdaq as ASPE on 30 October. Its lead asset, sasineprocel, turns a patient’s own skin cells into induced pluripotent stem cells and then into dopamine-producing neurons to replace those lost in Parkinson’s disease. Fifteen patients had been dosed in an open-label Phase 1/2a as of 18 August, and Aspen expects to dose patients in Phase 3 in the second half of 2027 after FDA feedback, Renaissance Capital reported.
The IPO bought time. Aspen’s registration filing said existing cash would not fund a year of operations and flagged substantial doubt about its ability to continue as a going concern, BioPharma Dive reported. It lost $62 million in 2025.
- No efficacy data are public from the Phase 1/2a; sasineprocel holds RMAT and Fast Track designations.
- About $347 million was raised privately before the IPO, since the company’s founding in 2018.
- The range implied about $494 million fully diluted at the midpoint, before Aspen priced at the bottom.
Korea will cover Xcopri for drug-resistant epilepsy and Spravato for treatment-resistant depression from November
Korea’s Ministry of Health and Welfare said on 30 October that its Health Insurance Policy Deliberation Committee had approved coverage for five drugs for rare and hard-to-treat diseases from November, Korea Biomedical Review reported. They include Xcopri (cenobamate) for drug-resistant epilepsy, a Korean-developed drug that launched overseas first, so Korean patients imported it for several years. Spravato nasal spray is covered for moderate or severe major depression that has not responded to at least two antidepressants.
A drug developed in Korea reaches Korean patients last. SK Biopharmaceuticals’ Xcopri launched abroad first, and the ministry gave no prices or patient numbers for either drug.
- The eligible pools are large. About 5% of Korean adults have major depression and up to 30% of them may be treatment-resistant, and about 30% of epilepsy patients struggle to control seizures, the article says.
- Rare disease patients pay 10% of covered drug costs and cancer patients 5%.
- The same package covers Oxlumo, adult Crysvita and Servier’s Tibsovo, covered in OrphanPulse this week.
BrainsWay says more than 118 million Americans now have coverage for Deep TMS in OCD, without naming the payers
BrainsWay said on 26 October that insurance policies covering Deep TMS for obsessive-compulsive disorder now reach more than 118 million covered lives in the US. The company describes itself as the first to receive FDA clearance for a non-invasive brain stimulation treatment for OCD. It did not name the insurers behind the latest additions or give the previous total.
Covered lives measure policy, not use. The figure counts members whose plans have a coverage policy, not patients treated or claims paid, and BrainsWay gave no utilisation data.
- Earlier OCD policies came from Cigna, Premera Blue Cross and Highmark, according to past BrainsWay announcements.
- Depression coverage is moving too. BrainsWay cited a recent increase in coverage for SWIFT, its accelerated protocol for major depressive disorder, without figures.
- Q3 results are due on 10 November, the first test of whether coverage shows up in revenue.
When a development gets big enough, it earns a full report.
Synopulse’s monthly intelligence lines: deep, single-subject reports on the moves that reshape a field.
Tracking this market more closely?
We prepare custom briefings on market access, pricing and deal activity, cut by therapy area, company or country.