Neuroscience & Neurodegenerative

Shionogi & Co., Ltd. announced that its Board of Directors approved an agreement to acquire IntraBio Inc., a biopharmaceutical company developing and commercializing therapies for neurodegenerative diseases, for an upfront consideration of USD 2.0 billion payable to IntraBio shareholders. Under the agreement signed on October 5, 2026, IntraBio would become a wholly owned subsidiary of New Jersey-based Shionogi Inc., with the transaction scheduled to close between November 2026 and December 2026, subject to competition-law waiting periods and other customary conditions. The deal would add AQNEURSA (levacetylleucine) to Shionogi's rare disease portfolio; the drug was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease type C and by the European Medicines Agency in January 2026, and on September 18, 2026 it became the first and only FDA-approved treatment for Ataxia in patients with Ataxia-Telangiectasia, for which it is also under EMA review. Shionogi said the acquisition builds on the rare disease foundation it established through its April 2026 acquisition of global rights to edaravone, known as RADICAVA in the U.S. and RADICUT in Japan, and would strengthen its pipeline across Pompe disease, Fragile X syndrome, Jordan's syndrome and early-stage rare neurodegenerative programs. The impact on Shionogi's consolidated financial results for the fiscal year ending March 2027 is currently under review.

AnnJi Pharmaceutical announced it is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial of AJ201, also known as rosolutamide, in patients with spinal and bulbar muscular atrophy, or SBMA, also called Kennedy's disease. The company said it submitted the Phase 3 protocol to the U.S. FDA under its active Investigational New Drug application and will now activate U.S. sites for the global trial. The ROMA-KD study is a global, multicenter, randomized, double-blind, placebo-controlled trial expected to enroll approximately 200 ambulatory patients with symptomatic SBMA worldwide, with the United States as a key region, and is intended to support potential global regulatory submissions. AJ201, an investigational oral small molecule and a potential first-in-class treatment for SBMA, has received Fast Track Designation from the U.S. FDA and Orphan Drug Designation in both the United States and the European Union. AnnJi said the Phase 3 program builds on encouraging results from its completed Phase 2 study announced in May 2025, and the company also noted its SBMA Patient and Care Partner Advisory Council, first announced in collaboration with the Kennedy's Disease Association at the 2026 KDA International Patient and Scientific Conference.

Wells Fargo initiated coverage of Design Therapeutics with an overweight rating, citing the company's Friedreich ataxia candidate DT-216P2 as having potentially best-in-disease functional improvement based on results from the RESTORE-FA study released in May. The bank set a $26 price target, implying roughly 112% upside based on the October 1 close. Analyst TianQi Hang wrote that the May update showed pharmacokinetics look good, and that blood-FXN protein, muscle-mRNA data, plus an early mFARS signal further de-risk the platform. Hang estimates that the blood FXN protein increase seen after 6 weeks can translate to at least a 2-point mFARS change, and said that if the drug kinetics sustain for 12 weeks, which he believes they will, DT-216P2 could deliver best-in-disease functional benefits. He assigns DT-216P2 a 60% probability of success, with peak sales of approximately $600M in the US and approximately $900M outside it. If approved, DT-216P2 would compete against Biogen's Skyclarys, also known as omaveloxolone, and Hang sees it gaining a peak share of the FA treatment market of 30% in the US and 20% ex-US.

AbbVie has secured FDA approval for Juvmo, a once-daily oral treatment for Parkinson's disease, with a commercial launch targeted for October 2026. Juvmo is the first selective D1/D5 dopamine receptor agonist that can be used both as a standalone treatment and in combination with levodopa, the current standard of care for Parkinson's symptoms. The approval expands AbbVie's Parkinson's portfolio, which already includes Vyalev and Duopa, and management expects the three therapies to collectively represent a peak-sales opportunity of more than $5 billion. Neuroscience now accounts for nearly a fifth of AbbVie's overall topline and generated $6.1 billion in revenues in the first half of 2026, up 22% year over year, with the company expecting approximately $12.7 billion in neuroscience revenues for the full year. The approval could also deliver a commercial payoff from AbbVie's approximately $8.7 billion acquisition of Cerevel Therapeutics in 2024, a deal that came under pressure after emraclidine failed in two registration-enabling phase II studies in schizophrenia and prompted a $3.5 billion impairment charge. AbbVie competes in neuroscience with Biogen, which markets Leqembi with Eisai and Zurzuvae, and Johnson & Johnson, whose portfolio is anchored by Spravato and Invega Sustenna and was strengthened by last year's acquisition of Intra-Cellular Therapies, adding Caplyta.

Roche Holding received FDA acceptance of its New Drug Application for fenebrutinib in multiple sclerosis under priority review. The filing covers fenebrutinib as an oral BTK inhibitor for both relapsing and primary progressive forms of multiple sclerosis. If approved, fenebrutinib could become the first high efficacy oral BTK inhibitor targeting both relapse control and disability progression in MS, adding an oral, CNS penetrant option alongside Ocrevus in Roche's neurology offering. Roche Holding is a CHF279.4 billion pharmaceuticals and diagnostics group, and the milestone reinforces the view that late stage assets like fenebrutinib can help offset ongoing loss of exclusivity and pricing pressure. The key signpost now is the FDA's priority review timeline for fenebrutinib, including the final decision date and any label details regulators set, with three Phase III trials supporting the filing.

AbbVie secured FDA approval for Juvmo, a once-daily Parkinson's treatment, though its shares slipped nearly 0.8% to $264.29. Juvmo works through D1/D5 dopamine receptors, a different target from older dopamine agonists. In the TEMPO-3 trial, patients taking it alongside levodopa gained 1.7 hours a day with symptoms controlled and no troublesome involuntary movement, compared with 0.6 hours for those given a placebo. AbbVie expects U.S. availability in October but has not disclosed Juvmo's price or a sales forecast, and nausea, dizziness and hallucinations are among the treatment concerns physicians must weigh. At $264.29, the stock sits 19.43% above its $221.28 GF Value estimate.

Eisai's full-year results for the fiscal year ending March 2026 showed revenue of 825.3 billion yen, up 4.6 percent year on year and a record high, while operating profit fell 18.8 percent to 44.1 billion yen and profit attributable to owners of the parent declined 17.0 percent to 38.5 billion yen. According to the company, the profit decline stemmed from the reversal of one-off gains booked in the prior year, including a lump-sum payment related to the transfer of rights to certain products and the termination of a strategic alliance, as well as higher selling, general and administrative expenses from aggressive investment in the Alzheimer's treatment Leqembi and structural reforms in Europe. Meanwhile, core operating profit, which the company positions as a measure of underlying profitability, rose sharply by 110.7 percent year on year to 50.1 billion yen. In terms of sales, Leqembi, the anticancer drug Lenvima and the insomnia treatment Dayvigo continued to grow. For the first quarter of the fiscal year ending March 2027, revenue was 234.3 billion yen, operating profit 24.7 billion yen and net profit 18.2 billion yen, putting progress toward the full-year operating profit forecast of 70 billion yen at 35.3 percent, ahead of the 25 percent pace considered a benchmark for the first quarter. The company expects consolidated revenue of 883.5 billion yen and operating profit of 70 billion yen for the full fiscal year ending March 2027, with Lenvima planned at 345 billion yen, the largest by amount, Leqembi at 143.5 billion yen and Dayvigo at 73.5 billion yen.

Johnson & Johnson said Caplyta met the main goal of a late-stage study in bipolar mania, a potential new use for the drug acquired through its $14.6 billion purchase of Intra-Cellular Therapies. In the Phase 3 trial, a once-daily 42-milligram dose of Caplyta significantly reduced symptoms of bipolar mania compared with placebo in adults with manic episodes associated with bipolar I disorder, with roughly 46% of treated patients achieving a clinical response versus about 21% on placebo. Patients began showing improvement as early as three days after starting treatment, and the benefits were maintained through the study's three-week duration; the trial also met a key secondary goal measuring overall severity of illness. Caplyta is currently approved in the United States for depressive episodes associated with bipolar I and bipolar II disorder, as well as schizophrenia, and J&J noted that bipolar disorder affects an estimated 37 million people worldwide. The use remains experimental for bipolar mania, and investors will be watching regulatory timelines, potential sales contributions and the size of the addressable market to gauge how much value the indication could add to J&J's neuroscience business.

Biogen's growth portfolio generated $1.06 billion in second-quarter 2026 revenues, up 24% year over year and 25% sequentially, surpassing the company's legacy MS portfolio, which brought in $767 million. Even excluding newly acquired Syfovre and Empaveli, growth-product revenues reached $933 million, up 9% year over year and 10% quarter over quarter, still above the legacy MS portfolio. Within the growth portfolio, Skyclarys revenues rose 29% to $168 million, Zurzuvae increased 53% to $71 million, Spinraza grew 2% to $402 million, and Vumerity fell 7.4% to $196.5 million, while Syfovre and Empaveli contributed $97.4 million and $30.4 million respectively. Alzheimer's collaboration revenues from Eisai for Leqembi rose 16% to $63.7 million, and the companies expect blood-based diagnostics and the launch of Leqembi Iqlik to drive further growth from 2027 onward. Biogen expects the growth portfolio to maintain its lead in the second half of 2026, with Syfovre and Empaveli contributing more than in the second quarter since revenue recognition began only in mid-May.

Oryzon Genomics, S.A. announced that the European Medicines Agency has authorized its Clinical Trial Application to initiate a Phase II study of vafidemstat for the treatment of Phelan-McDermid Syndrome. The study, named HOPE-2, is a single-center, single-arm, open-label Phase IIa trial that will enroll 12 adult patients with PMS, a severely disabling genetic disorder related to autism with a U.S. prevalence estimated at approximately 1 in 7,300 people. The primary objective is to evaluate the safety and tolerability of vafidemstat, with secondary objectives assessing its effect on anger and aggression and overall disease efficacy; vafidemstat will be administered for 12 weeks, after which the investigator will assess whether participants may continue treatment through week 24 based on clinical benefit. The study will be conducted in Spain as part of Oryzon's VANDAM project, which is part of the Med4Cure Important Project of Common European Interest on Health and has received funding from the Spanish Ministry of Science, Innovation and Universities and the Centre for the Development of Industrial Technology and Innovation under the Recovery, Transformation and Resilience Plan funded by the European Union – NextGenerationEU. Oryzon will collaborate with the Spanish Phelan-McDermid Syndrome Association to support identification of potential participants.

B. Riley analyst Mayank Mamtani has identified six top biotech picks heading into third-quarter 2026 earnings season, following the Federal Reserve's 25 basis point rate hike to 4.00%, its first increase since July 2023. The unanimous 12-0 vote left room for one to two additional hikes before year-end, and with the 10-year Treasury at its highest level since 2007, small and mid-cap biotech stocks face a macro-driven drawdown that historically has presented buying opportunities. The XBI index is up 27% year-to-date but down 2% quarter-to-date, compared to the S&P 500's 13% and 4% gains respectively, while loss of exclusivity-driven pharma mergers and acquisitions track toward their strongest year since 2019, permanent FDA leadership is installed, and drug-pricing risk is largely resolved through most favored nation agreements covering 26 companies. Late third quarter and fourth quarter carry over 40 company-specific catalysts across the coverage list. The picks are Axsome Therapeutics, rated Buy with a $300 price target and up 6% year-to-date, where the analyst models AUVELITY revenue at approximately $205 million to $210 million against consensus of $215.8 million and total revenue of approximately $259 million; Cytokinetics, rated Buy with a $146 price target and up 4% year-to-date, with MYQORZO wholesale tracking to approximately $18 million for the full third quarter versus $8.9 million for all of second quarter; Eton Pharmaceuticals, rated Buy with a $70 price target and up 229% year-to-date, with consensus third-quarter 2026 and fiscal year 2026 revenue of $42 million and $149.7 million versus management's guidance of over $145 million for fiscal year 2026; Harrow, rated Buy with a $60 price target and down 31% year-to-date, with fiscal year 2026 guidance of $350 million to $365 million; TG Therapeutics, rated Buy with an $86 price target and up 95% year-to-date, positioned to compound off an approximately $1 billion U.S. Briumvi exit run-rate at year-end 2026; and Zymeworks, rated Buy with a $44 price target and up 4% year-to-date, where partner Jazz Pharmaceuticals' third-quarter 2026 report in early November provides the first commercial read on the first-line HER2-positive gastroesophageal adenocarcinoma launch following the August 25 approval and $250 million milestone.

Otsuka Pharmaceutical and U.S.-based Ionis Pharmaceuticals announced on the 22nd that their jointly developed treatment for hereditary amyotrophic lateral sclerosis, ulefnersen, met its primary goal in a late-stage clinical trial. In patients with FUS mutation ALS, a rare inherited form of ALS that damages the nerve cells controlling movement, ulefnersen improved function and extended survival compared with the placebo group. The drug reduced markers of nerve cell damage and slowed disease progression, and most side effects were mild or moderate, indicating a favorable safety profile. No approved treatment currently targets the genetic cause of FUS-ALS, and the two companies plan to discuss the results with the U.S. Food and Drug Administration and global health authorities as they explore a path toward accelerated approval. Separately, Otsuka Pharmaceutical has launched a global early access program for FUS-ALS patients unable to participate in the trial, allowing physicians to request access to ulefnersen before approval.

Ionis Pharmaceuticals announced positive late-stage results from two partnered phase III programs. The FUSION study of ulefnersen in amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene met its primary endpoint assessing functional impairment and survival at 72 weeks, with Ionis reporting the results as statistically significant without disclosing supporting numbers; Otsuka Pharmaceutical, which holds worldwide commercialization rights licensed in 2024, plans to discuss the data with the FDA and other global health authorities regarding potential expedited regulatory submission pathways. Separately, the IMAgINATION study of sefaxersen in adults with primary immunoglobulin A nephropathy, conducted by Ionis' partner Roche, met its primary endpoint in a prespecified interim analysis, showing statistically significant and clinically meaningful reductions in proteinuria after 37 weeks, and will continue in a blinded manner to evaluate kidney function over two years with estimated glomerular filtration rate at week 105 as the longer-term measure. Roche licensed sefaxersen from Ionis in 2022 and is responsible for the phase III study and future global development, regulatory and commercialization activities, while Ionis is eligible for milestone payments and tiered royalties on net sales of both drugs. The two wins follow back-to-back cardiovascular setbacks for Ionis, including the phase III CARDIO-TTRansform failure of Wainua with AstraZeneca in July and the phase III Lp(a)HORIZON miss for pelacarsen with Novartis, and come after the FDA approval of Zanvastro for Alexander disease earlier this month. Year to date, Ionis shares have lost 42% compared with the industry's 2% decline.

Alkermes plc reported positive topline results from a phase 1b proof-of-concept study of ALKS 7290, an investigational orexin 2 receptor agonist, which showed clinically meaningful, dose-dependent improvements in adult ADHD symptoms over 14 days with a generally well-tolerated safety profile. These data represent the first clinical evidence of an orexin receptor agonist's effects in ADHD, potentially widening Alkermes' orexin franchise beyond sleep disorders and other neurologic indications already under exploration. The ALKS 7290 news follows the launch of the Brilliance phase 3 alixorexton studies in narcolepsy earlier in 2026. Alkermes' narrative projects $2.3 billion revenue and $374.7 million earnings by 2029, requiring 10.6% yearly revenue growth and about a $308.6 million earnings increase from $66.1 million today, while the most cautious analysts had penciled in only about 7.5% annual revenue growth and US$333.7 million of 2029 earnings.

Johnson & Johnson announced positive top-line data from a pivotal phase III study evaluating Caplyta (lumateperone) for treating manic episodes, with or without mixed features, in adults with bipolar I disorder. In Study 451, the first of two pivotal phase III studies of Caplyta in bipolar I mania, patients treated with once-daily Caplyta (42 mg) achieved a 4.8-point greater reduction in Young Mania Rating Scale total score versus placebo at week three, with significant improvement seen as early as day three, the study's primary endpoint. The proportion of patients achieving at least a 50% reduction in YMRS total score was more than twice as high with Caplyta as with placebo, at 45.8% versus 20.9%, respectively, and the safety and tolerability profile was similar to Caplyta's established profile. Management noted that the second pivotal phase III study, Study 452, has been completed, with data analysis underway. Caplyta, an atypical antipsychotic added to J&J's neuroscience portfolio through the 2025 acquisition of Intra-Cellular Therapies, generated sales of $631 million in the first half of 2026 and is not presently approved for the treatment of manic episodes associated with bipolar I disorder; J&J believes it has the potential to deliver peak sales of $5 billion.

AbbVie reported second-quarter revenues of $16.99 billion, up 10.2% year on year and 1.2% above analysts' consensus expectations, though the quarter was mixed with only a narrow beat of EPS estimates and a slight miss of full-year EPS guidance estimates. The results came as the 11 therapeutics stocks tracked by the report collectively beat consensus revenue estimates by 8% in Q2, with share prices up 24.8% on average since the latest earnings results. Biogen posted the group's best quarter, with revenues of $2.74 billion, up 3.4% year on year and 12.1% above expectations, while Myriad Genetics delivered the weakest performance, reporting revenues of $190.7 million, down 10.5% year on year and 8.2% below expectations, alongside full-year revenue guidance that missed and a significant EPS miss. Gilead Sciences reported revenues of $7.80 billion, up 10.2% year on year and 6.2% above expectations, and Moderna reported revenues of $145 million, up 2.1% year on year and 35.8% above expectations, the biggest analyst estimate beat of the group. AbbVie's stock is up 2.9% since reporting and currently trades at $264.79.

Johnson & Johnson's Caplyta met its primary endpoint in a late-stage bipolar mania study, sending shares up about 0.8% to $272.34 on Monday. Roughly 46% of patients on the once-daily 42-milligram dose achieved a clinical response versus about 21% on placebo, with improvement emerging within three days and continuing through the three-week study. Caplyta already treats schizophrenia and depressive episodes associated with bipolar disorder, so a mania win could push it deeper into the broader bipolar treatment cycle. The result matters because Johnson & Johnson spent $14.6 billion to acquire Caplyta developer Intra-Cellular Therapies, and a broader label would help justify that price. A positive trial is not regulatory approval, however, and investors still need to watch the filing timeline, eventual label language and physician uptake, especially with the stock trading 40.72% above its GF Value of $193.54.

Viatris Inc. announced on September 16 that Japan's Ministry of Health, Labour and Welfare approved WAKIX, or pitolisant, for narcolepsy and for excessive daytime sleepiness associated with obstructive sleep apnea syndrome in patients receiving treatment for airway obstruction. The narcolepsy approval covers types 1 and 2, with and without cataplexy, and the oral medicine is Japan's first approved histamine H3 receptor antagonist/inverse agonist for these indications. Viatris holds Japan-only rights under a license from Bioprojet, which discovered pitolisant, and the exclusive Japanese development and commercialization rights entered the portfolio through the October 2025 acquisition of Aculys Pharma. Viatris paid $35 million upfront to acquire Aculys Pharma, with additional consideration contingent on regulatory and commercial milestones, plus royalties on net sales, and the transaction also included Spydia rights in Japan and certain other Asia-Pacific markets. The approval announcement did not disclose a Japanese reimbursement price, launch date, sales forecast, or expected product margin, and Viatris reported approximately $3.8 billion of second-quarter revenue, up 5% on a reported basis.

AbbVie announced top-line results from a Phase 3 clinical trial showing its migraine treatment Qulipta significantly reduced migraine days versus placebo during the perimenstrual period in patients with menstrual migraine, the study's primary endpoint, while also meeting all secondary endpoints with no new safety signals. Qulipta, first approved in the U.S. in 2021 for migraine prevention and later expanded to chronic migraine in adults, could become the first medicine approved specifically for menstrual migraine, a niche with no approved treatments; AbbVie estimates a potential U.S. market of more than nine million adult patients in this indication. In the second quarter, Qulipta posted $350 million in sales, up 31% year over year, and some analysts expect peak sales of over $2 billion, against $1 billion in revenue last year, though AbbVie's total second-quarter revenue grew 10% year over year to $17 billion. AbbVie's larger growth drivers remain the immunology drugs Skyrizi and Rinvoq, which management expects to generate more than $31 billion in combined revenue this year, a milestone originally forecast for 2027 and raised by $4 billion from an earlier projection. The company also recently closed its $10.9 billion acquisition of Apogee Therapeutics, gaining zumilokibart, a late-stage immunology asset that some analysts say could reach $5.2 billion in peak eczema sales alone.

Axsome Therapeutics said AUVELITY prescriptions are accelerating after its sales-force expansion and the drug's launch in Alzheimer's disease agitation, with weekly new-to-brand prescriptions reaching a record 3,800. Speaking at Morgan Stanley's Global Healthcare Conference, Chief Financial Officer Nick Pizzie said patients aged 65 and older now account for about 31% of new-to-brand prescriptions, up from roughly 19% before the indication's approval, and that agitation is the fastest-growing segment. Average weekly new-to-brand prescriptions were about 2,400 in the first quarter and 3,000 in the second quarter. Management reiterated its $8 billion long-term peak-sales outlook for AUVELITY, split evenly between $4 billion in major depressive disorder and $4 billion in Alzheimer's disease agitation, and reported 89% coverage of total covered lives, including 100% Medicare coverage. Pizzie said second-quarter selling, general and administrative expense of $208 million should be a reasonable reference point for the back half of the year, while research and development expense of $46 million is expected to rise modestly, and management expects revenue to grow faster than operating expenses on a very clear path toward positive cash flow and profitability.

Novartis agreed to acquire full rights to Sironax's proprietary brain delivery platform for neurological disease treatments. The deal gives Novartis control of Sironax's technology designed to transport therapeutics across the blood brain barrier, expanding its toolkit for research into central nervous system disorders where treatment options remain limited. Novartis is a large pharmaceuticals group with a CHF218.8 billion market cap that researches, develops, manufactures, distributes, markets, and sells medicines globally, so gaining access to differentiated delivery technology directly links into its existing focus on complex neurological treatments. The Sironax platform speaks to the same advanced-therapy thesis that underpins Cosentyx's CHMP progress and remibrutinib's Phase 3 data, as Novartis seeks to own hard-to-reach biology where rivals like Roche and Biogen also compete for neurologists' attention. The flip side is that every new modality adds complexity, and recent trial setbacks such as pelacarsen and del-desiran keep development risk front and center, with analysts flagging that stacking capital-intensive projects on top of share buybacks could stretch the balance sheet of a business already carrying a high level of debt.