The brain is the most complex mass in the body, and the one place 98% of drugs can't reach. That's why diseases like Alzheimer's, Parkinson's, and depression have been the field where drugs fail most often in the history of pharma — but after decades of silence, 2024–2026 suddenly brought new drugs breaking through the wall, one at a time. And a market that grows with the world's aging population is now opening up.
Axsome Therapeutics Reports New Clinical Data in Depression and Alzheimer's Agitation
Axsome Therapeutics reported new clinical data in major depressive disorder and Alzheimer's disease agitation at a leading psychiatry congress. The presentations focused on key programs in Axsome's central nervous system pipeline aimed at treating mood and neuropsychiatric symptoms, and management highlighted how the new findings may inform future study designs and regulatory interactions for these indications. The company is advancing multiple late-stage clinical programs, including AXS-05, AXS-12, AXS-14, and several solriamfetol indications, and the fresh congress results give more clinical detail around AXS-05 in depression and Alzheimer's agitation, feeding the thesis that future CNS indications can broaden the revenue mix beyond Auvelity, Sunosi and SYMBRAVO. Axsome, a mid-sized US pharmaceuticals player with a market cap of $11.2b, competes alongside larger CNS-focused drug developers such as Biogen and Eli Lilly and smaller specialists. The same update also stress tests a key risk: management must commercialise several CNS assets at once while running high R&D and SG&A spending, with payer scrutiny and strong rivals in both categories.
Novartis Acquires Full Rights to Sironax Brain Delivery Platform
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.
AbbVie Unveils New VRAYLAR Real-World and Pediatric Safety Data at Psych Congress 2026
AbbVie shared new real-world and pediatric safety data for VRAYLAR in major depressive disorder and bipolar I at Psych Congress 2026. The findings covered routine clinical practice settings, including patients with complex comorbidities and underserved groups, while the pediatric data added information on safety and use in younger patients with mood disorders. AbbVie, a research-focused biopharmaceutical group with a US$466.6b market cap, positions VRAYLAR within a broad portfolio targeting chronic conditions including complex psychiatric disorders. The company said the next concrete checkpoint will be how these data feed into prescribing and label use over the next year, particularly uptake of newly approved low dose VRAYLAR in pediatric and adjunctive MDD settings, trackable in reported prescription trends and segment sales through 2027.
Teva Study Finds Providers Prioritize Drowsiness Risk When Choosing Tardive Dyskinesia Treatment for Older Patients
Teva Pharmaceuticals announced new data showing that healthcare providers prioritize drowsiness risk, the duration of available long-term response data and drug-drug interaction profile when selecting a VMAT2 inhibitor for tardive dyskinesia in patients aged 55 and older. In a discrete choice experiment, 489 healthcare providers ranked somnolence risk, accounting for 26.8% to 36.2% of decision-making weight, and short-term symptom improvement, at 24.4% to 29.5%, as their top priorities, followed by dose formulation at 16.7% to 20.2% and drug-drug interaction risk at 14.4% to 17.3%. Applying those preferences across four patient profiles, AUSTEDO, also known as deutetrabenazine, had the highest predicted choice probability, driven mainly by somnolence risk, duration of long-term response data and drug-drug interaction. The findings were presented at Psych Congress, held September 15 to 19, 2026, in New Orleans. A separate interim analysis from the IMPACT-TD Registry found that among patients with probable tardive dyskinesia who remained untreated with any VMAT2 inhibitor for 24 months, roughly 89% to 96% experienced at least a mild global impact and 60% to 74% experienced moderate-to-severe impact, while 55% to 69% reported stable or worsening severity with no evidence of spontaneous resolution.
Teva's UZEDY Outperforms Other Long-Acting Injectables in Real-World Schizophrenia Data
Teva Pharmaceuticals announced new real-world data showing its UZEDY (risperidone) extended-release injectable suspension helps schizophrenia patients stay on treatment longer while cutting emergency care and healthcare costs compared with other options. The findings, presented at Psych Congress in New Orleans, included a matched cohort of 1,166 patients, 583 per group, in which fewer once-monthly UZEDY patients had an emergency department visit than those on paliperidone palmitate once-monthly, 22.0% versus 27.8%, with a 40% lower all-cause ED visit rate and inpatient hospitalizations of 11.1% versus 14.8%. Estimated mean monthly direct healthcare costs were significantly lower for UZEDY, $4,688.10 versus $5,724.80, an adjusted mean monthly savings of $1,036.70 per patient. Against risperidone microspheres every two weeks, UZEDY patients achieved 3.6 times higher odds of adherence and an 80% lower likelihood of discontinuation, while outcomes versus aripiprazole once-monthly were generally comparable. In bipolar I disorder, a retrospective chart review of 358 matched patients, 179 per group, found those who switched from oral antipsychotics to UZEDY had significantly less treatment failure, 47% versus 63%.
Xenon Downgraded by Deutsche Bank After Pausing Depression Trial Enrollment
Deutsche Bank downgraded Xenon Pharmaceuticals to Hold from Buy after the Canadian biotech paused enrollment in multiple Phase 3 trials of its lead asset azetukalner in major depressive disorder and bipolar depression following several neuropsychiatric adverse events, including psychosis. Analyst David Hoang also slashed his price target on the stock to $46 from $90 per share, and Xenon shares fell 30% to their lowest level since November. Xenon management called the pause a temporary precaution with no impact on ongoing epilepsy studies, saying the events were in line with the drug's known safety and tolerability profile, but Hoang disagreed, saying the pause impairs the molecule's profile as a potential best-in-class drug and go-to branded antiseizure medication in focal epilepsy. Stifel, Baird, and RBC Capital Markets reaffirmed their bullish ratings, with Stifel analyst Paul Matteis citing minimal readthrough to focal onset seizures, RBC's Brian Abrahams trimming his target to $70 from $81 while keeping an Outperform rating, and Baird's Brian Skorney lowering his target to $90 from $97.
Trump to Announce Most Favored Nation Drug Pricing for Medicaid in All 50 States
President Trump will announce on Friday that Medicaid beneficiaries in all 50 states will receive Most Favored Nation pricing discounts on some prescription drugs. The White House Council of Economic Advisers projects the move will generate $27.6B in savings for state governments and $36.6B for the federal government over 10 years, according to Semafor, which broke the news. Trump is also scheduled to meet today with the governors of Arkansas, Mississippi, and South Dakota to discuss progress on lowering drug prices. Separately, an AARP Public Policy Institute report released in August found that applying MFN pricing to the top 10 drugs by Medicare Part D and Part B spending in 2025 would cut Medicare spending on those drugs from $273B to $76B between 2029 and 2033.
FDA Approves Ultragenyx Gene Therapy FAYUVI for Sanfilippo Syndrome Type A
The U.S. Food and Drug Administration has approved FAYUVI, also known as UX111, a gene therapy developed by Ultragenyx Pharmaceutical Inc. for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare and progressive neurodegenerative lysosomal storage disorder. Abeona Therapeutics Inc. congratulated Ultragenyx on the approval, noting that the therapy originated from pioneering research by Drs. Haiyan Fu and Douglas McCarty at The Ohio State University and Nationwide Children's Hospital and was advanced through clinical development by Abeona as ABO-102 before Abeona out-licensed global development and commercialization rights to Ultragenyx in May 2022. Abeona Chief Executive Officer Vish Seshadri called the approval an extraordinary milestone for patients and families affected by a devastating disease that has lacked effective therapeutic options. The approval also represents a meaningful value-creation event for Abeona, which under its licensing agreement with Ultragenyx is eligible to receive certain commercial milestone payments and royalties tied to future product sales. FAYUVI is designed to deliver a functional copy of the SGSH gene to the central nervous system via an AAV9 viral vector, addressing the root genetic cause of MPS IIIA.
CHMP Backs Roche's Ocrevus for Children and Teens With Relapsing MS
The European Medicines Agency's Committee for Medicinal Products for Human Use has recommended approval of Roche's Ocrevus, or ocrelizumab, intravenous infusion for patients aged 10 years and older with relapsing forms of multiple sclerosis, making it the first high-efficacy anti-CD20 treatment option for people with MS as young as 10 years old. The positive opinion rests on the Phase III OPERETTA 2 study, in which Ocrevus was non-inferior to fingolimod, the current standard treatment in paediatric MS, at controlling relapses and reduced the risk of relapses by 48% compared with fingolimod. In the same trial Ocrevus was superior at reducing brain inflammation, with significant reductions in new or enlarging T2 lesions of 48% and gadolinium-enhancing active T1 lesions of 87%, and its safety profile in children and teens was consistent with that seen in adults, with no patients stopping treatment due to side effects. The U.S. FDA approved Ocrevus for paediatric RMS patients in May 2026, and a final decision from the European Commission is expected in the near future. At least 40,000 children and adolescents are living with MS worldwide, with roughly one-third in Europe.
Eli Lilly Partners With QurCan Therapeutics on Genetic Medicines for Nervous System Diseases
Eli Lilly has entered an exclusive research collaboration with QurCan Therapeutics to develop genetic medicines for nervous system diseases. The agreement centers on QurCan's polymer lipid nanoparticle delivery platform for central and peripheral nervous system targets, with Eli Lilly responsible for later development and commercialization. Lilly is also making a strategic investment in QurCan Therapeutics, tying capital directly to progress in genetic medicine delivery technology. The deal pushes Lilly further toward expansion in neurodegenerative and other specialty drugs alongside its well known GLP 1 portfolio, though pricing pressure and payer pushback still hang over Mounjaro, Zepbound and Foundayo. Investors will want to see a first concrete output such as a nominated development candidate or an announced IND timeline for at least one nervous system genetic therapy.
Neurocrine Posts KINECT-PRO Data as Revenue Jumps 39%
Neurocrine Biosciences on August 12 published KINECT-PRO Phase 4 findings showing INGREZZA eased the daily burden of tardive dyskinesia beyond what clinician rating scales capture. The study followed 59 tardive dyskinesia patients on daily INGREZZA for 24 weeks, with 52 reaching the final visit; among the 45 patients in the efficacy analysis, about 58% hit the threshold for symptomatic remission on clinician-rated movement severity, with gains exceeding the clinically meaningful threshold by Week 4 on movement severity and by Week 8 on patient-reported impact. The data landed weeks after Neurocrine's July 30 second-quarter report, which showed total revenue up 39% year over year to $959 million and INGREZZA sales climbing 15% to $716 million on record new prescriptions, prompting management to raise full-year INGREZZA guidance to $2.825 billion to $2.875 billion. CRENESSITY and the newly acquired VYKAT XR added $184 million and $54 million respectively. The growth came at a cost: cash and marketable securities fell from roughly $2.5 billion at the end of 2025 to about $482 million by June 30, largely because Neurocrine paid $53.00 a share in cash to acquire Soleno Therapeutics in a $2.9 billion deal that closed in May, backstopped by a new $1.0 billion revolving credit facility. Second-quarter R&D rose to $327 million from $244 million a year earlier, SG&A jumped to $440 million from $286 million, and the Soleno deal added roughly $2.2 billion of intangible assets that will amortize over 16 years, while INGREZZA still supplied about three-quarters of quarterly revenue.
Xenon Submits Azetukalner NDA to FDA for Focal Seizures, Pauses Psychiatry Enrollment
Xenon Pharmaceuticals has submitted a New Drug Application to the U.S. Food and Drug Administration for azetukalner in focal seizures, based on positive data from the Phase 2b X-TOLE and Phase 3 X-TOLE2 trials in which all four doses produced statistically significant reductions in monthly seizure frequency versus placebo. The company also said it has voluntarily paused enrollment of new patients in its ongoing psychiatry studies in major depressive disorder and bipolar depression after an analysis of neuropsychiatric adverse events, though patients already enrolled remain on study and the pause is expected to be temporary. Enrollment in the X-NOVA2 study in MDD has reached approximately 80% of its initial target of 450 patients, and topline data are now expected in the first quarter of 2027. The Phase 3 X-TOLE3 and X-ACKT epilepsy studies continue to enroll focal seizure and primary generalized tonic-clonic seizure patients, respectively, and the epilepsy program has generated more than 1,500 patient-years of safety and exposure data. Xenon will host a conference call today at 4:30 pm Eastern Time to discuss the NDA submission and the psychiatry program update.
Scholar Rock Wins FDA Approval for ISEMBYLD in Spinal Muscular Atrophy
Scholar Rock Holding Corporation secured FDA approval on September 11 for ISEMBYLD, apitegromab-mstn, to treat spinal muscular atrophy in adults and children aged two and older currently receiving survival motor neuron 2-targeted therapy. In the Phase 3 SAPPHIRE trial, children aged 2 to 12 receiving the approved 10 mg/kg dose showed a 2.2-point advantage over placebo after one year on the Hammersmith Functional Motor Scale-Expanded, with a gain of at least three points occurring in 34.2% of treated patients versus 13.5% receiving placebo. Scholar Rock said its U.S. launch was underway, with shipments expected in the coming days, supported by a patient-support program for insurance coverage, financial assistance, and infusion logistics. Fractures occurred in 9% of patients receiving the approved dose versus 2% receiving placebo, and the safety information warns of fractures, including serious fractures, with clinicians possibly stopping treatment following a fracture. Insider Monkey's database showed 51 hedge funds holding Scholar Rock at the end of 2Q2026, up from 49 funds three months earlier.
Novartis Halts Development of ALS Drug Rifonebart After Mid-Stage Trial Misses Primary Endpoint
Swiss pharmaceutical giant Novartis has discontinued development of its candidate ALS treatment VHB937, also known as rifonebart, after a mid-stage clinical trial failed to achieve its primary endpoint. Endpoints News and Bloomberg News reported the news early on the 16th, and Novartis later confirmed the reports in an email to Reuters. Rifonebart failed to meet both its primary and secondary endpoints in the mid-stage trial, which evaluated the efficacy and safety of the drug in 251 patients with early-stage ALS within two years of symptom onset. The drug is designed to stabilize TREM2, a protein that helps regulate immune responses, inflammation, and the clearance of waste products in the brain. The decision to halt development follows the failure of a trial for a muscle-wasting disease treatment announced last week, in addition to setbacks with a closely watched heart disease treatment, and the company's move this month to pause eight of ten trials of its experimental cell therapy rap-cel after three patient deaths, heightening investor scrutiny of Novartis's development pipeline. Novartis is also pursuing research into the drug in patients with Alzheimer's disease, and according to a clinical trial registry, a mid-stage trial in Alzheimer's is still recruiting participants.
J&J's Caplyta Shows Improved Sexual Functioning in Late-Stage Depression Trial
Johnson & Johnson reported new late-stage data showing that Caplyta improved sexual functioning among depression patients, adding a potentially important commercial argument for the drug. More than 80% of the study's 480 participants entered the trial reporting sexual dysfunction, and patients receiving 42 milligrams of Caplyta alongside their existing antidepressants recorded statistically significant improvements versus placebo across measures including desire, arousal, orgasm and pleasure. Caplyta already carries approvals for schizophrenia, bipolar depression and adjunctive treatment of major depressive disorder, so better tolerability could give J&J another lever to differentiate the drug in a crowded psychiatric market. J&J picked up Caplyta through its $14.6 billion acquisition of Intra-Cellular Therapies, a sizable bet equal to roughly 57.7% of the company's latest $25.3 billion in quarterly sales. J&J shares were roughly flat, slipping about 0.1% to $266.965, while the stock trades 38.04% above the $193.40 GF Value estimate.
AbbVie Raises 2026 Neuroscience Outlook to $12.7 Billion on Broad-Based Growth
AbbVie has raised its full-year 2026 neuroscience revenue outlook to approximately $12.7 billion, $100 million above its previous forecast, after the franchise generated $6.1 billion in first-half 2026 revenues, up 21.8% year over year on an operational basis. The growth was broad-based across the portfolio's three pillars of psychiatry, migraine and Parkinson's disease. Vraylar, the cornerstone of the psychiatry franchise, generated $1.98 billion in the first half, up 18.6%, and is now expected to approach $4.1 billion in full-year sales, while Botox Therapeutic brought in $2.05 billion and is expected to reach nearly $4.2 billion. In migraine, Ubrelvy generated $731 million and Qulipta $646 million, with Qulipta leading growth at 39.2% and Ubrelvy at 26.4%. The Parkinson's therapy Vyalev generated $457 million in first-half sales, with second-quarter sales up more than 27% sequentially, and AbbVie said it is well on track to achieve blockbuster sales this year. AbbVie also raised its overall 2026 revenue outlook by $300 million to approximately $67.6 billion, and expects an FDA decision on tavapadon, a once-daily oral Parkinson's therapy, in the third quarter of 2026, with management projecting more than $5 billion in peak sales across its three Parkinson's therapies.
Eisai's Subcutaneous Leqembi Wins Health Ministry Approval, Enabling At-Home Dosing
Eisai and U.S. pharmaceutical giant Biogen announced on the 16th that the subcutaneous formulation of lecanemab, a treatment for Alzheimer's disease sold under the brand name Leqembi, has received manufacturing and marketing approval from the Ministry of Health, Labour and Welfare. The product, named Leqembi Subcutaneous Injection 250mg Pen, can be administered in about 15 seconds per pen, with two pens used per dose. Until now, patients needed intravenous infusions at a hospital once every two weeks, but the subcutaneous formulation will allow once-weekly self-administration at home. Doctor prescriptions and hospital visits to confirm safety will still be required. Following deliberation and approval by the Central Social Insurance Medical Council, prescriptions are expected to begin between mid-November and mid-December, making this the third country to approve the subcutaneous version, after the United States and China.
Barclays Downgrades Scholar Rock to Equal Weight Despite FDA Approval of Isembyld
Barclays downgraded Scholar Rock to Equal Weight from Overweight even after the FDA approved its lead asset, Isembyld, for spinal muscular atrophy last week. Analyst Etzer Darout cited a more balanced risk-reward setup post-approval and a cautious tone on the company's non-SMA pipeline, writing that additional clinical validation will likely be required before meaningful incremental value can be ascribed to the broader pipeline beyond SMA. The downgrade extended a slide in Scholar Rock shares, which have lost more than 14% since Friday, when the company became a commercial-stage biotech following the FDA nod for Isembyld as a late-line option for patients aged two years and older with SMA, though the stock is still up 7% year to date. Darout raised his price target on Scholar Rock to $58 from $55 per share, noting that the pricing management announced for Isembyld on Monday slightly exceeded his prior estimates. He added that future upside to his above-consensus estimates will increasingly depend on commercial execution that could surpass his expectations and on pipeline expansion opportunities beyond SMA.
AbbVie's Atogepant Meets Goal in Late-Stage Menstrual Migraine Trial
AbbVie Inc. said its migraine treatment atogepant, sold as Qulipta in the U.S., met the main goal of a late-stage study evaluating its ability to prevent migraines associated with the menstrual period. Patients taking the drug experienced an average of 0.8 fewer migraine days than those receiving placebo over three menstrual cycles, with the treatment taken for seven consecutive days around the expected start of menstruation. Because atogepant is already approved in the U.S. for migraine prevention, the new data could potentially support an additional indication without requiring AbbVie to establish an entirely new drug franchise. The company may still need to show a meaningful advantage for menstrual migraine patients, since the benefit was modest and competition remains intense from rivals including Eli Lilly and Pfizer. The new indication could also have a limited incremental revenue impact, as menstrual migraine represents a narrower patient population than the broader migraine-prevention market.
Study finds Trump's Medicare drug price crackdown risks pushing drugmakers to raise prices or withdraw medicines worldwide
A new study published in the medical journal The Lancet suggests that the Trump administration's efforts to cut U.S. drug spending could give pharmaceutical companies an incentive to raise prices or reduce sales of medicines in other countries around the world to offset lost revenue in the U.S. market. The Trump administration wants prices for drugs under the federal Medicare health program to align with levels in other wealthy countries, but researchers found that for about three in four of the medicines studied, if companies had to cut the prices they charge Medicare to match those of cheaper reference countries, the revenue lost in the U.S. would exceed the drug's entire sales in the reference country. The study analyzed 195 patented drugs, accounting for a total of 87.9 billion dollars in Medicare spending in 2024, comparing them with drug prices in 19 reference countries. It found that aligning the prices Medicare pays with those of the reference countries would save the two pilot programs the U.S. government uses to test the measure about 11.6 billion dollars. But if the 17 drugmakers that struck separate pricing agreements with the White House were exempted, the savings would fall to just 3.3 billion dollars. The effects are already emerging: Astellas Pharma said it was able to negotiate a higher price for a new eye disease treatment in Japan this year, while Chris Viehbacher, chief executive of Biogen, said the company would launch Zurzuvae, a treatment for postpartum depression, in only a few European countries, and Roche Holding said it may not launch a new oral breast cancer drug, which has not yet been approved in Switzerland, the company's home country.
Apeloa Pharmaceutical Subsidiary's Brivaracetam Tablets Receive US FDA Approval
Apeloa Pharmaceutical Co., Ltd. announced that its wholly owned subsidiary Zhejiang Apeloa Jutai Pharmaceutical Co., Ltd. has received notification from the US FDA that its abbreviated new drug application for Brivaracetam Tablets has been approved. The ANDA number is 220136, with approved strengths of 25 mg, 50 mg, and 100 mg, primarily indicated for the treatment of partial-onset seizures. The manufacturer is Zhejiang Apeloa Kangyu Pharmaceutical Co., Ltd. Brivaracetam Tablets are a novel antiepileptic drug and a derivative of the second-generation antiepileptic drug levetiracetam. According to relevant data, sales of Brivaracetam Tablets in the US market in 2025 were approximately 823 million US dollars. The company stated that this approval marks its qualification to sell the product in the US market, will further enhance the product's global market competitiveness, and actively expand into the US and other overseas markets. It also noted that production and sales of the product may be affected by uncertainties such as policy and market conditions.
Biogen Touts 10 Phase III Programs as It Diversifies Beyond Neurology
Biogen executives said the company is advancing 10 Phase III programs with clinical readouts beginning in the fourth quarter as it works to offset declines in its legacy multiple sclerosis business and return to growth. Speaking at a Wells Fargo conference, Chief Financial Officer Robin Kramer said the pipeline is at a pivotal point for growth, with the Fit for Growth initiative redirecting investment from the MS portfolio into newer launches including LEQEMBI, SKYCLARYS and ZURZUVAE. Head of corporate development Adam Keeney said Biogen has expanded into immunology and nephrology through deals including the HI-Bio acquisition and its felzartamab asset, whose initial focus is antibody-mediated rejection in kidney transplantation, where there are no approved therapies and roughly 11,000 U.S. patients experience secondary rejection. The Apellis acquisition added commercial products and nephrology capabilities, including SYFOVRE in geographic atrophy, and Biogen expects the deal to be dilutive in 2026 mainly on interest expense while targeting roughly $250 million in annualized cost savings exiting next year and paying down the related debt by the end of 2027. Keeney said Biogen expects to focus more on earlier-stage business development through 2027, particularly in immunology, rare disease and neurology, and is not financially constrained for early-stage transactions.
Teva Named Stalking-Horse Bidder for BioXcel Assets in $125 Million Deal
Teva Pharmaceutical Industries has agreed to serve as the stalking-horse bidder for substantially all of BioXcel Therapeutics' assets in a transaction valued at up to $125 million, structured as a $57.5 million upfront payment plus up to $67.5 million in conditional milestone payments. The bid sets the initial baseline in a court-supervised Section 363 auction, meaning other buyers can compete for the assets before the sale closes. BioXcel filed for Chapter 11 bankruptcy protection on August 27 after a weeks-long cash crisis, securing $19 million in debtor-in-possession funding from current lenders, and its shares fell more than 70% to roughly $0.19. The assets center on BioXcel's neuroscience portfolio: Igalmi, an already-approved dexmedetomidine sublingual film for acute agitation in adults with schizophrenia or bipolar I or II disorder, which will remain commercially available during bankruptcy, and the pending label expansion for BXCL501 for at-home use, with an FDA PDUFA target action date of November 14. The deal fits Teva's "Pivot to Growth" neuroscience strategy, following its separate $700 million acquisition of Emalex Biosciences and its lead asset ecopipam, which closed in June.
Ovid Therapeutics Posts Wider Q2 Loss, Advances Two Clinical Programs
Ovid Therapeutics reported second-quarter financial results on August 13, with cash of $169.8 million that the company says funds operations into 2029. The biopharmaceutical company said OV4071, described as the first oral direct KCC2 activator, is in an ongoing Phase 1 study in healthy volunteers, with data expected to support a Phase 2 proof-of-concept study in acute schizophrenia in 2027, and a ketamine challenge study planned for the second half of 2026. Its second program, OV329, a next-generation GABA-AT inhibitor, entered a Phase 2 randomized, placebo-controlled study in treatment-resistant focal onset seizures expected to finish in the second half of 2027. Ovid also secured equity and milestone rights in a new company formed by Perceptive Advisors that acquired global rights to soticlestat from Ovid and Takeda, making Ovid eligible for up to $294.5 million in potential clinical, regulatory, and commercial milestones plus royalties. Research and development expenses rose to $10.0 million from $6.5 million a year earlier, total operating expenses reached $16.4 million, and the net loss widened to $15.0 million, or $0.08 per share, from $4.7 million, or $0.06 per share.
Lilly completes acquisition of AtaiBeckley to advance treatment-resistant depression therapies
Eli Lilly and Company has completed its acquisition of AtaiBeckley Inc., a clinical-stage biopharmaceutical company developing rapid-acting neuroplastogens for mental health conditions. AtaiBeckley's lead investigational program, BPL-003, targets treatment-resistant depression. The transaction was approved by AtaiBeckley shareholders at a special meeting held prior to closing. Carole Ho, Lilly executive vice president and president of Lilly Neuroscience, said AtaiBeckley's neuroplastogens have the potential to open a new paradigm of treatment with rapid-acting therapeutics designed to move away from chronic dosing. Lilly said it is committed to advancing the pipeline for patients who need more options.
AbbVie's Qulipta Hits Phase 3 Goal in Menstrual Migraine
AbbVie said its migraine drug Qulipta achieved the main goal of a Phase 3 trial targeting menstrual migraine, sending shares up about 0.4% to $252.25 Thursday morning. The trial tested a sharper, more targeted dosing schedule, giving patients atogepant for seven straight days starting three days before menstruation across three cycles. Qulipta reduced perimenstrual migraine days by 0.8 versus placebo while also improving headache burden, rescue-medication use, disability and cognitive symptoms. Commercially, Qulipta delivered $350 million in second-quarter revenue, rising 30.9% and accounting for roughly 10.8% of AbbVie's $3.23 billion neuroscience portfolio. The $252.25 share price stands 14.55% above the $220.20 GF Value estimate, and detailed safety results, regulatory approval and insurer coverage still separate positive data from new revenue.
Roche Wins FDA Priority Review for Enspryng in Rare MOGAD Disorder
Roche announced that the FDA has accepted its supplemental biologics license application seeking approval of Enspryng, also known as satralizumab, for the treatment of myelin oligodendrocyte glycoprotein antibody-associated disease, or MOGAD, and granted the application a priority review with a decision expected on Jan. 10, 2027. If approved, Enspryng would become the first and only disease-modifying therapy for MOGAD, a rare autoimmune disease of the central nervous system that can cause unpredictable attacks affecting the optic nerves, spinal cord or brain and for which there are currently no approved treatments. The FDA's acceptance was based on positive data from the phase III METEOROID study, which met its primary endpoint, showing that treatment with Enspryng reduced the risk of a new MOGAD relapse by 68% compared with placebo, and that at 48 weeks 87% of patients treated with Enspryng remained relapse-free compared with 67% in the placebo arm. Separately, the European Medicines Agency has validated Roche's application for Enspryng in MOGAD, with a decision from the European Commission expected in the third quarter of 2027. Enspryng is currently approved in several countries for the treatment of neuromyelitis optica spectrum disorder, including the European Union and the United States, and Roche is also evaluating it in additional neurological autoimmune and inflammatory disorders, with the FDA having accepted and granted priority review in June 2026 to a separate sBLA seeking label expansion for thyroid eye disease and a final decision expected on Oct. 15, 2026.
Novartis Plunges 10.1% After Lead Drug Fails in Trial
Novartis shares plunged 10.1% to $138.51 on Tuesday after its drug del-desiran failed to meet the primary endpoint in a pivotal trial for myotonic dystrophy, erasing about CHF24 billion in market value. The setback directly impacts Novartis's $12 billion acquisition of Avidity Biosciences, of which del-desiran is the lead asset. The HARBOR study, involving roughly 150 patients, showed no statistically significant improvement over placebo in video hand-opening time, though some secondary measures hinted at clinical activity and safety remained consistent. Management will review the full data before deciding on further investment. The failure leaves Avidity's two other programs under greater scrutiny, and while Novartis still targets 5% to 6% annual sales growth through 2030, investors now demand harder evidence from the remaining pipeline.
Lilly Builds Neuroscience into a New Long-Term Growth Driver
Eli Lilly is expanding its neuroscience portfolio through internal development and acquisitions, aiming to diversify beyond its dominant cardiometabolic franchise. Neuroscience currently accounts for only about 2% of Lilly's total sales, but first-half 2026 revenues in this segment grew 32% to $811 million, driven by products like Kisunla for Alzheimer's disease. The company has built a pipeline spanning Alzheimer's, sleep disorders, pain, and schizophrenia, with key candidates including remternetug, cleminorexton, and a GBA1 gene therapy. Recent acquisitions, such as AtaiBeckley and Centessa, have added promising assets like BPL-003 for treatment-resistant depression and cleminorexton for hypersomnia. While neuroscience is still a small part of Lilly's business, its rapid growth and pipeline potential could make it an increasingly important growth driver, reducing reliance on GLP-1 therapies. Lilly's stock trades at 26.85 times forward earnings, below its five-year average of 34.57, and the Zacks Consensus Estimate for 2026 has risen to $35.93 per share.
Johnson & Johnson to Present New CAPLYTA Bipolar Mania Data at Psych Congress
Johnson & Johnson announced that 24 abstracts featuring clinical data and real-world evidence across its neuropsychiatry portfolio will be presented at the 2026 Psych Congress Annual Meeting in New Orleans from September 15-19. Among the highlights are the first presentation of pivotal Phase 3 data for CAPLYTA in adults with bipolar mania, along with additional CAPLYTA data in bipolar depression and major depressive disorder, and new analyses of SPRAVATO on anhedonia. The company will also present Phase 3 data for seltorexant in major depressive disorder with insomnia symptoms, and real-world studies on its long-acting injectables for schizophrenia. Jane Tiller, Vice President and Global Head of Development for Neuroscience at Johnson & Johnson, emphasized the company's commitment to advancing research in areas where patients and clinicians face significant treatment challenges.
Novartis Faces $3 Billion MS Opportunity and Key Risks
Novartis AG could be staring at a $3 billion annual peak-sales opportunity in multiple sclerosis if its drug remibrutinib successfully advances through the regulatory process and gains meaningful market share. Bank of America estimates that remibrutinib could generate about $3 billion in peak annual sales in multiple sclerosis alone and potentially more than $10 billion across all indications. The comments follow encouraging results from the REMODEL-1 and REMODEL-2 Phase 3 trials announced on September 2, which showed that remibrutinib reduced relapse activity and met primary endpoints in patients with relapsing multiple sclerosis. The global MS therapeutics market is expected to grow at a 5.9% compound annual growth rate to approximately $38.6 billion by 2030, and Novartis already has a major presence through Kesimpta, which generated approximately $1.42 billion in second-quarter sales, up 32% year over year. However, risks include potential slowdown in Kesimpta growth, competition from Roche's BTK inhibitor, and the uncertainty of regulatory approval and physician adoption. Hedge fund interest is rising, with 38 funds holding stakes in the second quarter, up from 31 in the first quarter, while short interest remains low at approximately 0.26% of public float.
Roche Gets FDA Clearance for Alzheimer's Blood Test
Roche Holding announced a collaboration with Treeline Biosciences to study combination immunotherapies for B-cell lymphomas, and received FDA clearance for its Elecsys pTau217 blood test, the first single biomarker assay to help rule in and rule out amyloid pathology in Alzheimer's disease. The test is intended to support earlier and more accessible Alzheimer's diagnostic assessments. Roche, a pharmaceuticals and diagnostics group with a CHF285.2 billion market cap, is expanding its oncology pipeline through partnerships and its diagnostics portfolio with neurology assays. The lymphoma partnership and the blood test clearance align with Roche's strategy of combining pipeline breadth and diagnostics depth, though the company faces competition from Novartis, Bristol Myers Squibb, Abbott, and Siemens Healthineers.
Ionis Gains FDA Approval for First Alexander Disease Treatment
The U.S. FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen) as the first treatment for Alexander disease, a rare neurological disorder affecting fewer than 1,000 people in the U.S. The drug, designed to reduce abnormal GFAP protein production, showed statistically significant improvement in gait speed in a pivotal study and is approved for both pediatric and adult patients. Ionis plans to commercialize Zanvastro independently in the U.S., marking a key step in its strategy to launch wholly owned neurology medicines, while Recordati holds rights outside the U.S. Analysts at William Blair estimate peak annual sales of approximately $295 million, though the small patient population and complex administration may limit uptake. The approval validates Ionis' RNA-targeting platform and strengthens its neurology portfolio, which includes Spinraza, Wainua, and Qalsody.
Ultragenyx plunges 46% on trial failure, multiple downgrades
Ultragenyx Pharmaceutical plunged more than 46% to an all-time low on Thursday after its Phase 3 Aspire study for apazunersen, an experimental Angelman syndrome therapy, failed to meet the primary endpoint. The Novato, California-based biopharma said it will review future operations, including R&D plans for the drug. In response, RBC Capital Markets downgraded the stock to Sector Perform from Outperform and cut its price target to $19 from $40, while Baird downgraded to Neutral from Outperform, and Bank of America downgraded to Neutral from Buy with a target cut to $20 from $48. Analysts noted the lack of numerical data and suspected the trial was a complete miss, though Ultragenyx's commercial operations provide some valuation floor.
Biohaven Licenses Epilepsy Drug Opakalim to SK Biopharma for Up to $795M
Biohaven and SK Biopharmaceuticals have announced a global licensing agreement for opakalim, Biohaven's lead epilepsy candidate, in a deal worth up to $795 million plus royalties. Under the agreement, SK Biopharmaceuticals obtains exclusive worldwide rights to Biohaven's Kv7 ion channel platform, while Biohaven receives $400 million in near-term cash, including $350 million at closing and $50 million in 2027. The deal also includes up to $150 million in development and regulatory milestones, with tiered royalties on US net sales ranging from the mid-teens to low twenties. SK Biopharmaceuticals will assume Kv7 program costs, including certain Knopp Biosciences obligations worth up to $245 million plus mid-single-digit royalties. The agreement follows positive proof-of-concept data in epilepsy, where opakalim extended the median time to a second seizure to 141 days versus 47 days on placebo, and 54% of patients in an open-label extension study saw at least a 50% reduction in seizure frequency. Biohaven's CEO stated the deal demonstrates the company's ability to monetize its pipeline through partnerships, providing non-dilutive cash to fund other programs such as BHV-1300 and BHV-1400. The pivotal RISE3 trial results are expected in the second half of 2026.
China Approves LEQEMBI Subcutaneous Formulation for Early Alzheimer's
Eisai Co., Ltd. and Biogen Inc. announced that China's National Medical Products Administration has approved the subcutaneous formulation of LEQEMBI (lecanemab) as an initiation treatment for early Alzheimer's disease, making it the first and only anti-amyloid therapy in China that enables at-home administration. The approval covers a once-weekly 500 mg regimen delivered via a subcutaneous autoinjector, offering an alternative to the intravenous infusion given every two weeks in a hospital. This marks the second country globally to approve the subcutaneous initiation treatment, following the United States in July 2026. The approval is based on data from the Phase 3 Clarity AD study and subcutaneous administration sub-studies, which showed equivalent exposure and similar safety and efficacy to the IV form. Eisai plans to launch the product in China during its fiscal year ending March 31, 2027, and estimates that there were 17 million patients with early Alzheimer's in China in 2024.
ST Renfu's RFUS-949 Tablets Enter Phase II Clinical Trial
ST Renfu announced that RFUS-949 tablets, developed by its controlling subsidiary Yichang Renfu, have recently entered Phase II clinical trial research. The study is a multicenter, randomized, double-blind, parallel, placebo-controlled design, aimed at evaluating the efficacy and safety of the drug for postoperative analgesia. The drug received a drug clinical trial approval notice from the National Medical Products Administration in May 2025, with the approved indication for the treatment of acute and chronic pain.
Novartis Remibrutinib Meets Primary Endpoint in Late-Stage MS Studies
Novartis announced positive top-line results from the late-stage REMODEL-1 and REMODEL-2 studies, showing that its oral BTK inhibitor remibrutinib significantly reduced annualized relapse rate compared to Aubagio in patients with relapsing multiple sclerosis. The identical phase III trials, which enrolled approximately 2,000 patients worldwide, also demonstrated favorable safety and clinically meaningful reductions in disability progression. Following the news, Novartis shares rose 6%, bringing year-to-date gains to 17%. The company plans to present full data at MSToronto2026 and pursue global regulatory approvals for remibrutinib in RMS. This comes as Novartis already markets remibrutinib as Rhapsido for chronic spontaneous urticaria, which generated $101 million in first-half 2026 sales.
SciSparc Subsidiary Seeks FDA Orphan Drug Status for Tourette Drug
SciSparc Ltd. announced that its subsidiary, NeuroThera Labs Inc., is pursuing U.S. Food and Drug Administration Orphan Drug Designation for SCI-110, its drug candidate for treating adults with Tourette Syndrome, and has engaged RK Strategies, a U.S. government and public affairs firm, to support the regulatory pathway. The designation, if granted, could strengthen the product's market profile, as it is reserved for therapies targeting rare diseases affecting fewer than 200,000 people in the United States. SCI-110, a cannabinoid-based candidate combining dronabinol and palmitoylethanolamide, recently advanced into a Phase IIb program at three academic centers, building on Phase IIa results that showed an average tic reduction of 21% as measured by the Yale Global Tic Severity Scale. The engagement with RK Strategies aims to boost visibility among federal stakeholders and reinforce the importance of new treatment options for adult Tourette Syndrome.
Novartis Jumps 6% as MS Drug Meets Goals in Two Phase 3 Trials
Novartis shares surged about 6% to $161.10 on Tuesday after its oral drug remibrutinib met primary goals in two Phase 3 trials for multiple sclerosis, showing fewer relapses and brain lesions than teriflunomide without the liver-safety concerns investors had feared. The company also reported that pooled results indicated slower disability progression, potentially giving remibrutinib another edge in a large treatment market. The drug, already sold as Rhapsido for chronic hives, generated only $64 million in second-quarter sales, but full results are due in October, with global regulatory submissions to follow. At $161.10, Novartis trades 23.13% above its GF Value estimate of $130.84, reflecting high investor expectations. The rally also distinguishes remibrutinib from eight suspended rap-cel studies after three deaths linked to severe immune reactions, with two deaths in the remibrutinib trials ruled unrelated to treatment.