Nature Medicine Publishes Results from the Pivotal DEVOTE Study of High-Dose Regimen of Nusinersen in Spinal Muscular Atrophy

Rhea-AI Summary

Biogen (NASDAQ: BIIB) announced publication in Nature Medicine of pivotal DEVOTE results showing the high-dose nusinersen regimen (50 mg loading, 28 mg maintenance) produced statistically significant motor improvements and faster neurofilament decline versus a prespecified matched sham in infantile-onset SMA.

DEVOTE enrolled 139 participants; Part B infants showed a CHOP-INTEND mean difference of 26.19 points versus matched sham. The high-dose safety profile was broadly consistent with the 12 mg regimen. The high-dose regimen is approved in the EU and Japan and is under FDA review with a PDUFA date of April 3, 2026.

Positive

• CHOP-INTEND mean difference +26.19 points versus matched sham
• DEVOTE enrolled 139 participants across ages and SMA types
• Faster reduction in neurofilament observed with high-dose regimen
• High-dose regimen approved in EU and Japan

Negative

• Serious adverse events ≥10%: pneumonia, pneumonia aspiration, respiratory failure
• Most common AEs (≥15%) included respiratory infections and pneumonia
• High-dose regimen under FDA review with PDUFA date April 3, 2026

Market Reaction

+4.86% $185.35

15m delay 22 alerts

+4.86% Since News

$185.35 Last Price

$177.10 $186.24 Day Range

+$1.26B Valuation Impact

$27.19B Market Cap

0.8x Rel. Volume

Following this news, BIIB has gained 4.86%, reflecting a moderate positive market reaction. Our momentum scanner has triggered 22 alerts so far, indicating elevated trading interest and price volatility. The stock is currently trading at $185.35. This price movement has added approximately $1.26B to the company's valuation.

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Key Figures

DEVOTE enrollment: 139 participants High-dose loading: 50 mg/5 mL High-dose maintenance: 28 mg/5 mL +5 more

8 metrics

DEVOTE enrollment 139 participants Phase 2/3 DEVOTE study across ages and SMA types

High-dose loading 50 mg/5 mL Two loading doses 14 days apart in high-dose regimen

High-dose maintenance 28 mg/5 mL Maintenance dose every four months in high-dose regimen

CHOP-INTEND difference 26.19 points High-dose vs prespecified matched sham group in treatment‑naïve infants

CHOP-INTEND change +15.1 vs -11.1 High-dose vs sham group motor score change, DEVOTE Part B

Statistical significance p<0.0001 CHOP-INTEND comparison for treatment‑naïve symptomatic infants

Part C cohort size 40 participants Open-label DEVOTE Part C ages 4–65 transitioning to high-dose

Motor scale gains HFMSE +1.8; RULM +1.2 Mean change from baseline at Day 302 after transition

Market Reality Check

Price: $176.76 Vol: Volume 1,537,710 is near ...

normal vol

$176.76 Last Close

Volume Volume 1,537,710 is near the 20-day average of 1,567,381 (relative volume 0.98). normal

Technical Shares at $176.76 trade 7.07% below the 52-week high and above the 200-day MA at 146.3.

Peers on Argus

BIIB was down 1.3% while key peers were mixed; GSK appeared in momentum scans up...

1 Up

BIIB was down 1.3% while key peers were mixed; GSK appeared in momentum scans up 4.14% with no news, and other majors like BMY and PFE showed smaller gains, indicating stock-specific trading for BIIB rather than a coordinated sector move.

Historical Context

5 past events · Latest: Jan 28 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 28	Regulatory designation	Positive	+0.4%	FDA Breakthrough Therapy Designation for litifilimab in cutaneous lupus.
Jan 25	Regulatory filing	Positive	+0.9%	FDA accepts LEQEMBI IQLIK sBLA with Priority Review for SC dosing.
Jan 12	EU approval	Positive	-1.1%	European Commission approval of high‑dose SPINRAZA regimen for 5q SMA.
Dec 22	Clinical data	Positive	-0.1%	JAMA Neurology publication of long‑term Phase 3 VALOR and OLE QALSODY data.
Dec 05	Clinical data	Positive	-0.3%	AES presentation of zorevunersen Phase 1/2a and OLE results in Dravet syndrome.

Pattern Detected

Recent positive R&D and regulatory catalysts have often seen muted or negative next-day moves, with 3 of 5 upbeat announcements followed by share price declines.

Recent Company History

Over the past few months, Biogen has highlighted multiple late-stage and regulatory milestones. In Dec 2025, long‑term QALSODY Phase 3 data in SOD1‑ALS and zorevunersen epilepsy data showed disease‑modifying potential. In Jan 2026, the EU approved the high‑dose SPINRAZA regimen supported by DEVOTE data, the FDA accepted a priority sBLA for subcutaneous LEQEMBI, and litifilimab received Breakthrough Therapy Designation. Today’s DEVOTE publication further reinforces this pattern of data‑driven neurology updates across Biogen’s pipeline and franchises.

Market Pulse Summary

This announcement highlights Nature Medicine publication of pivotal DEVOTE results for high‑dose nus...

Analysis

This announcement highlights Nature Medicine publication of pivotal DEVOTE results for high‑dose nusinersen, showing a CHOP‑INTEND mean difference of 26.19 points and additional motor gains after transition from 12 mg dosing. It reinforces earlier data used for the EU high‑dose SPINRAZA approval and comes ahead of a U.S. FDA PDUFA decision. Investors may track regulatory outcomes, uptake of the higher‑dose regimen in regions where it is approved, and how these results interact with Biogen’s broader neuromuscular and Alzheimer’s franchises.

Key Terms

neurodegeneration, neurofilament, phase 2/3, open-label, +1 more

5 terms

neurodegeneration medical

"slowed neurodegeneration more rapidly, as measured by neurofilament, than"

Neurodegeneration is the gradual damage and loss of nerve cells in the brain and nervous system, producing symptoms such as memory decline, impaired movement, or thinking problems. Think of it like electrical wiring in a house slowly fraying, which undermines how the system runs. For investors it matters because it drives demand for diagnostics, treatments and long-term care, influences regulatory risk and trial outcomes, and can significantly affect the value of health-care and biotech companies.

neurofilament medical

"more rapid reduction in neurofilament, a marker of neurodegeneration. In DEVOTE,"

A neurofilament is a structural protein that helps give nerve cells their shape and strength and is released into spinal fluid or blood when those cells are damaged; think of it like a building’s steel beam that sheds rust when the structure is harmed. Investors care because measuring neurofilament levels offers a simple, measurable signal of nerve injury used to track disease progression, select patients and show whether a drug is working in clinical trials, which can affect a therapy’s market prospects and risk profile.

phase 2/3 medical

"published results from the Phase 2/3 DEVOTE study evaluating the high-dose"

A phase 2/3 trial is a combined clinical study that first evaluates how well a treatment works and the best dose, then expands into a larger test to confirm those results and safety. For investors, it matters because moving into a phase 2/3 signals that an experimental therapy has shown initial promise and will be tested at scale, which can materially change the odds and timeline for regulatory approval and commercial potential.

open-label medical

"In the open-label Part C (n=40) of DEVOTE, a diverse group"

Open-label describes a situation where everyone involved in a study or process knows the full details, such as who is receiving a treatment or intervention. For investors, understanding whether a project or product is open-label helps gauge the level of transparency and potential biases, influencing trust and decision-making. It’s like knowing whether a test or experiment is conducted openly or behind closed doors.

pdufa regulatory

"and has a Prescription Drug User Fee Act (PDUFA) action date of April 3."

PDUFA, short for the Prescription Drug User Fee Act, is a law that allows drug companies to pay fees to the government to speed up the review process for new medicines. This helps bring important drugs to market more quickly, which can impact their availability and pricing. For investors, PDUFA timelines can influence the timing of a drug’s approval and potential market success.

AI-generated analysis. Not financial advice.

• Findings from DEVOTE support clinical benefits of the high-dose regimen of nusinersen (50 mg and 28 mg) in both treatment-naïve individuals and those previously treated with 12 mg nusinersen
• The high-dose regimen of nusinersen also slowed neurodegeneration more rapidly, as measured by neurofilament, than the 12 mg regimen
  
  

CAMBRIDGE, Mass., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that Nature Medicine published results from the Phase 2/3 DEVOTE study evaluating the high-dose regimen of nusinersen, comprised of 50 mg/5 mL loading and 28 mg/5 mL maintenance doses, in spinal muscular atrophy (SMA). The high-dose regimen of nusinersen offers a more rapid loading regimen, two 50 mg doses 14 days apart, and a higher maintenance regimen, 28 mg, every 4 months, compared to the 12 mg nusinersen regimen (SPINRAZA^®). The results showed the safety and effectiveness of the high-dose regimen of nusinersen across a broad range of people living with SMA, irrespective of age, prior treatment experience, and baseline functional status.

“One of the most notable changes seen with the higher dose regimen was a more rapid reduction in neurofilament, a marker of neurodegeneration. In DEVOTE, treatment with the high-dose regimen led to improvement across important domains like motor and bulbar function, respiratory health and hospitalizations, and survival,” said Richard Finkel, M.D., director, Center for Experimental Neurotherapeutics (CENT) at St. Jude Children’s Research Hospital. “Publication of the DEVOTE results in Nature Medicine further validate the importance of these data and the future role that I expect the high dose regimen will play as we look to continue to improve outcomes for people living with SMA.”

DEVOTE is a three-part study that enrolled 139 participants across ages and SMA types. Results from the pivotal cohort of the study (Part B) showed that treatment-naïve, symptomatic infants who received the high-dose regimen of nusinersen experienced statistically significant improvements in motor function as measured by the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), when compared to a prespecified matched sham (untreated) group from the ENDEAR study* (mean difference: 26.19 points; +15.1 vs. -11.1, p<0.0001). Results favored the high-dose regimen relative to sham across secondary endpoints and trended in favor of the high-dose regimen over the currently approved 12mg regimen on key biomarker and efficacy measures. 

“The publication of the DEVOTE data are an important step in our commitment to bring the high dose regimen of nusinersen to people living with SMA as quickly as possible,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. “We are grateful to the participants, their families, study investigators and site staff, and our co-authors who have made the DEVOTE study and publication of these results a reality.”

In the open-label Part C (n=40) of DEVOTE, a diverse group of participants, age 4-65, transitioned to the high-dose regimen (one 50 mg dose four months after their last 12 mg dose, followed by the 28 mg maintenance regimen, every four months) after a median of 3.9 years on the 12 mg regimen. Participants experienced improvements in motor function after transitioning with mean increases of 1.8 points on the Hammersmith Functional Motor Scale – Expanded (HFMSE) and 1.2 points on the Revised Upper Limb Module (RULM) from baseline at Day 302.

The safety profile of the high-dose regimen of nusinersen was broadly consistent with the known safety profile of the 12 mg regimen. In the Part B infantile-onset cohort of 50 participants, the most common adverse events (AEs; ≥15% of participants) in the high-dose regimen group were pneumonia, respiratory failure, pyrexia, COVID-19, and upper respiratory tract infection. The most common serious AEs (occurring in at least 10% of participants in the high-dose regimen group) were: pneumonia, pneumonia aspiration, and respiratory failure.

The high-dose regimen of SPINRAZA (nusinersen) is approved in the European Union and Japan. The high-dose regimen is under review with the United States Food and Drug Administration (FDA) and has a Prescription Drug User Fee Act (PDUFA) action date of April 3.

*ENDEAR is one of the two pivotal studies that formed the basis of regulatory approvals for nusinersen 12 mg.

About SPINRAZA
The high dose regimen of SPINRAZA (nusinersen) which is comprised of 50 mg/5 mL and 28 mg/5mL injections are approved in the European Union and Japan to treat infants, children and adults with spinal muscular atrophy (SMA). The high dose regimen of nusinersen is currently under review with the U.S. Food and Drug Administration (FDA) a Prescription Drug User Fee Act (PDUFA) action date of April 3, 2026. SPINRAZA 12 mg/5 mL injection is approved for SMA in more than 71 countries.¹

The low dose regimen of SPINRAZA has shown efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years,^52,3 combined with unsurpassed real-world experience. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. 

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). For more information, visit your respective country's product website. For the U.S., please click here for Important Safety Information and full Prescribing Information .

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube.

Biogen Safe Harbor
This news release contains forward-looking statements, including, among others, relating to: the potential benefits, efficacy and safety of higher doses of nusinersen (marketed as SPINRAZA); the potential to improve outcomes for, and address unmet needs of, patients with SMA; potential regulatory discussions, submissions, decisions and approvals and the timing thereof; the anticipated benefits, risks and potential of our collaboration arrangements; the potential of our commercial business and pipeline programs, including nusinersen; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would” or the negative of these words or other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements.

These forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to differ materially from those stated or implied in this document, including, among others, uncertainty of our long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans, prospects and timing of actions relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third-parties, intellectual property, competitive and market challenges and regulatory compliance; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; and the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; and any other risks and uncertainties that are described in reports we have filed with the U.S. Securities and Exchange Commission, which are available on the SEC’s website at www.sec.gov.

These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.

Digital Media Disclosure
From time to time we have used, or expect in the future to use, our investor relations website (investors.biogen.com), the Biogen LinkedIn account (linkedin.com/company/biogen-) and the Biogen X account (https://x.com/biogen) as a means of disclosing information to the public in a broad, non-exclusionary manner, including for purposes of the SEC’s Regulation Fair Disclosure (Reg FD). Accordingly, investors should monitor our investor relations website and this social media channel in addition to our press releases, SEC filings, public conference calls and webcasts, as the information posted on them could be material to investors.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022. 
2. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.
3. Finkel RS, et al. Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA. Presented at: Cure SMA Conference; 2024; Austin, Texas.
   
   

MEDIA CONTACT: Biogen Madeleine Shin + 1 781 464 3260 public.affairs@biogen.com

INVESTOR CONTACT: Biogen Tim Power +1 781 464 2442 IR@biogen.com

FAQ

What did Biogen (BIIB) report about the DEVOTE high-dose nusinersen results on Feb 4, 2026?

The DEVOTE study showed the high-dose nusinersen regimen produced significant motor improvements and faster neurofilament decline. According to the company, Part B infants had a CHOP-INTEND mean difference of 26.19 points versus a prespecified matched sham.

How many participants did the DEVOTE study (BIIB) enroll and what cohorts were included?

DEVOTE enrolled 139 participants across ages and SMA types, including infantile-onset and older participants. According to the company, Part B was the pivotal infant cohort and Part C included participants aged 4–65 who transitioned from 12 mg to high-dose.

What motor function changes did participants show after switching to the high-dose nusinersen in DEVOTE Part C?

Participants in Part C showed modest motor gains after transition to high-dose nusinersen. According to the company, mean increases were +1.8 HFMSE and +1.2 RULM from baseline at Day 302.

What safety findings did Biogen (BIIB) report for the high-dose nusinersen regimen in DEVOTE?

The high-dose safety profile was broadly consistent with the 12 mg regimen, but serious AEs were reported. According to the company, serious events ≥10% included pneumonia, pneumonia aspiration, and respiratory failure in the infant cohort.

Is the high-dose nusinersen regimen approved and what is the FDA timeline for BIIB?

The high-dose regimen is approved in the European Union and Japan and is under FDA review in the U.S. According to the company, the PDUFA action date for the FDA review is April 3, 2026.