RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Special Review Project (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Priority Review (China)

Structure/Sequence

Molecular FormulaC22H23N7O

InChIKeyASKZRYGFUPSJPN-UHFFFAOYSA-N

CAS Registry1825352-65-5

Clinical Trials associated with Risdiplam

NCT07047144

/ RecruitingPhase 2

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score <55.

Start Date15 Sep 2025

Sponsor / Collaborator

Scholar Rock, Inc.

JPRN-jRCTs031240475

/ RecruitingNot Applicable

A specific clinical study on the efficacy and safety of combined therapy with Risdiplam treatment and HAL-based robotic rehabilitation in patients with spinal muscular atrophy (HARMONY-SMA)

Start Date08 Nov 2024

Sponsor / Collaborator-

NCT05861999

/ RecruitingPhase 4

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children <2 years of age genetically diagnosed with SMA.

Start Date14 Aug 2024

Sponsor / Collaborator-

100 Clinical Results associated with Risdiplam

100 Translational Medicine associated with Risdiplam

100 Patents (Medical) associated with Risdiplam

313

Literatures (Medical) associated with Risdiplam

01 Aug 2026·NEUROSCIENCE

Beyond SMN1 mutation: environmental predispositions and epigenetic contributors to phenotypic variability and disease severity in spinal muscular atrophy

Review

Author: Arockiaraj, Jesu ; Prethiba, S ; Gopi, Sanjay ; Karthick Raja Namasivayam, S ; Chandhru, M

Spinal Muscular Atrophy (SMA) is an autosomal recessive neurodegenerative disorder caused by loss-of-function mutations in the survival motor neuron 1 gene (SMN1), and with SMN2 being a known principal modifier yet significant phenotypic variability exists even among individuals sharing identical genetic backgrounds, including siblings, suggesting that factors beyond SMN2 copy number contribute substantially to clinical heterogeneity. Eventhough with only lesser direct relevances, emerging evidences implicate environmental exposures and epigenetic mechanisms as key modulators of this variability. Hypoxia accelerates disease progression by promoting SMN2 exon 7 skipping through upregulation of the splicing repressors hnRNP A1 and Sam68, while metabolic dysfunctions including hepatic steatosis and dyslipidemia, independent of motor neuron degeneration, have been reported in approximately one third of SMA patients at autopsy. Differential DNA methylation at CpG sites upstream of the SMN2 promoter correlates with disease severity, identifying a potential epigenetic prognostic biomarker. The three currently approved disease-modifying therapies, nusinersen, risdiplam, and onasemnogene abeparvovec, have fundamentally transformed SMA management, yet their efficacy may be modulated by environmental factors including nutritional status, respiratory function, infection burden, and pre-existing immunity to viral vectors, underscoring the need for integrated environmental and epigenetic assessment in SMA clinical care and research. This review integrates SMA-specific evidence with mechanistically plausible hypotheses drawn from related neurodegenerative conditions, clearly distinguishing established findings from emerging concepts. We propose that integrating environmental monitoring, nutritional optimization, and epigenetic modulation with genetic therapies may enable precision medicine approaches that maximize therapeutic benefit across the SMA clinical spectrum.

01 Jul 2026·PEDIATRIC NEUROLOGY

Characterization of Safety Signals for Spinal Muscular Atrophy Treatments in Children Aged 0 to 3 Years Potential Safety Patterns for Further Investigation

Article

Author: Tang, Xinghang ; Chen, Li ; Zhang, Yupeng

BACKGROUND:

Spinal muscular atrophy (SMA) has become a manageable chronic condition with the advent of three major disease-modifying therapies. However, real-world safety data in children aged 0-3 years remain limited. This study aimed to characterize safety signals using long-term pharmacovigilance data.

METHODS:

Adverse drug event reports related to SMA therapies submitted to the FDA Adverse Event Reporting System were extracted from the first quarter of 2015 to the third quarter of 2025. Disproportionality analyses were conducted to detect signals.

RESULTS:

A total of 1,155; 408; and 957 Adverse drug event reports were identified for onasemnogene abeparvovec (OA), risdiplam, and nusinersen as the primary suspected drugs, respectively, with balanced sex distributions across the three therapies. Reports involving OA more frequently included abnormalities in liver function-related parameters. Signals related to respiratory failure, atelectasis, and pneumothorax were identified across all three therapies. Reports involving OA also showed a broader spectrum of renal and urinary preferred terms, including proteinuria, hematuria, and glycosuria.

CONCLUSIONS:

Distinct safety signal patterns were identified among the three SMA disease-modifying therapies in children 0-3 years. These findings provide postmarketing insights into their safety profiles. Further prospective studies are warranted to validate these findings.

01 Jul 2026·CLINICAL THERAPEUTICS

Advancements in Prenatal Diagnosis and Potential Fetal Therapies for Spinal Muscular Atrophy

Review

Author: Baptiste, Caitlin ; Arditi, Brittany ; Trahan, Marie-Julie

PURPOSE:

Spinal Muscular Atrophy (SMA) is a rare autosomal recessive disorder caused by SMN1 gene mutations, resulting in muscle weakness and atrophy, respiratory failure, and death. SMA disease modifying therapies (DMTs) include the antisense oligonucleotide (ASO) nusinersen administered intrathecally, onasemnogene abeparvovec, single-dose intravenous gene replacement therapy that introduces functional SMN1 via an adeno-associated viral vector, and oral risdiplam, which modifies SMN2 splicing to increase SMN protein production. With DMTs, infants can achieve previously unattainable developmental milestones and survive beyond infancy. Prenatal carrier screening and universal newborn screening allow early identification and prompt postnatal treatment. However, with severe early-onset SMA, motor neuron loss begins in utero and irreversible damage may occur prior to treatment initiation. Therefore, fetal therapies for SMA are a focus of ongoing research. This review article focuses on current postnatal therapies, summarizes research on potential fetal therapies and their potential clinical integration, and reviews the ethical implications of fetal therapy for SMA.

METHODS:

This is a narrative review. Prospective study data for FDA-approved DMTs are discussed, focusing on presymptomatic patients. For articles related to fetal therapies, Pubmed and Ovid/MEDLINE were searched using the terms "spinal muscular atrophy" and "in utero therapy," "prenatal therapy," or "fetal therapy." Eleven articles were identified; nine were included.

FINDINGS:

Prenatal SMA is diagnosed via chorionic villus sampling or amniocentesis. SMN2 copy number testing can identify fetuses with severe disease who may benefit from fetal therapy. The three FDA-approved DMTs are potential fetal therapy targets. ASOs have been administered by intracranial and intraamniotic injection to lambs, demonstrating feasibility of prenatal ASOs; however, this approach requires refinement before human use. SMA gene therapy has been studied in mice and lambs; CNS transduction following cordocentesis in lambs was observed. However, further study of potential maternal and fetal adverse effects is required to ensure safety. Finally, a case of third trimester maternal risdiplam use was recently published with promising results: the two-year-old infant has no clear SMA manifestations and normal motor function.

IMPLICATIONS:

Early postnatal treatment is currently standard of care for prenatally- and postnatally diagnosed SMA with improvement in outcomes demonstrated following early treatment initiation. Fetal therapy is an emerging research area and shows promise for infants with severe disease in whom motor neuron loss begins in utero. Fetal therapy for SMA is ethically acceptable and likely feasible based on animal studies and a single case report. Ongoing rigorous attention to maternal and fetal safety is of utmost importance as fetal therapy for SMA approaches clinical use.

274

News (Medical) associated with Risdiplam

30 Jul 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Phase 1Financial StatementExecutive ChangePhase 2Phase 3

29 Jul 2026

·www.fiercepharma.com

Biogen flips '26 guidance from expected decline to sales increase

With increased sales of its newer drugs compensating for declining revenues from its multiple sclerosis (MS) portfolio, Biogen is once again positioned for a “growth story,” CEO Chris Viehbacher said Wednesday during the company’s second-quarter earnings call. To that end, Biogen reported revenue of $2.74 billion for the period, marking its highest quarterly haul since Q3 of 2021. With the result, the company flipped its 2026 revenue guidance from an expected mid-single digit decline in 2026 to a mid-single digit increase. “This quarter is a reflection of the significant progress Biogen has made repositioning the company for long-term growth,” Viehbacher added. Biogen acknowledged that part of the guidance reversal can be attributed to its $5.6 billion buyout of Apellis Pharmaceuticals. The May acquisition brought two growth drivers—geographic atrophy drug Syfovre and rare disease treatment Empaveli—into the commercial fold. Combined sales for the duo, which were approved in 2023 and 2021, respectively, increased by 22% for the full quarter. Also playing a role in the revenue boost (PDF) were three drugs that reached the market in 2023. Alzheimer’s disease treatment Leqembi was up 15% year over year to $184 million. Friedreich’s ataxia drug Skyclarys saw a 29% gain to $168 million. And postpartum depression therapy Zurzuvae realized a 53% increase to $71 million. Also pointing to Zurzuvae’s momentum was a 28% sequential boost in sales. Biogen also reported positive news on the launch of its new high-dose (HD) formulation of Spinraza. In March, when Biogen gained approval for the new version of the spinal muscular atrophy (SMA) treatment 10 years after it was originally endorsed, the company touted its improved efficacy and convenience for patients. “What I think you’re seeing is Spinraza still setting the bar on efficacy in this space,” Alisha Alaimo, Biogen’s president of North America operations, said during the conference call. “If we think about metrics, Spinraza high dose is exceeding the original launch of Spinraza in both start forms and grads in the first 13 weeks of launch and we are growing every single week.” Spinraza was the first drug on the market for SMA, but its sales have been eroding since peaking at $2.10 billion in 2019, last year coming in at $1.55 billion. Meanwhile, Roche’s Evrysdi, which was approved in SMA in 2020, pulled in sales of 1.76 billion Swiss francs ($2.11 billion) in 2025, and Novartis’ gene therapy Zolgensma, which reached the market in 2019 in the same indication, generated sales of $1.23 billion last year. With sales of $402 million in the second quarter, which were up 2%, Spinraza remains Biogen’s second best-selling drug behind aging MS treatment Tysabri, which itself enjoyed a “resilient performance” in Q2, according to Mizuho analyst Salim Syed. Tysabri sales hit $451 million for the quarter, which were down 1% year over year and up 2% sequentially, Syed pointed out. Biogen's overall annual revenue, which peaked at $14.4 billion in 2019, was in decline each year until 2025, when it ticked upward by 2% to $9.9 billion. Now, with the Apellis acquisition and the positive momentum of its new products, the company is on track for another sales boost. “This is a major shift for Biogen. Three and [a] half years ago, when I came, we really were only visiting neurologists. We had a few products that we could still promote in MS and we had Spinraza," Viehbacher said. "We are now looking at visiting rheumatologists, dermatologists, nephrologists, outside the U.S. epileptologists, and [kidney] transplants. There’s been a significant growth in the breadth of our portfolio.”

Drug ApprovalAcquisitionClinical Result

28 Jul 2026

·www.globenewswire.com

Bicara Therapeutics Announces Appointments of Jeremy Bender and Christy Oliger to Board of Directors

BOSTON, July 28, 2026 (GLOBE NEWSWIRE) -- Bicara Therapeutics Inc. (Nasdaq: BCAX), a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors, today announced the appointments of Jeremy Bender, Ph.D., MBA and Christy Oliger to its Board of Directors. Dr. Bender, who most recently served as Chief Executive Officer at Day One Biopharmaceuticals through its acquisition by Servier in 2026, brings more than 20 years of corporate strategy and operating experience in biotechnology to Bicara. Ms. Oliger brings more than 30 years of commercial and operating experience in global biopharmaceutical organizations, having most recently served as Senior Vice President of the Oncology Business Unit at Genentech. "We are delighted to welcome Jeremy and Christy to our Board of Directors at this pivotal time for Bicara. Together, they bring deep commercial, strategic, and operational expertise that will be invaluable as we continue our evolution from a clinical-stage organization to one focused on successfully delivering ficerafusp alfa to patients,” said Claire Mazumdar, Ph.D., MBA, Chief Executive Officer of Bicara Therapeutics. “Each of these distinguished professionals has an established track record of guiding innovative biopharmaceutical companies through critical stages of growth, including pipeline development, corporate strategy, and commercialization. Their perspectives will strengthen our Board as we advance our portfolio, execute on our long-term vision, and continue building Bicara into a mature biopharmaceutical organization.” In addition, Kiran Mazumdar-Shaw has retired from Bicara’s Board of Directors. "As one of our founding Board members, Kiran has been instrumental in shaping Bicara from its earliest days. Her impact extends beyond the initial seed capital that helped launch the company, but also the strategic guidance and steadfast support that has enabled Bicara to advance ficerafusp alfa into the clinic and reach the important milestone of a registration-enabling clinical study,” said Mike Powell, PhD, Chairman of Board of Bicara Therapeutics. “On behalf of the Board, management team, and Bicara employees, I want to thank Kiran for her vision, leadership, and indelible contributions to Bicara.” About Jeremy Bender, Ph.D., MBADr. Bender most recently served as Chief Executive Officer, President and a member of the Board of Directors of Day One Biopharmaceuticals, Inc. from September 2020 to April 2026, where he led the company through its evolution into a commercial-stage oncology company, including the approval and launch of OJEMDA® for pediatric low-grade glioma and the company's approximately $2.5 billion acquisition by Servier in 2026. Prior to joining Day One, Dr. Bender was Vice President of Corporate Development at Gilead Sciences, Inc., a pharmaceutical company, from March 2018 to September 2020. Prior to that, he was Chief Operating Officer of Tizona Therapeutics, Inc. from July 2015 to March 2018 and Chief Business Officer of Sutro Biopharma, a biotechnology company specializing in cancer and autoimmune therapeutics, from October 2012 to July 2015. Prior to joining Sutro Biopharma, Inc., he was Vice President of Corporate Development at Allos Therapeutics Inc., a biotechnology company focused on cancer treatments, from January 2006 to September 2012. Dr. Bender began his career in the life sciences practice at Boston Consulting Group, a management consulting company. Dr. Bender also sits on the boards of Mereo BioPharma Group plc and Aura Biosciences, Inc. From October 2023 to June 2024, Dr. Bender also served as a director of Fusion Pharmaceuticals, Inc., a biopharmaceutical company. Dr. Bender holds a B.S. in Biological Sciences from Stanford University, a Ph.D. in Microbiology and Immunology from the University of Colorado, and an MBA from the MIT Sloan School of Management. About Christy OligerMs. Oliger is recognized for building industry leading capabilities and teams and was the Senior Vice President, Business Unit Head for Oncology and Genentech from 2017-2020, managing a portfolio of 15 products, contributing U.S. revenue exceeding $13 billion, delivering launches in lung cancer, breast cancer, chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), melanoma and leading an organization of more than 800 people. Prior to this role, Ms. Oliger served as Senior Vice President and Business Unit Head, Neurology, Rare Disease, Infectious disease where she was responsible for building the teams and driving successful launches in multiple sclerosis (MS) (Ocrevus®), hemophilia (Hemlibra®) and spinal muscular atrophy (SMA) (Evrysdi®), together the most significant growth drivers in the U.S. business. Prior to these roles, she spent three years as Global Head of Portfolio and Project Management where her team was responsible for the Late-Stage Portfolio Committee comprised of a late-stage development portfolio of greater than 300 projects across multiple therapeutic areas. Christy began her career in life sciences at Schering Plough, a pharmaceutical company. In addition to Bicara, Christy also currently sits on the boards of Vera Therapeutics, Karyopharm Therapeutics, and Replimune Inc. She previously served as a director at Nuvalent Inc. prior to acquisition by GSK, Sierra Oncology prior to acquisition by GSK, Reata Pharmaceuticals prior to acquisition by Biogen, Rayze Bio prior to acquisition by BMS, and Lava Oncology prior to acquisition by Xoma Therapeutics. She holds a B.A. in Economics from University of California, Santa Barbara. About Bicara Therapeutics Bicara is a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors. Bicara has built a platform designed to facilitate the development of bifunctional therapies that precisely target the tumor and deliver a tumor-modulating payload to the tumor site. This approach was deployed in the development of Bicara’s lead program ficerafusp alfa, formerly BCA101, a bifunctional epidermal growth factor receptor (EGFR) directed monoclonal antibody bound to a human transforming growth factor beta (TGF-β) ligand trap. By combining these two clinically validated targets, ficerafusp alfa has the potential to exert potent anti-tumor activity by simultaneously blocking both cancer cell-intrinsic EGFR survival and proliferation, as well as the immunosuppressive TGF-β signaling within the tumor microenvironment (TME). Ficerafusp alfa directs the TGF-β inhibitor into the immediate TME through the binding of EGFR on tumor cells, which Bicara believes will lead to deep and durable responses and an increase in overall survival, while reducing the potential adverse effects previously associated with systemic TGF-β inhibition. Ficerafusp alfa is being developed in head and neck squamous cell carcinoma, where there remains a significant unmet need, as well as other solid tumor types. For more information, please visit www.bicara.com or follow us on LinkedIn and X. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “may,” “might,” “will,” “could,” “would,” “should,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and similar words or expressions, or the negative thereof, are intended to identify forward-looking statements, although not all contain identifying words. Any statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, express or implied statements regarding Bicara’s strategy, business plans and focus; the clinical development of ficerafusp alfa, including planned and ongoing clinical trials; the expected therapeutic potential and clinical benefits of ficerafusp alfa, including potential efficacy, depth, durability and tolerability; Bicara’s ability to scale and prepare for potential commercialization of ficerafusp alfa; the potential for U.S. regulatory approval and launch of ficerafusp alfa; and anticipated contributions of members of Bicara’s Board of Directors. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks relating to Bicara’s research and development activities; Bicara’s ability to execute on its business plans and strategy, including obtaining the requisite regulatory approvals on the expected timeline, if at all; uncertainties relating to the clinical development of ficerafusp alfa; the Company’s dependence on third parties; risks related to the Company’s financial condition and need for additional funds in order to commercialize ficerafusp alfa, if approved; risks related to regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; risks related to establishing and maintaining Bicara’s intellectual property protections; and risks related to the competitive landscape for ficerafusp alfa; as well as other risks described in “Risk Factors,” in Bicara’s most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Bicara’s subsequent filings with the U.S. Securities and Exchange Commission (SEC). In addition, any forward-looking statements represent Bicara’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Bicara explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Bicara intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company’s Investor Relations website, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcasts. Contacts InvestorsRachel Frank IR@bicara.com MediaTim PalmerTim.Palmer@bicara.com

Executive ChangeAcquisition

100 Deals associated with Risdiplam

External Link

KEGG	Wiki	ATC	Drug Bank
D11406	Risdiplam	M09A	DB15305

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Spinal Muscular Atrophy	United States	Genentech, Inc.	07 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	United States	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	United States	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Japan	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Japan	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Belgium	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Belgium	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Brazil	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Brazil	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Croatia	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Croatia	Roche China Holding Ltd.	19 Dec 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


AAN 12026 \| AAN 22026 \| AAN 32026 \| AAN 42026 \| AAN 52026 \| AAN 62026 \| AAN 72026 \| AAN 82026 \| AAN 92026 \| AAN 102026	Not Applicable	Spinal Muscular Atrophy	268	Risdiplam	qiquvrrbmg(hlvdolikso) = ytgzlcsjnq prgyiqatmr (xgyywyzznu ) View more	Positive	25 Apr 2026
NCT03779334 (RAINBOWFISH, MDA2026)	Phase 2	Spinal Muscular Atrophy	23	Risdiplam	zipdgwdwnc(casedozxwi) = oopmhxntkh stmclnpave (trnypnvsqd ) View more	Positive	08 Mar 2026
MDA2026	Not Applicable	Spinal Muscular Atrophy	950	Risdiplam	axtiazdync(axbgeigwil) = yqbceihlmb fongenddil (mifemavxdj ) View more	Negative	08 Mar 2026
MDA2026	Not Applicable	Spinal Muscular Atrophy	950	Nusinersen	axtiazdync(axbgeigwil) = uuoqmdlluk fongenddil (mifemavxdj ) View more	Negative	08 Mar 2026
NCT03032172 (Jewelfish, CTgov)	Phase 2	Spinal Muscular Atrophy	174	Risdiplam	oewhagwkjv(cgdoisvaxq) = yncaeqmuln dtikakjcqn (bwtgthmqkt, tpfedyajpx - kazziksteb) View more	-	01 Oct 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	EVRYSDI	nvwgwdtjuf(lxsvhjlfxv) = hacthuhmwq kptmescgec (tegyabijim, 0.61 - 2.11) View more	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	Placebo	nvwgwdtjuf(lxsvhjlfxv) = hwzhrwcppu kptmescgec (tegyabijim, -1.22 to 0.84) View more	Positive	11 Feb 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (full treated population)	fbetzdjueb(ydbvkiumyp) = ocdhqyemsg vsjvvxfbqv (jkgtyyzwxw ) View more	Positive	11 Feb 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (2 copies of SMN2)	fbetzdjueb(ydbvkiumyp) = amykblvczi vsjvvxfbqv (jkgtyyzwxw )	Positive	11 Feb 2025
NCT02913482 (FDA_CDER) Manual	Phase 2	HMN (Hereditary Motor Neuropathy) Proximal Type I	62	EVRYSDI	wdyegscxcs(pdsrvxazyo) = aonwbljbtj qrervngujo (jtqonqjyoj ) View more	Positive	11 Feb 2025
NCT03779334 (RAINBOWFISH, GlobeNewswire) Manual	Phase 2	Spinal Muscular Atrophy SMN2 copies	23	Evrysdi	hmywsvtbrv(ldirldxsls) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). rnrqtthsfa (yyqjnrqszx ) Met View more	Positive	14 Oct 2024
NCT02913482 (FIREFISH, NEWS) Manual	Not Applicable	Spinal Muscular Atrophy	-	Evrysdi	jrktzkmisf(rprplxfmjx) = eexghxylmo oexmljhvbu (hktiowelma ) View more	Positive	14 Jun 2024
NCT03779334 (RAINBOWFISH, CTgov)	Phase 2	Spinal Muscular Atrophy	26	risdiplam (2 SMN2 Copies, Risdiplam)	ggtfldogxf = jvqhnomhcc edldabrfdo (ytrmnsfirl, nuzerczfwg - gprnydvcgi) View more	-	05 Mar 2024
NCT03779334 (RAINBOWFISH, CTgov)	Phase 2	Spinal Muscular Atrophy	26	risdiplam (3 SMN2 Copies, Risdiplam)	bwedjkwkro(wicdmmhtnj) = duizgabeaf penwgkeblu (xabvfbpiux, bpkvvitmfo - fvtyjcnkok) View more	-	05 Mar 2024

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