A Phase III Multicenter Randomized, Double-Blind, Double-Dummy, Parallel-Group Study To Evaluate The Efficacy And Safety Of Fenebrutinib Compared With Teriflunomide In Adult Patients With Relapsing Multiple Sclerosis

Start Date28 Feb 2023

Sponsor / Collaborator

Hoffmann-La Roche, Inc.

100 Clinical Results associated with Fenebrutinib

100 Translational Medicine associated with Fenebrutinib

100 Patents (Medical) associated with Fenebrutinib

Literatures (Medical) associated with Fenebrutinib

01 Jun 2024·Current Opinion in Neurology

Bruton tyrosine kinase inhibitors in multiple sclerosis: evidence and expectations

Review

Author: Wiendl, Heinz ; Kraemer, Julia

Purpose of reviewDespite availability of high-efficacy therapies for multiple sclerosis (MS), many patients experience significant disability worsening due to limited effects of currently available drugs on central nervous system (CNS)-compartmentalized inflammation. Bruton tyrosine kinase (BTK) is an intracellular signaling molecule involved in regulation of maturation, survival, migration, and activation of B cells and microglia, which are central players in the immunopathogenesis of progressive MS. Therefore, CNS-penetrant BTK inhibitors may better prevent disease progression by targeting immune cells on both sides of the blood–brain barrier. This review gives an overview on the preliminary results of clinical trials.Recent findingsCurrently, the efficacy and safety of six BTK inhibitors are being evaluated in clinical trials in patients with relapsing and progressive MS. Evobrutinib, tolebrutinib and fenebrutinib have shown efficacy and safety in relapsing MS in phase 2 studies, and evobrutinib and tolebrutinib in their extension studies up to 3–5 years. However, evobrutinib failed to distinguish itself from the comparator drug teriflunomide in reduction of relapse rate (primary end point) in two phase 3 studies in relapsing MS.SummaryInhibition of BTK has emerged as a promising therapeutic approach to target the CNS-compartmentalized inflammation. Results from phase 3 clinical trials will shed light on differences in efficacy and safety of BTK inhibitors and its potential role in the future MS landscape.

01 Jun 2024·Drugs in R&D

Comparative CNS Pharmacology of the Bruton’s Tyrosine Kinase (BTK) Inhibitor Tolebrutinib Versus Other BTK Inhibitor Candidates for Treating Multiple Sclerosis

Article

Author: Turner, Timothy J ; Ofengeim, Dimitry ; Gruber, Ross C ; Brun, Pricilla

BACKGROUND AND OBJECTIVESTolebrutinib is a covalent BTK inhibitor designed and selected for potency and CNS exposure to optimize impact on BTK-dependent signaling in CNS-resident cells. We applied a translational approach to evaluate three BTK inhibitors in Phase 3 clinical development in MS with respect to their relative potency to block BTK-dependent signaling and exposure in the CNS METHODS: We used in vitro kinase and cellular activation assays, alongside pharmacokinetic sampling of cerebrospinal fluid (CSF) in the non-human primate cynomolgus to estimate the ability of these candidates (evobrutinib, fenebrutinib, and tolebrutinib) to block BTK-dependent signaling inside the CNS.RESULTSIn vitro kinase assays demonstrated that tolebrutinib reacted with BTK 65-times faster than evobrutinib, while fenebrutinib, a classical reversible antagonist with a K_i value of 4.7 nM and slow off-rate (1.54 x 10^-5 s^-1), also had an association rate 1760-fold slower (0.00245 μM^-1 * s^-1). Estimates of cellular potency were largely consistent with the in vitro kinase assays, with an estimated IC₅₀ of 0.7 nM for tolebrutinib against 33.5 nM for evobrutinib and 2.9 nM for fenebrutinib. We then observed that evobrutinib, fenebrutinib, and tolebrutinib achieved similar levels of exposure in non-human primate CSF after oral doses of 10 mg/kg. However, tolebrutinib CSF exposure (4.8 ng/mL) (k_p,uu CSF=0.40) exceeded the IC90 (the estimated concentration inhibiting 90% of kinase activity) value, while evobrutinib (3.2 ng/mL) (k_p,uu CSF=0.13) and fenebrutinib (12.9 ng/mL) (kp,uu CSF=0.15) failed to reach the estimated IC₉₀ values.CONCLUSIONSTolebrutinib was the only candidate of the three that attained relevant CSF exposure in non-human primates.

01 May 2024·Journal of Neuroimmunology

Recent advances in the treatment of primary and secondary progressive Multiple Sclerosis

Review

Author: Jaiswal, Shruti ; Amatya, Suban ; Sriwastava, Shitiz ; Bhatia, Dipika ; Elkhooly, Mahmoud ; Lisak, Robert P ; Shrestha, Kriti ; Kagzi, Yusuf ; Gupta, Rajesh

BACKGROUNDThe article highlights upcoming potential treatments, which target different phases of inflammation and offer remyelinating strategies as well as direct and indirect neuroprotective and oligodendrocyte protective effects, providing a hopeful outlook for patients with primary and secondary progressive multiple sclerosis (PPMS and SPMS).OBJECTIVESThe review aims to identify potential treatments and ongoing clinical trials for PPMS and SPMS, and compare their mechanisms of action, efficacy, and side effects with current treatments.METHODSWe reviewed ongoing clinical trials for PPMS and SPMS on the NIH website, as well as articles from PubMed, Embase, and clinicaltrails.gov since 2010.RESULTSBTKIs like, tolebrutinib, and fenebrutinib are being explored as potential PMS treatments. Vidofludimus calcium, an orally available treatment, has shown a reduction of active and new MRI lesions. Other treatments like simvastatin, N-acetylcysteine (NAC), and alpha-lipoic acid are being explored for their antioxidant properties. AHSCT and mesenchymal stem cell therapy are experimental options for younger patients with high inflammatory activity.CONCLUSIONSSPMS and PPMS are being studied for new treatments and future trials should consider combination therapies targeting inflammation, demyelination, and neuronal death, as the pathogenesis of PMS involves complex factors.

News (Medical) associated with Fenebrutinib

30 Jan 2025

·www.fiercepharma.com

Vabysmo crosses $4B mark, Polivy joins blockbuster club as Roche preps for key readouts

Roche CEO Thomas Schinecker highlighted 12 key pivotal trial readouts, including from four new molecular entities, that could happen in 2025. After repeatedly wowing investors, Roche’s star eye med Vabysmo collected more than $4 billion in sales during its third year on the market. Still, questions continue to swirl about an evolving market showdown with Regeneron’s rival drug.Vabysmo has supplanted cancer immunotherapy Tecentriq to take the No. 3 spot on Roche’s annual sales ranking. The bispecific eye injection brought in 3.86 billion Swiss francs ($4.3 billion), including 1.05 billion Swiss francs in the fourth quarter.Despite 43% year-over-year growth, Vabysmo’s fourth-quarter sales missed analyst expectations by 2%, according to Intron Health. The performance came after several quarters of Street-beating sales for the drug.During an investor call Thursday, Roche’s pharma chief Teresa Graham suggested that the fourth-quarter haul was affected by a “spend-down” and “significant channel filling” right after the launch of a more convenient prefilled syringe version of Vabysmo, which was approved by the FDA in July and EU in December.Starting in January, Roche is seeing “a strong trajectory” for Vabysmo sales, Graham said, assuring investors of continued strong growth and further market-share expansion in 2025.Vabysmo’s market share in the U.S. in the fourth quarter was 31% in neovascular age-related macular degeneration, 22% in diabetic macular edema and 23% in retinal vein occlusion. The numbers were 30%, 22% and 20%, respectively, in the third quarter.Graham stressed that Vabysmo’s fourth-quarter share numbers were not directly comparable to previous quarters because a data aggregator recently issued a “restatement” of third-quarter share figures. If analyzed by prior metrics, Vabysmo expanded by roughly three percentage points across each of the three indications, she said.Lately, the competition between Vabysmo and a high-dose version of Regeneron’s Eylea has been the focus of industry watchers. In September, European regulators also approved a prefilled syringe of Eylea HD. Nevertheless, Roche is “really not seeing any impact in any part of the world” from the arrival of Eylea HD, Graham said.The Roche exec highlighted how the two products’ syringes are “materially different,” as the Vabysmo product offers one-hand administration, she said. “When you think about it, if you’re doing […] 100 shots a day, having that real ease of administration is super important,” she added. “I would say we definitely have device preference going on our side.” As for Tecentriq, sales of the PD-L1 inhibitor declined by 3.3%—or remained flat at constant exchange rates—last year.“We do believe, as we have mentioned previously, that Tecentriq is getting close to peak, and we expect sales growth to be in the zero to low-single-digit range going forward,” Graham said.In 2024, Roche welcomed antibody-drug conjugate Polivy to the blockbuster club. With full-year sales of 1.12 billion Swiss francs, the drug ranked 16th on the company’s list of top meds.Although Polivy’s fourth-quarter sales, at 304 million Swiss francs, were flat over the prior quarter and came in 6% below Wall Street’s estimates, the drug expanded its U.S. market share in the key first-line diffuse large B-cell lymphoma indication to 29%, according to Graham.Roche has increasingly been leaning on new drugs like Vabysmo for growth. In 2024, a “young portfolio” of drugs contributed to 56% of Roche’s 46.2 billion Swiss francs of pharma sales, up from 51% in 2023. On Thursday’s call, Roche CEO Thomas Schinecker highlighted 12 key pivotal trial readouts, including from four new molecular entities, that could happen in 2025.One of the key assets is giredestrant. The oral SERD is nearing phase 3 readouts from the persevERA and evERA trials in HR-positive, HER2-negative breast cancer, around the middle or the second half of 2025, according to Roche. Schinecker has put the drug’s peak sales potential across multiple indications at above $3 billion.The other three new meds with pivotal readouts expected in 2025 are astegolimab in COPD, fenebrutinib in multiple sclerosis and vamikibart in uveitic macular edema.

Drug ApprovalPhase 3ADCBiosimilar

13 Nov 2024

·www.prnewswire.com

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

07 Nov 2024

·www.globenewswire.com

Rapport Therapeutics Reports Third Quarter Financials and Provides Business Update

Biotech industry leaders added to the Board of Directors, bringing deep expertise in drug discovery, neuroscience clinical development, and operational leadershipRAP-219 MAD-2 and PET trials ongoing; topline data expected in Q1 2025Actively recruiting patients for Phase 2a trial of RAP-219 in focal epilepsy; trial on track and topline data expected in mid-2025 Investigational New Drug Application (IND) in diabetic peripheral neuropathic pain placed on clinical hold while the Company addresses U.S. Food and Drug Administration (FDA) Phase 2a trial protocol feedback Ended the quarter with $320.7 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations through the end of 2026 BOSTON and SAN DIEGO, Nov. 07, 2024 (GLOBE NEWSWIRE) -- Rapport Therapeutics, Inc. (Nasdaq: RAPP), a clinical-stage biotechnology company focused on discovery and development of small molecule precision medicines for patients suffering from central nervous system (CNS) disorders, today announced financial results for the third quarter of 2024 and provided a business update. "We are pleased with the progress we’re making with RAP-219, particularly as we continue the execution of our Phase 2a proof-of-concept trial in focal epilepsy,” said Abraham N. Ceesay, chief executive officer of Rapport. “The continued learnings from our ongoing clinical activities, including the MAD-2 and PET trials as well as our pharmaceutical development efforts, have only strengthened our confidence in the pipeline-in-a-product potential of RAP-219 as a potentially transformational treatment for focal epilepsy, peripheral neuropathic pain, and bipolar disorder.” BUSINESS UPDATES Board of Directors The Company announced the appointment of new members to its Board of Directors to help guide its next phase of growth and innovation, including Rob Perez, operating partner at General Atlantic and former chief executive officer of Cubist Pharmaceuticals; Raymond Sanchez, MD, former chief medical officer of Cerevel Therapeutics; Paul Silva, former chief accounting officer at Vertex Pharmaceuticals; and Wendy B. Young, PhD, former head of small molecule drug discovery at Genentech. With these appointments, Jeff Tong, PhD, partner at Third Rock Ventures, has resigned from the Board of Directors. “On behalf of the entire Board of Directors, I extend our deep appreciation to Jeff for his incredible leadership in helping build Rapport from its inception,” said Steve Paul MD, founder and chair of Rapport’s Board of Directors. “We are pleased to welcome additional experienced biotech leaders to our board who will provide significant scientific, medical, and business expertise to help us realize the full potential of our science for patients and shareholders alike. With the extensive experience and accomplishments of Rob, Ray, Paul, and Wendy, together with our existing board members, we are well positioned for future success.” "I want to express my sincere gratitude to Jeff for his guidance and leadership in helping us launch and shape Rapport,” said Ceesay. “It’s a true honor to welcome such exceptional industry leaders to our Board of Directors. They bring a wealth of knowledge and experience in scaling biotech organizations, and their deep understanding of neuroscience drug development, operational excellence, and innovation will be instrumental in driving the Company's growth and long-term success. Their joining the Board of Directors is a testament to the promise of the science of receptor associated proteins and RAP-219." Perez is currently an operating partner at General Atlantic. Previously, he was the chief executive officer of Cubist Pharmaceuticals where he led the launch of Cubicin® as well as multiple acquisitions and international expansion efforts leading up to its sale to Merck in 2015. Prior to that, Perez served as vice president of Biogen’s US CNS business unit, where he was responsible for commercial leadership of an $800 million business. Perez is also the founder and chairman of Life Science Cares, and the co-founder of Biopharma Leaders of Color. Sanchez is a psychiatrist with over 20 years of experience in academia, medicine, and the pharmaceutical industry, specializing in areas including CNS and analgesia. He served as the chief medical officer of Cerevel Therapeutics until its acquisition by AbbVie in 2024 and is now a senior advisor at Bain Capital Life Sciences among other roles. Sanchez has a proven track record in global asset development, leading to regulatory approvals for multiple compounds, including Abilify®. He has also played key roles in business development, capital formation, regulatory interactions, and strategic oversight of global medical portfolios across various therapeutic areas. An experienced finance and operations executive, Silva played a key role in transforming Vertex Pharmaceuticals into a high-growth company with over $6 billion in annual revenue. During his 15-year tenure, he led Vertex's accounting, tax, and treasury functions, supporting global growth and critical business development initiatives, including key international reimbursement agreements for its cystic fibrosis medicines. Silva also contributed to business development transactions that enabled Vertex to advance into genetic and cell therapies. Additionally, Silva was an inaugural Board Member of the Vertex Foundation and founding executive sponsor of Vertex “PRIDE.” Young is a biotechnology and life science executive with over 30 years of experience in drug discovery and development. As former senior vice president of small molecule drug discovery at Genentech, she oversaw the advancement of over 25 clinical candidates in oncology, immunology, neurology, and anti-infectives. Notably, she led the BTK program and co-invented fenebrutinib. Young was recently inducted into the American Chemistry Society Hall of Fame, and she has also been named “One of the Top 20 Women in Biopharma” by Endpoints News and “Most Influential Women in SF Bay Area” by the San Francisco Times. RAP-219 Lead Program RAP-219 is designed to selectively target TARPγ8, a receptor-associated protein (RAP) which is associated with the neuronal AMPAR (neuronal α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor), a clinically validated target for epilepsy. The Company is also evaluating RAP-219 as a potential treatment for peripheral neuropathic pain and bipolar disorder. The Company is conducting a second multiple ascending dose trial (MAD-2) of RAP-219, expected to be completed in Q4 2024. The trial will assess additional dosing regimens specifically to inform dosing for its Phase 2a trial for the treatment of bipolar acute mania. The initial MAD trial demonstrated that RAP-219 was generally well tolerated at target therapeutic exposures, with no serious adverse events and no drug-related treatment-emergent adverse events (TEAEs) above Grade 1.A Phase 1 human positron emission tomography (PET) trial in healthy adult volunteers is currently underway, utilizing a companion PET radiotracer to confirm brain target receptor occupancy and brain region specificity across a range of RAP-219 dosing and exposure levels.Topline results from both the MAD-2 and PET trials will be released in Q1 2025.The Company continues to progress the development of a long-acting injectable formulation of RAP-219 as the first potential anti-seizure medication (ASM) in a depot formulation, offering greater ease-of-use and potentially improved patient adherence. Focal Epilepsy Patient recruitment and screening is underway for the Company’s Phase 2a proof of concept trial in focal epilepsy. The trial is on track, and topline results are expected in mid-2025. The Phase 2a trial is enrolling adult patients with drug-resistant focal epilepsy who have an implanted responsive neurostimulation (RNS) device and are demographically similar to those expected in future registrational trials.The RNS device continually captures intracranial electroencephalography data and records the frequency of long episodes (LEs), which are often referred to as subclinical seizures and serve as a biomarker to clinical seizures. LEs function as a biomarker-based endpoint, supporting more efficient and objective efficacy results. After the initiation of new antiseizure medication, a 30% reduction of LEs has been shown to predict meaningful changes (≥ 50% reduction) in clinical seizure frequency. At the Annual Epilepsy Society meeting in December, the Company will have four poster presentations focused on RAP-219, highlighting preclinical data, SAD/MAD trial results, food effect data, and new analyses on the correlation between RNS-measured long episode and clinical seizure frequency in adult patients with refractory focal epilepsy.In September, the Company presented on the underlying mechanism of action for RAP-219 as well as the novel design of the Phase 2a proof-of-concept trial for the treatment of focal epilepsy at the International League Against Epilepsy 15th Annual European Epilepsy Congress and the 2024 Epilepsy Foundation Pipeline Conference. Peripheral Neuropathic Pain The Company was recently notified by the FDA that the IND submitted by the Company for the initiation of a Phase 2a proof-of-concept trial of RAP-219 for the treatment of diabetic peripheral neuropathic pain (DPNP) was placed on clinical hold. The FDA requested additional information and amendments specific to the protocol design. The clinical hold is specific to the IND for DPNP and has not impacted the Company’s ongoing Phase 2a trial in focal epilepsy or planned proof-of-concept trial in bipolar disorder. The Company believes in its ability to resolve the clinical hold in DPNP and will provide an update on the anticipated timing of the Phase 2a trial initiation once available. Bipolar Disorder The Company plans to initiate a Phase 2a trial in bipolar disorder patients with acute mania in 2025. Preclinical and Discovery Programs With growing confidence in RAP-219 and a commitment to disciplined capital allocation, the Company is deferring further investment in RAP-199 and focusing resources on execution of its three RAP-219 proof-of-concept clinical trials.The Company continues to advance its RAP-enabled nicotinic acetylcholine receptor (nAChR) discovery-stage programs – modulators of a6 nAChR for the potential treatment of chronic pain, and modulators of a9a10 nAChR for the potential treatment of hearing loss. THIRD QUARTER 2024 FINANCIAL RESULTS Net loss was $17.5 million for the third quarter of 2024, as compared to $8.7 million for the prior year period.Research and development expense was $15.5 million for the third quarter of 2024, as compared to $7.6 million for the prior year period. The increase in research and development expense was primarily driven by operational costs related to clinical development and costs to support the progression of the Company’s overall pipeline.General and administrative expense was $6.1 million for the third quarter of 2024, as compared to $2.0 million for the prior year period. The increase in general and administrative expense was primarily driven by costs associated with the growth of the business, in addition to costs incurred to satisfy the requirements of becoming and operating as a public company.The Company ended the third quarter with $320.7 million in cash, cash equivalents and short-term investments, compared to $336.1 million as of June 30, 2024. The decrease was primarily due to cash outflows on operating activities in the third quarter of 2024.The Company expects that current cash, cash equivalents, and short-term investments as of September 30, 2024, will enable the Company to fund its operating expenses and capital expenditure requirements through the end of 2026. About RAP-219 RAP-219 is a clinical-stage AMPAR (α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor) negative allosteric modulator (NAM) designed to achieve neuroanatomical specificity through its selective targeting of a RAP known as TARPγ8, which is associated with the neuronal AMPAR. Whereas AMPARs are distributed widely in the central nervous system (CNS), TARPγ8 is expressed only in discrete regions, including the hippocampus and cortex. Because of this restricted expression of TARPγ8 in forebrain regions, the Company believes RAP-219 has the potential to provide a differentiated clinical profile, including improved activity and tolerability along with a higher therapeutic index, potentially providing more patients with sustained therapeutic benefit without intolerable side effects, as compared to traditional neuroscience medications. Due to the role of AMPA biology in various neurological disorders and the precision approach of selective targeting of TARPγ8, the Company believes RAP-219 has significant pipeline-in-a-product potential and is currently evaluating the compound as a transformational treatment for patients with focal epilepsy, peripheral neuropathic pain, and bipolar disorder. Availability of Other Information About Rapport Therapeutics Rapport Therapeutics uses and intends to continue to use its Investor Relations website and LinkedIn (Rapport Therapeutics) 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 and LinkedIn, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcasts. The contents of the Company’s website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. About Rapport Therapeutics Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing small molecule precision medicines for patients suffering from central nervous system (CNS) disorders. The Company’s founders have made pioneering discoveries related to the function of receptor associated proteins (RAPs) in the brain. Their findings form the basis of Rapport’s RAP technology platform, which enables a differentiated approach to generate precision small molecule product candidates with the potential to overcome many limitations of conventional neurology drug discovery. Rapport’s precision neuroscience pipeline includes the Company’s lead clinical program, RAP-219, designed to achieve neuroanatomical specificity through its selective targeting of a RAP expressed in only discrete regions of the brain. The Company is currently advancing RAP-219 in clinical trials in focal epilepsy, peripheral neuropathic pain, and bipolar disorder. Additional preclinical and late-stage discovery stage programs are also underway, targeting CNS disorders including chronic pain and hearing disorders. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: the clinical development of RAP-219 for the treatment of drug-resistant focal epilepsy, peripheral neuropathic pain and bipolar disorder, including the initiation, timing, progress and results of our ongoing and planned clinical trials; the Company’s ability to resolve a clinical hold with the FDA; the potential activity and tolerability of RAP-219; the potential of Rapport’s RAP technology platform; the ongoing and planned development of RAP-199 and Rapport’s discovery-stage programs; and expectations for Rapport’s uses of capital, expenses and financial results, including its cash runway through the end of 2026. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect Rapport’s business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to the company’s research and development activities; Rapport’s ability to execute on its strategy including obtaining the requisite regulatory approvals on the expected timeline, if at all; uncertainties relating to preclinical and clinical development activities; the company’s dependence on third parties to conduct clinical trials, manufacture its product candidates and develop and commercialize its product candidates, if approved; Rapport’s ability to attract, integrate and retain key personnel; risks related to the company’s financial condition and need for substantial additional funds in order to complete development activities and commercialize a product candidate, 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 Rapport’s intellectual property protections; and risks related to the competitive landscape for Rapport’s product candidates; as well as other risks described in “Risk Factors,” in the company’s Registration Statement on Form S-1, and most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Rapport’s subsequent filings with the Securities and Exchange Commission (the SEC). Rapport expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Condensed Consolidated Balance Sheet Data(In thousands) (unaudited) September 30,2024 December 31,2023 Assets Current assets Cash and cash equivalents $39,314 $70,169 Short-term investments 281,347 77,309 Restricted cash 105 85 Prepaid expenses and other current assets 5,173 3,309 Total current assets 325,939 150,872 Property and equipment, net 3,409 1,916 Operating lease right of use asset, net 1,607 2,084 Other assets 189 551 Total assets $331,144 $155,423 Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) Current liabilities Accounts payable $1,323 $2,502 Accrued expenses and other current liabilities 5,056 5,631 Operating lease liability 720 670 Total current liabilities 7,099 8,803 Series B preferred stock tranche right liability — 4,200 Operating lease liability, net of current portion 931 1,476 Total liabilities 8,030 14,479 Commitments and contingencies Series A convertible preferred stock — 89,487 Series B convertible preferred stock — 77,091 Stockholders’ equity (deficit) Undesignated preferred stock — — Common Stock 36 4 Additional paid-in capital 426,443 19,796 Accumulated other comprehensive income 400 4 Accumulated deficit (103,765) (45,438)Total stockholders’ equity (deficit) 323,114 (25,634)Total liabilities, convertible preferred stock, and stockholders’ equity $331,144 $155,423 Condensed Consolidated Statement of Operations(In thousands, except share and per share data) (unaudited) For the three months ended September 30, 2024 2023 Operating expenses Research and development $15,543 $7,580 General and administrative 6,097 1,984 Total operating expenses 21,640 9,564 Loss from operations (21,640) (9,564)Other income (expense): Interest income 4,103 856 Change in fair value of preferred stock tranche right liability — — Total other income, net 4,103 856 Net loss before income taxes (17,537) (8,708)Provision for income taxes — 1 Net loss $(17,537) $(8,709)Net loss per share attributable to common stockholders, basic and diluted $(0.50) $(5.70)Weighted-average common shares outstanding, basic and diluted 34,855,907 1,529,216 Condensed Consolidated Statements of Cash Flows(In thousands)(unaudited) For the Three Months Ended September 30, 2024 2023 Net cash used in operating activities $(16,415) $(5,424)Net cash used in investing activities (53,041) (52)Net cash provided by (used in) financing activities (1,394) 85,360 Net increase in cash, cash equivalents and restricted cash $(70,850) $79,884 Contact Julie DiCarloHead of Communications & IRRapport Therapeuticsjdicarlo@rapportrx.com

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100 Deals associated with Fenebrutinib

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D11457	-	-	DB14785

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Multiple Sclerosis, Primary Progressive	Phase 3	Israel	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Peru	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Greece	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Bulgaria	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Poland	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	France	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Puerto Rico	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	Chile	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis, Primary Progressive	Phase 3	North Macedonia	Hoffmann-La Roche, Inc.	26 Oct 2020
Multiple Sclerosis	Preclinical	Switzerland	Genentech, Inc.	21 Aug 2020

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05119569 (FENopta-OLE, GlobeNewswire) Manual	Phase 2	Multiple sclerosis relapse	109	Fenebrutinib	evivypcnfi(xdaqmotjwn) = oimsvueohy wcicrwaclr (hdbzrtktro ) View more	Positive	04 Sep 2024
AAN2024	Phase 1	-	-	Fenebrutinib 400 mg	(rzcudstxeg) = Both doses were well tolerated, and no serious adverse events (AEs), AEs of special interest or Grade ≥2 AEs were reported moivtlxidb (zqevkrhgvq )	Positive	09 Apr 2024
				Fenebrutinib 700 mg
NCT04586010 (FENhance, Biospace) Manual	Phase 3	Multiple sclerosis relapse	-	fenebrutinib	(qhktamctjd) = rrfwkjwoyi bykkwshrdc (ejthaeuivb )	Negative	05 Dec 2023
NCT05119569 (FENopta, ECTRIMS2023) Manual	Phase 2	Multiple Sclerosis	106	Fenebrutinib 200 mg	(whgeqtdgdn) = fzazleaivd btllntyauy (xnspefqltf ) View more	Positive	17 Oct 2023
				Placebo	(whgeqtdgdn) = danlwragub btllntyauy (xnspefqltf ) View more
AAN2023	Phase 3	Multiple Sclerosis BTK	-	Tolebrutinib	mwbfdrzgeq(jgshkmywrf) = mfozdcjdeo tzdxukmypq (ntvhtzfxav, 0.40) View more	Positive	25 Apr 2023
				Evobrutinib	mwbfdrzgeq(jgshkmywrf) = ghzyzeevrl tzdxukmypq (ntvhtzfxav, 0.13) View more
ACTRIMS2022	Not Applicable	-	-	Tolebrutinib	tdcgdwrfwx(dsjryvubxq) = biqdokblzt jjvxbpnxgd (sqrsbnboul ) View more	-	16 May 2022
				Evobrutinib	tdcgdwrfwx(dsjryvubxq) = tqtcpzjjlp jjvxbpnxgd (sqrsbnboul ) View more
NCT03407482 (CTgov)	Phase 2	Systemic Lupus Erythematosus	160	GDC-0853	xukjuocomj(mnxkenlkcv) = tcrkactpyn xjepazczeu (laaytwvpdt, dbareuodtq - fzljgqtpqn) View more	-	19 Dec 2020
ECTRIMS2020	Not Applicable	Multiple Sclerosis	-	Fenebrutinib	ksvdvphyxe(jdkltbepjv) = bqozjnlivq vepnlynisn (enwdagtrzj ) View more	-	07 Dec 2020
NCT03137069 (CTgov)	Phase 2	Chronic Urticaria	134	Placebo (Cohort 1: Placebo)	jahfpcpqgq(mgwsosrujb) = cjxxchzmtk yfnrqkzugf (flxltzzcfq, wfgdhtyxos - oxtxkrplgr) View more	-	29 Sep 2020
				GDC-0853 (Cohort 1: GDC-0853 200mg BID)	jahfpcpqgq(mgwsosrujb) = onijiuldwl yfnrqkzugf (flxltzzcfq, srtoaqoitf - elzqgqqryx) View more
NCT03693625 (CTgov)	Phase 2	Chronic Urticaria	31	GDC-0853 (Parent Study: GDC-0853)	vvfzdswytu(uhvsfkbdwk) = yjzdtdxasu jffgqiwbsx (cimrewxmvp, hkfapebuva - agvdpccvct) View more	-	25 Sep 2020
				GDC-0853 (Parent Study: Placebo)	vvfzdswytu(uhvsfkbdwk) = leeoccgcqd jffgqiwbsx (cimrewxmvp, hcscgxbtdh - ocdnsqgwkr) View more

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