Full Press Release Details
NEW HAVEN, Conn. , Jan. 13, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN ) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative and transformative therapies to treat individuals with rare and common diseases, today highlighted broad portfolio progress at the 43 rd Annual J.P. Morgan Healthcare Conference, including positive Phase 1 data for BHV-1400, its highly differentiated investigational therapeutic for IgA nephropathy. BHV-1400 is a second generation TRAP™ degrader from its proprietary MoDE™ platform. A copy of the slide presentation is available on the Events and Presentations section of the Biohaven website.
Biohaven 2025 Portfolio Review and Anticipated Milestones
Biohaven is positioned to achieve significant milestones in 2025 across a broad spectrum of early- and late-stage programs targeting indications with high unmet need:
Molecular Degrader of Extracellular Proteins (MoDE) Platform: Biohaven's novel immune-modulating extracellular degrader platform harnesses selectivity, rapidity, and patient-friendly self-administration to remove disease-causing proteins from the body to potentially treat a wide range of diseases. Biohaven introduced next generation TRAP degraders, which are highly selective, each targeting a specific disease-causing protein for proteolysis. Four INDs for MoDE and next generation TRAP degrader molecules (targeting IgG1, IgG2, IgG4 , Gd-IgA1, and β1AR autoantibodies) have been accepted by the FDA in 2024 with several additional investigational agents in development. Three assets have been dosed in Phase 1 trials with the fourth anticipated to be dosed in the first half of 2025.
BHV-1400 and BHV-1600, currently in Phase 1 clinical trials, represent the next generation TRAP degraders focused on selectively clearing very specific pathogenic antibodies, while sparing healthy immunoglobulin to preserve immune function. TRAP molecules commence a new age of immune-modulating treatments, targeted removal of disease-causing proteins for removal while sparing the normal function of the healthy immune system. Data from the first, and lowest, dose cohort of BHV-1400 demonstrated clear differentiation from competitors in the IgA nephropathy space, with rapid lowering of Gd-IgA1 within four hours and preservation of host immunoglobulins including IgG, IgA, IgE, and IgM. IgA nephropathy is a rare chronic kidney disease affecting young and middle-aged adults that is caused by Gd-IgA1 and leads to kidney failure in up to 40% of patients within 10-20 years.
Taken in total, the selectivity of MoDE and TRAP degraders demonstrated to date refine immune-modulating treatment representing a clear next generation of drug development technology in immunoglobulin and extracellular protein lowering. Existing mechanisms, both pharmaceutical and device (plasmapheresis), broadly reduce immunoglobulins subclasses and/or isotypes, leading to inefficient dosing, safety risks, necessity of procedures, delays in therapy, and potential efficacy impacts. MoDE and TRAP's new paradigm builds off the prior successes of immune modulation, while also providing a novel technology to fine tune therapies for immune-mediated diseases. As described below, the implications and applications of this selective targeting could be multi-organ, multi-disease.
Upcoming milestones in the degrader program include:
BHV-2100: First-in-clinic, oral, selective TRPM3 antagonist that offers a novel, non-addictive treatment for migraine and neuropathic pain. Based on favorable pharmacokinetic and safety data from the Phase 1 studies in healthy subjects, a Phase 1b laser-evoked hyperalgesia trial was performed and a proof-of-concept in the acute treatment of migraine is ongoing. Preliminary data from the laser-evoked hyperalgesia study demonstrated that BHV-2100 reduced laser heat-induced pain and brain evoked potentials in healthy volunteers. This exciting result is a culmination of years of laboratory research and represents a powerful entree into the field. It provides the first indication of potential clinical efficacy in pain with the novel TRPM3 mechanism recapitulating antinociceptive preclinical efficacy across a spectrum of pain models. Data from the laser-evoked potential study and proof-of-concept migraine study expected in 1H 2025.
BHV-7000: Selective activator of Kv7.2/7.3 potassium channels, a breakthrough target in neurology and neuropsychiatry with blockbuster potential. Kv7 activation is a clinically validated target for treating mood disorders and epilepsy. Registrational studies ongoing in bipolar disorder, major depressive disorder, focal epilepsy, and generalized epilepsy.
Upcoming milestones in the BHV-7000 program include:
Troriluzole: Troriluzole is a novel glutamate modulator currently in Phase 3 development for Spinocerebellar ataxia (SCA) and obsessive-compulsive disorder (OCD). A new drug application (NDA) was submitted to US FDA for troriluzole in all SCA genotypes, following completion of pre-NDA meeting in 4Q 2024. Troriluzole has Orphan Drug and Fast-Track designations and qualifies for potential Priority Review. EU marketing authorization application also under review for troriluzole in all SCA genotypes. There are no FDA-approved treatments for SCA. Additionally, two Phase 3 trials with troriluzole in OCD are ongoing.
Upcoming milestones in the troriluzole program include:
Taldefgrobep alfa: Taldefgrobep is a novel myostatin inhibitor that is optimized to block both myostatin and activin A signaling, two key regulators of muscle and fat metabolism. Biohaven is studying taldefgrobep in a global Phase 3 expansion study in Spinal Muscular Atrophy (SMA), as an adjunctive therapy to enhance muscle mass and function in patients treated with standard-of-care therapies. Analyses of prespecified subgroups by race and ethnicity demonstrated that the largest study population (87% Caucasian; n=180) showed clinically meaningful improvements on the MFM-32 at all timepoints, including Week 48, compared to the corresponding placebo+SOC group (p < 0.05), though the overall primary endpoint was not met. Robust target engagement (myostatin reduction) and beneficial impacts on body composition parameters (fat mass, lean muscle mass, and bone density) were noted, offering a potential paradigm shift in the treatment of obesity with opportunity to improve quality of weight loss; lower total body weight by specifically reducing fat mass while also preserving or increasing lean muscle mass.
Upcoming milestones in the taldefgrobep program include:
BHV-8000: BHV-8000 is a highly selective, oral, brain-penetrant, selective TYK2/JAK1 inhibitor with broad potential for neurodegenerative and neuroinflammatory disorders. In the Phase 1 SAD/MAD study in healthy participants, BHV-8000 was generally safe and well-tolerated while producing significant reductions in inflammatory biomarkers relative to placebo. Target indications for BHV-8000 include Parkinson's disease, Alzheimer's disease, prevention of amyloid-related imaging abnormalities (ARIA), and multiple sclerosis (MS). In 2024, Biohaven completed interactions with FDA enabling registrational programs for Parkinson's disease and the prevention of ARIA.
Upcoming milestones in the BHV-8000 program include:
Oncology antibody drug conjugate (ADC) portfolio:
BHV-1510 (Trop2 ADC): Preliminary data from the initial Phase 1 study dosing cohorts of BHV-1510 have demonstrated promising clinical activity, including tumor shrinkage, with a tolerable safety profile of the novel topoisomerase 1 (TopoIx) payload. The Phase 1/2 study of BHV-1510 is progressing with robust enrollment in dose escalation and optimization, both as monotherapy and in combination with the anti-PD1 monoclonal antibody Libtayo ® (cemiplimab-rwlc) through a clinical supply agreement with Regeneron.
Based on these encouraging early results, Biohaven has entered into an expanded collaboration agreement with GeneQuantum, which provides broad target exclusivity for up to 18 ADC targets incorporating the novel topoisomerase 1 inhibitor (TopoIx) payload.
Biohaven has incorporated the TopoIx payload into its next clinical-stage investigational agent, BHV-1530. BHV-1530 is an FGFR3-directed ADC with potential indications in cancers driven by FGFR3 alterations and/or upregulated FGFR3 protein expression, including urothelial cancers and other solid tumors. While FGFR3 has been clinically validated as a target in oncology, there are no other FGFR3 ADCs currently in clinical development. Biohaven retains global rights for BHV-1530 under the agreement via an exclusive license with GeneQuantum and Aimed Bio. The US IND has been opened, and a first-in-human study for solid tumors is planned for 1H 2025.
In addition, Biohaven today announced a multi-target collaboration with Merus, an oncology-focused biotechnology company developing innovative, multi-specific (Biclonics ® and Triclonics ® ) antibodies. This collaboration will co-develop three programs encompassing highly differentiated next generation dual-targeted bispecific ADCs leveraging Biohaven's proprietary conjugation and payload technologies along with Merus' leading Biclonics technology platform.
Together, the announced milestones and collaborations represent a significant expansion of Biohaven's ADC portfolio, positioning the Company with potential to deliver highly differentiated therapeutics and address significant unmet needs in Oncology.
Nushmia Khokhar , M.D., Biohaven Chief Medical Officer of Oncology, commented, "These are exciting times for Biohaven's oncology pipeline as we are well-positioned to introduce differentiated, next generation ADCs to the clinic. The early Phase 1 data with BHV-1510 is promising, showing not only signs of clinical activity but also minimal toxicities related to the free payload. This affirms the advantages of our conjugation technology, which provides high ADC stability. The distinct profile of the novel TopoIx payload, and its potential to synergize with checkpoint inhibitor therapy, could significantly benefit patients across various cancer types. Furthermore, our collaboration with Merus and the expanded partnership with GeneQuantum—utilizing the TopoIx payload for multiple targets—demonstrate the potential of Biohaven's upcoming innovative ADCs. This includes dual-targeting ADCs, which are exciting for their potential to address challenges like tumor heterogeneity and delivery of stable ADCs with an improved therapeutic index."
Upcoming milestones in the oncology program include:
About Biohaven Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and nonclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA (spinocerebellar ataxia); myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer. For more information, visit www.biohaven.com .
Forward-looking Statements This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "continue", "plan", "will", "believe", "may", "expect", "anticipate" and similar expressions, is intended to identify forward-looking statements. Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties. Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable US regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates. Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
MoDE is a trademark of Biohaven Therapeutics Ltd. TRAP is a trademark of Biohaven Therapeutics Ltd. Biclonics and Triclonics are registered trademarks of Merus NV. Libtayo ® is a registered trademark of Regeneron.
Investor Contact: Jennifer Porcelli Vice President, Investor Relations [email protected] +1 (201) 248-0741
Media Contact: Mike Beyer Sam Brown Inc. [email protected] +1 (312) 961-2502
SOURCE Biohaven Ltd.