Biohaven Q2

 

• Cash, cash equivalents, marketable securities and restricted cash totaled approximately $440 million on June 30, 2024
• Biohaven's Molecular Degrader of Extracellular Proteins (MoDE™) platform advancing multiple new targets and reported positive interim data from its lead investigational drug in an ongoing Phase 1 study of BHV-1300:
  • The Company reported dose-dependent and rapid IgG reductions in its ongoing Phase 1 trial with its lead investigational degrader BHV-1300
  • No serious adverse events (SAEs) reported with BHV-1300 to date; most adverse events (AEs) were mild, deemed unrelated to study drug and resolved spontaneously
  • Phase 1 study also completed an assessment of an optimized subcutaneous (SC) formulation of BHV-1300 that demonstrated approximately a 44% higher than expected exposure compared to the dose-equivalent intravenous formulation previously studied; this new human data further confirms feasibility of convenient self-administered SC auto-injector and the SC formulation was not associated with injection site reactions
  • Degrader platform expected to deliver 3 INDs for new MoDE programs before year-end in addition to continued data from SAD/MAD with BHV-1300
  • Biohaven's beta-1 adrenergic receptor (β1AR) autoantibody targeting MoDE, BHV-1600, granted INTERACT meeting with FDA in 2H 2024 regarding development program for dilated cardiomyopathy
• Advancing novel ion channel program targeting Kv7 activation and TRPM3 antagonism across multiple neurological, pain and neuropsychiatric disease indications:
  • 5 Phase 2/3 trials with BHV-7000 underway in epilepsy and mood disorders
  • Released positive Phase 1 data with TRPM3 antagonist BHV-2100 showing drug concentrations above EC90 target and well-tolerated profile across all doses in SAD/MAD study; advancing Phase 2 study in acute migraine and proof-of-concept (POC) study in pain in 2H 2024
• Taldefgrobep alfa, a myostatin-inhibitor, progressing on track with Phase 3 topline data in spinal muscular atrophy (SMA) and Phase 2 trial initiation in obesity expected in 2H 2024
  • Taldefgrobep alfa has demonstrated direct effects on reducing adipose tissue (including lipid storage and mitochondrial content) independent of increases in muscle mass
  • In a MAD study, conducted in healthy adults, taldefgrobep alfa (45 mg SC QW) produced significant reductions in total body fat while increasing total body lean mass
  • Preclinical data released at the 2024 American Diabetes Association conference demonstrated that taldefgrobep alfa, as a monotherapy or in combination with a GLP-1 agonist, demonstrated significant reductions in fat and total body weight. Taldefgrobep alfa-treated animals showed significant increases in lean muscle, despite co-administration with a GLP-1 receptor agonist
• Tyrosine Kinase 2/Janus Kinase 1 (TYK2/JAK1) selective inhibitor, BHV-8000, completed Phase 1 and confirmed biomarker target engagement with reductions in inflammatory markers and demonstrated central nervous system penetration with confirmed cerebrospinal fluid (CSF) target exposures in healthy subjects
  • Advancing registrational programs for Parkinson's disease and prevention of Amyloid-Related Imaging Abnormalities (ARIA) following interactions with the FDA
• Expect interim data analysis from second ongoing Phase 3 OCD trial with troriluzole in 2H 2024; topline data from first Phase 3 OCD trial expected in 1H 2025
• SCA interactions with troriluzole filing in Europe ongoing and constructive interactions in the US with the FDA
  • New real-world evidence (RWE) protocol, incorporating feedback from the FDA, assessing 3-years of treatment with troriluzole expected to deliver topline results in 2H 2024
• Biohaven antibody drug conjugate (ADC) portfolio positioned to deliver differentiated profiles and address unmet needs in oncology:
• BHV-1510 currently dosing cancer patients in Phase1/2 study and now advancing towards combination with Libtayo® by 4Q 2024
• Portfolio of multiple advanced nonclinical BHVN ADCs demonstrate improved plasma stability and in vitro/in vivo differentiation

https://www.prnewswire.com/news-releases/biohaven-reports-second-quarter-2024-financial-results-and-recent-business-developments-302218343.html