Biohaven .(US:BHVN)2024-08-08 20:17Key Business Highlights & Pipeline Update Biohaven reported significant progress across its diverse pipeline in Q2 2024, advancing multiple candidates and achieving positive clinical and preclinical data MoDE™ Platform (BHV-1300, BHV-1600) The MoDE™ platform's lead candidate BHV-1300 showed positive Phase 1 data with a promising subcutaneous formulation, and the platform is expanding with new INDs planned - Reported positive interim Phase 1 data for BHV-1300, demonstrating dose-dependent and rapid IgG reductions with no serious adverse events1 - An optimized subcutaneous formulation of BHV-1300 showed approximately 44% higher exposure than the IV dose, supporting self-administration14 - The company expects to submit 3 new INDs for MoDE programs by year-end and has an FDA INTERACT meeting for BHV-1600 in 2H 202416 Ion Channel Platform (BHV-7000, BHV-2100) The ion channel platform is rapidly advancing with five Phase 2/3 trials for BHV-7000 and planned Phase 2 studies for BHV-2100 following positive Phase 1 data - Five Phase 2/3 trials for Kv7 activator BHV-7000 are underway, targeting epilepsy and mood disorders14 - TRPM3 antagonist BHV-2100 showed positive Phase 1 data, achieving target drug concentrations and a well-tolerated profile, with Phase 2 studies planned for 2H 202414 Myostatin Platform (Taldefgrobep alfa) Taldefgrobep alfa is advancing with Phase 3 data in SMA and a Phase 2 obesity trial expected in 2H 2024, supported by preclinical data showing dual fat reduction and muscle increase - Topline Phase 3 data for taldefgrobep alfa in SMA and initiation of a Phase 2 trial in obesity are both expected in 2H 202415 - Preclinical data showed taldefgrobep alfa directly reduces adipose tissue and, combined with a GLP-1 agonist, significantly reduced fat and total body weight while increasing lean muscle14 Neuroinflammation Platform (BHV-8000) The selective TYK2/JAK1 inhibitor BHV-8000 completed Phase 1, confirming CNS penetration and biomarker engagement, with registrational programs advancing for Parkinson's disease and ARIA prevention - BHV-8000 completed Phase 1, confirming biomarker target engagement and CNS penetration with CSF target exposures14 - Registrational programs for Parkinson's disease and ARIA prevention are advancing following favorable FDA feedback14 Glutamate Platform (Troriluzole) The glutamate modulator troriluzole is advancing in OCD and SCA, with interim Phase 3 OCD data and RWE results for SCA expected in 2H 2024 - Interim data analysis from the second Phase 3 OCD trial is expected in 2H 2024, with topline data from the first trial in 1H 202515 - A new real-world evidence protocol for troriluzole in SCA is expected to deliver topline results in 2H 2024, incorporating FDA feedback15 Oncology Platform (ADC - BHV-1510) Biohaven's oncology platform is now clinical-stage, with lead ADC BHV-1510 dosing patients in a Phase 1/2 study and combination dosing with Libtayo® expected by Q4 2024 - The lead ADC, BHV-1510 (Trop-2 directed), is currently dosing cancer patients in a Phase 1/2 study14 - A combination study of BHV-1510 with Regeneron's Libtayo® is expected to begin by Q4 202414 Expected Upcoming Milestones Biohaven anticipates significant pipeline milestones in 2H 2024 and 2025, including topline data, interim analyses, and multiple Phase 2 trial initiations - Troriluzole: - Topline results from a new RWE protocol in SCA (2H 2024)5 - Interim analysis of the second Phase 3 OCD trial (2H 2024)5 - Topline data from the first Phase 3 OCD trial (1H 2025)5 - Taldefgrobep alfa: - Initiate Phase 2 trial in obesity (2H 2024)5 - Report topline data from Phase 3 trial in SMA (2H 2024)5 - BHV-2100 (TRPM3 Antagonist): - Initiate Phase 2 trial in acute migraine (2H 2024)5 - Initiate POC study for pain (2H 2024)5 - MoDE™ Platform: - Submit a total of 4 INDs in 20246 - Provide a further update from Phase 1 SAD/MAD studies with BHV-1300 (2H 2024)6 Second Quarter 2024 Financial Results Biohaven reported a net loss of $319.8 million in Q2 2024, primarily due to increased R&D expenses including a $171.9 million non-cash charge, while maintaining a strong capital position of approximately $440 million Capital Position As of June 30, 2024, Biohaven maintained a strong financial position with approximately $440 million in cash and equivalents - Cash, cash equivalents, marketable securities, and restricted cash totaled approximately $440 million on June 30, 202416 R&D Expenses R&D expenses significantly increased to $314.8 million in Q2 2024, primarily due to a $171.9 million non-cash milestone and royalty buyback and increased clinical program spending R&D Expenses (Millions USD) | Expense Category | Q2 2024 | Q2 2023 | | :--- | :--- | :--- | | Research and Development (R&D) | $314.8 | $79.5 | - The increase was primarily due to a one-time, non-cash expense of $171.9 million for a milestone and royalty buyback related to the BHV-7000 and Kv7 platform7 G&A Expenses General and Administrative expenses increased to $19.0 million in Q2 2024, primarily driven by higher non-cash share-based compensation expense G&A Expenses (Millions USD) | Expense Category | Q2 2024 | Q2 2023 | | :--- | :--- | :--- | | General and Administrative (G&A) | $19.0 | $14.5 | Net Loss & EPS Biohaven reported a GAAP net loss of $319.8 million ($3.64 per share) for Q2 2024, with a Non-GAAP adjusted net loss of $307.4 million ($3.50 per share) Net Loss & EPS (Millions USD, except per share data) | Metric | Q2 2024 | Q2 2023 | | :--- | :--- | :--- | | GAAP Net Loss | $319.8 | $90.3 | | GAAP Net Loss Per Share | $3.64 | $1.32 | | Non-GAAP Adjusted Net Loss | $307.4 | $85.7 | | Non-GAAP Adj. Net Loss Per Share | $3.50 | $1.25 | Non-GAAP Financial Measures Non-GAAP financial measures are provided to offer a clearer view of ongoing operating performance by excluding non-cash share-based compensation and derivative fair value changes - Non-GAAP financial measures exclude non-cash share-based compensation and changes in derivative fair value for a clearer understanding of ongoing operating performance11 Consolidated Financial Statements (Unaudited) The unaudited financial statements for Q2 2024 show a significant increase in operating expenses and net loss, alongside an increase in total assets and liabilities Consolidated Statements of Operations For Q2 2024, total operating expenses were $333.8 million, resulting in a net loss of $319.8 million, significantly higher than the prior-year period Statement of Operations Summary (Three Months Ended June 30, In Thousands USD) | (In thousands) | 2024 | 2023 | | :--- | :--- | :--- | | Research and development | $314,819 | $79,490 | | General and administrative | $18,953 | $14,521 | | Total operating expenses | $333,772 | $94,011 | | Loss from operations | ($333,772) | ($94,011) | | Other income, net | $14,178 | $5,842 | | Net loss | ($319,771) | ($90,346) | Consolidated Balance Sheets As of June 30, 2024, total assets increased to $582.6 million, total liabilities rose to $198.9 million due to new derivative liabilities, and total shareholders' equity was $383.7 million Balance Sheet Summary (In Thousands USD) | (In thousands) | June 30, 2024 | Dec 31, 2023 | | :--- | :--- | :--- | | Total current assets | $511,268 | $442,446 | | Total assets | $582,641 | $513,212 | | Total current liabilities | $156,207 | $55,423 | | Total liabilities | $198,901 | $85,237 | | Total shareholders' equity | $383,740 | $427,975 | Reconciliation of GAAP to Non-GAAP Financial Measures For Q2 2024, the GAAP net loss of $319.8 million was adjusted to a Non-GAAP net loss of $307.4 million by excluding non-cash compensation and derivative fair value changes GAAP to Non-GAAP Reconciliation (Three Months Ended June 30, 2024, In Thousands USD) | (In thousands) | Amount | | :--- | :--- | | GAAP net loss | ($319,771) | | Add: non-cash share-based compensation | $12,232 | | Add: loss from change in fair value of derivatives | $110 | | Non-GAAP adjusted net loss | ($307,429) |