On August 8, 2024 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the second quarter ended June 30, 2024, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, AUG 8, 2024, View Source [SID1234645645]).
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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "Last quarter, we showcased an array of exciting updates across our pipeline spanning immunology, neuroscience, obesity, and oncology at our annual R&D Day. First, it has been incredible working with Yale Professor David Spiegel and my colleagues at Biohaven to drive the advancement of groundbreaking degrader science represented by BHV-1300 using our Molecular Degrader of Extracellular Protein (MoDE) technology — we consider our initial Phase 1 data to be paradigm-shifting and we are accelerating our entire platform based upon these early results. BHV-1300 has been well-tolerated with no significant adverse effects and no clinically significant lab abnormalities or ECG changes observed to date and emerging subcutaneous data from our Phase 1 study highlights the ease of use of this technology. The platform represents a brand-new technology that has potential to treat diseases like Type 1 diabetes, autoimmune dilated cardiomyopathy, IgA nephropathy, and many others. Our differentiation continues to focus on convenient SC patient self-administration, rapid onset of action, potential for deeper IgG reduction and concurrent use with Fc containing standard of care biologics. We are excited that we remain on timelines with multiple INDs from our MoDE platform for later this year, including drug candidates that target autoantibodies against β-1AR for the potential treatment of dilated cardiomyopathy, galactose deficient IgA for IgA nephropathy and a further optimized IgG degrader for use in rare diseases."
Dr. Coric continued, "While our degrader program continues to garner significant focus, our broader pipeline is increasingly derisked with several potentially paradigm-shifting treatments for conditions including epilepsy, mood disorders, pain, immunology, rare diseases, obesity and neurodegenerative disorders. Starting with our ion channel platform; five studies with Kv7 activator BHV-7000 (three in epilepsy and two in major depressive disorder and bipolar) are underway with brisk patient enrollment. In addition, our TRPM3 antagonist BHV-2100 has completed its initial Phase 1 SAD/MAD study, attaining an exceptional PK/PD profile for the treatment of migraine and other pain disorders. All doses being advanced have reached the targeted EC90 for this molecule with excellent preliminary safety. We are excited about being back in the migraine space with this novel target and will initiate a Phase 2 study in migraine before year end, as well as initiate a POC study as a non-opiate pain treatment. With BHV-8000, our oral and brain penetrant TYK2/JAK1 inhibitor, we have aligned with the FDA on two novel study designs: one for the prevention of ARIA associated with amyloid lowering agents in Alzheimer’s disease and another with the goal of slowing disease progression in early Parkinson’s disease. The Phase 1 profile of BHV-8000 is invigorating our translational team as we have data confirming both CNS penetration and biomarker engagement of inflammatory targets in humans. Regarding our anti-myostatin taldefgrobep alfa, we shared new preclinical data demonstrating improved, durable weight reduction and lean mass preservation in combo with GLP-1 semaglutide, which was particularly noteworthy, given the muscle wasting limitations that have plagued the GLP-1 class. We also showed that taldefgrobep has direct effects on modulating fat in addition to increasing muscle mass. Our oncology platform has also emerged into the clinic with our lead Trop-2 ADC, BHV-1510, enrolling cancer patients and our efforts advancing a portfolio of several Biohaven ADCs. We are excited to get preliminary safety and efficacy data from cancer patients suffering with select advanced or metastatic epithelial tumors in the Phase 1/2 study of BHV-1510 and will be evaluating the combination of BHV-1510 with Regeneron’s anti-PD1 Libtayo — combination dosing with BHV-1510 and Libtayo is anticipated to begin in 2H 2024. Finally, with our glutamate platform represented by troriluzole, topline data from an interim analysis of the second OCD trial is expected in 2H 2024 and our first OCD trial is expected to read out in 1H 2025. Separately, we continue to have constructive dialogue with the FDA regarding our SCA development program and our European Medicines Agency application for SCA3 remains under review. We now expect topline data from a new RWE protocol assessing the efficacy of troriluzole in SCA patients treated for up to 3 years— this includes new patient data that has not previously been available. I never cease to be amazed by the power of our small but mighty Biohaven R&D team that has innovated across each of these important medical areas. We are driven to fulfill a promise to patients and our investors that we will diligently evaluate and advance potentially transformative therapies to those suffering from disease."
Second Quarter 2024 and Recent Business Highlights
Additional data supports the paradigm-shifting potential of the MoDE Platform for the treatment of immunological and inflammatory disorders – In May 2024, the Company disclosed positive new data from its ongoing Phase 1 SAD study in healthy subjects with BHV-1300, a first-in-human IgG degrader from its innovative MoDE platform. Results from the initial dose cohorts in the SAD study confirmed that BHV-1300 rapidly and selectively lowers IgG in a dose-dependent manner. Emerging Phase 1 data also showed that our novel SC formulation of BHV-1300 delivered exposures higher than the intravenous formulation, enabling the profile of a convenient patient administered auto-injector to attain targeted reduction of IgG. The Company expects to provide an update from our SAD and MAD studies of BHV-1300 in the second half of 2024 and is on schedule to file multiple new MoDE INDs this year. FDA granted INTERACT meeting request for BHV-1600, β1AR autoantibody MoDE, development program.
Ion Channel Platform: Kv7 activator and TRPM3 antagonist candidates represent transformational targets in neurology and neuropsychiatry – The Company is enrolling participants in five ongoing pivotal clinical trials with Kv7 activator, BHV-7000, targeting focal epilepsy, generalized epilepsy, bipolar disorder and major depressive disorder; separately, Phase 2 and POC studies are planned in migraine and pain, respectively, with the TRPM3 antagonist, BHV-2100, in 2H 2024.
Committed to improving muscle health with Myostatin Platform – In May 2024, the Company presented new preclinical data showing that administration of taldefgrobep alfa directly reduced the increased adipose fat storage caused by myostatin; the Company also presented new preclinical data from a diet-induced obesity mouse model showing treatment with taldefgrobep alfa together with a GLP-1 agonist produced greater reductions in body weight and fat mass, and a larger increase in lean muscle mass, compared to treatment with GLP-1 alone. The Company plans to initiate a Phase 2 study in obesity in 2H2024. Separately, the Company continues to expect Phase 3 study topline results in SMA in 2H 2024.
Selectively targeting the immune system to treat neurodegenerative diseases with neuroinflammation platform – In May 2024, the Company reported positive results from the Phase 1 SAD and MAD study with our brain-penetrant TYK2/JAK1 inhibitor, BHV-8000 in healthy subjects, including demonstration of CSF target exposures and biomarker engagement along with a well-tolerated safety profile. The Company also announced key regulatory updates, including the successful completion of two FDA meetings with favorable feedback enabling registrational programs for Parkinson’s disease and for the prevention of ARIA, a novel indication.
Oncology Platform: Antibody Drug Conjugate portfolio: positioned to achieve enhanced stability and improved efficacy – In May 2024, the Company announced that its novel Trop-2 ADC, BHV-1510, entered into the clinic for patients with advanced or metastatic epithelial tumors; the first patient was dosed in a Phase 1/2 clinical trial as monotherapy. Biohaven also entered into a clinical supply agreement with Regeneron to study the combination of BHV-1510 with Regeneron’s anti-PD1 Libtayo (cemiplimab-rwlc) in the clinical study.
Expected Upcoming Milestones:
We believe Biohaven is well positioned to achieve significant milestones in 2024 and 2025 across numerous programs:
Selective Kv7 Activator:
Continue 5 ongoing Phase 2/3 trials with BHV-7000 in focal epilepsy, idiopathic generalized epilepsy, MDD, and bipolar disorder
Troriluzole:
Topline results from a new RWE protocol of 3-year data from troriluzole treated patients in SCA in 2H 2024
Two Phase 3 trials with troriluzole in OCD: conduct interim analysis of the second Phase 3 OCD trial in 2H 2024 and report topline data from first Phase 3 OCD trial in 1H 2025
Taldefgrobep alfa:
Initiate Phase 2 trial with taldefgrobep in obesity in 2H 2024
Report topline data from Phase 3 trial with taldefgrobep in SMA in 2H 2024
First-in-class TRPM3 Antagonist:
Initiate Phase 2 trial with BHV-2100 in acute migraine in 2H 2024
Initiate POC study with BHV-2100 for pain in 2H 2024
TYK2/JAK1 Inhibitor:
Complete SAD/MAD studies with BHV-8000 and advance to Phase 2 in the coming months
MoDE Platform
Submit a total of 4 INDs in 2024
Continue to advance Phase 1 SAD and MAD studies with BHV-1300, with a further study update in 2H 2024
INTERACT meeting with FDA regarding BHV-1600, β1AR autoantibody degrader, in 2H 2024
Next Generation ADC Platform:
Advance Phase 1 Trop-2 directed program BHV-1510 into a Phase 1 study in multiple tumor types
Capital Position:
Cash, cash equivalents, marketable securities and restricted cash totaled approximately $440 million on June 30, 2024.
Second Quarter 2024 Financial Highlights:
Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $314.8 million for the three months ended June 30, 2024, compared to $79.5 million for the three months ended June 30, 2023. The increase was primarily due to a one-time, non-cash expense of $171.9 million paid to Knopp Biosciences, LLC (Knopp) for a milestone and royalty buyback related to our BHV-7000 and broader Kv7 platform that was recognized during the three months ended June 30, 2024 (the buyback reduced our potential future milestone payments by $867.5 million, and replaced the scaled high single digit to low teens royalty payment obligations with a flat royalty payment in the mid-single digits for the Kv7 programs). The increase in R&D expenses was also due to advancing our 6 clinical platforms including 5 Phase 3 starts for BHV-7000, follow-on Kv7 assets, preclinical research programs, and increases in direct program spend for additional and advancing multiple clinical development programs. Non-cash share-based compensation expense was $7.1 million for the three months ended June 30, 2024, an increase of $4.6 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the second quarter of 2024, primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024.
General and Administrative (G&A) Expenses: General and administrative expenses were $19.0 million for the three months ended June 30, 2024, compared to $14.5 million for the three months ended June 30, 2023. The increase of $4.4 million was primarily due to increased non-cash share-based compensation expense, which was $5.2 million for the three months ended June 30, 2024, an increase of $2.9 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the second quarter of 2024 primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024.
Other Income, Net: Other income, net was a net other income of $14.2 million for the three months ended June 30, 2024, compared to a net other income of $5.8 million for the three months ended June 30, 2023. The increase of $8.3 million was primarily due to a $9.2 million gain recorded upon the settlement of our forward contract liability for share consideration issued under the amendment entered into with Knopp in May 2024, and increased investment income, partially offset by a decrease in other income recognized during the three months ended June 30, 2024, as compared to the same period in 2023 related to the Transition Services Agreement entered into with Biohaven Pharmaceutical Holding Company Ltd. (the Former Parent).
Net Loss: Biohaven reported a net loss for the three months ended June 30, 2024 of $319.8 million, or $3.64 per share, compared to $90.3 million, or $1.32 per share, for the same period in 2023. Non-GAAP adjusted net loss for the three months ended June 30, 2024 was $307.4 million, or $3.50 per share, compared to $85.7 million, or $1.25 per share for the same period in 2023. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.