On May 9, 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 first quarter ended March 31, 2024, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, MAY 9, 2024, View Source [SID1234643097]).
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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "We are excited about the clinical progress we are making across our entire portfolio and in particular, the recent initiation of the first-in-human study of BHV-1300 using our Molecular Degrader of Extracellular Protein (MoDE) technology. We were pleased to report preliminary safety and target engagement data showing degradation of IgG from the initial two low-dose cohorts of the ongoing BHV-1300 study. Thus far, BHV-1300 has also been well-tolerated with no significant adverse effects and no clinically significant lab abnormalities or ECG changes observed to date. We remain enthusiastic about the emerging profile of BHV-1300 and have multiple IND-enabling activities planned for several additional MoDEs directed at autoantibody-mediated disease targets over the next couple of years. MoDEs represent a transformative technology to modulate the immune system and specifically degrade extracellular targets known to cause disease. INDs from our MoDE platform planned for later this year include drug candidates that target autoantibodies against β-1AR for the potential treatment of dilated cardiomyopathy and galactose deficient IgA for IgA nephropathy."
Dr. Coric continued, "The Biohaven R&D team continues to make impressive progress across our broader pipeline, with enrollment having begun in our BHV-7000 epilepsy program. In addition, dosing is now complete in Phase 1 SAD and MAD cohorts of BHV-8000, our TYK2/JAK1 inhibitor, paving the way for multiple clinical trial initiations in 2024, such as Parkinson’s disease, Alzheimer’s disease and prevention of Amyloid-Related Imaging Abnormalities (ARIA) associated with amyloid lowering agents. Additionally, Phase 3 trials are underway in OCD (obsessive-compulsive disorder), and in SMA, with data anticipated in both studies later this year. Further, multiple Phase 2 and Phase 2/3 studies initiated in Q2 in MDD, Bipolar Disorder and Generalized Epilepsy, and Phase 2 studies are projected to start in the second half of the year in migraine and obesity. And finally, important updates are expected with our burgeoning antibody drug conjugate (ADC) platform, with our recent initiation of enrollment in the TROP2 Phase 1 study, multiple INDs and 5–7 new ADC targets projected in the next two years. We excitedly await a steady cascade of anticipated upcoming milestones and were pleased to complete a public offering to further advance our development plans and research efforts. We look forward to unveiling further updates across our clinical programs and future development plans at our annual R&D Day at the Yale Innovation Summit on May 29, 2024 in New Haven, CT."
First Quarter 2024 and Recent Business Highlights
Reported preliminary safety and IgG lowering data from ongoing SAD study with BHV-1300 – In April 2024, the Company provided preliminary safety and IgG lowering data from its ongoing SAD study of BHV-1300. In the study, 16 subjects completed two dosing cohorts to date. All cohorts have proceeded as initially planned without any cohort expansion or interruption.
To date, BHV-1300 has been safe and well-tolerated with no serious adverse events (SAEs) or moderate or severe adverse events (Aes) observed. Only mild Aes have been observed, which were deemed not to be related to BHV-1300 with most resolving spontaneously. No clinically significant laboratory abnormalities (including liver function tests and albumin) or electrocardiogram (ECG) changes have been observed to date.
Preliminary IgG lowering data is consistent with modeling based on non-clinical experience, with dose- and time-dependent IgG lowering observed even in initial low dose cohorts. Reductions were greater for IgG1, IgG2 and IgG4 subclasses compared to IgG3; BHV-1300 was designed to spare IgG3.
Based on initial findings, the Company plans to accelerate development across additional autoantibody-mediated targets.
Phase 2/3 program with BHV-7000 underway in epilepsy – Also initiated a Phase 2 study in MDD and Phase 2/3 studies in Bipolar Disorder and Generalized Epilepsy with BHV-7000.
Taldefgrobep alfa awarded "rare pediatric disease" designation – In April 2024, the Company announced that the Food and Drug Administration (FDA) granted "rare pediatric disease" designation for taldefgrobep alfa. The designation provides the potential for taldefgrobep to receive a PRV if ultimately approved for the indication of SMA.
Oral and poster presentations at AAN showcased breadth of development work across the platform – In April 2024, the Company delivered 8 oral presentations and 12 posters at the AAN Annual Meeting, showcasing development programs including Kv7 ion channel modulation, MoDEs, TRPM3 antagonism, TYK2/JAK1 inhibition, glutamate modulation, and myostatin inhibition.
AAN Abstract of Distinction awarded to BHV-2100, which demonstrated potent reversal of pain in preclinical models and favorable initial safety and pharmacokinetic data in Phase 1 studies, highlighting the potential for TRPM3 antagonism as a novel nonopioid target to treat pain and migraine.
Biohaven’s first-in-class Molecular Degrader of Extracellular Proteins (MoDE) technology targeting IgG removal, BHV-1300, was selected for an oral presentation at AAN highlighting its novel mechanism of action and the latest preclinical data demonstrating rapid, robust, and selective target removal.
Public offering – On April 22, 2024, the Company closed its previously announced underwritten public offering of 6,451,220 of its common shares, which included the full exercise of the underwriters’ option to purchase 841,463 additional shares, at the public offering price of $41.00 per share. The net proceeds raised in the offering, after deducting underwriting discounts and estimated expenses of the offering payable by the Company, were approximately $247.8 million. As of May 6, 2024, we had 88,291,909 common shares outstanding.
Expected Upcoming Milestones:
We believe Biohaven is well positioned to achieve significant, value-creating milestones in 2024 across numerous programs:
Selective Kv7 Activator:
Continue to advance Phase 2/3 programs in focal epilepsy, idiopathic generalized epilepsy, MDD and bipolar disorder
Troriluzole:
Continue two Phase 3 trials with troriluzole in OCD
Taldefgrobep alfa:
Initiate taldefgrobep Phase 2 study in obesity in 2H 2024
Report taldefgrobep Phase 3 topline results in SMA in 2H 2024
First-in-class TRPM3 Antagonist:
Initiate BHV-2100 Phase 2 study in acute migraine in 2H 2024
Conduct BHV-2100 proof of concept (POC) study for neuropathic pain in 2H 2024
TYK2/JAK1 Inhibitor:
Complete SAD/MAD studies with BHV-8000 and advance to Phase 2 in 2H 2024
Extracellular protein degradation platform
A total of 4 Investigational New Drug Applications (INDs) are expected for the degrader program in 2024
Provide progress updates regarding ongoing Phase 1 SAD study with BHV-1300 at annual R&D Day on May 29, 2024 followed up with a MAD update in 2H24
Next Generation ADC Platform:
Advance Phase 1 TROP2 directed ADC program with BHV-1510 in multiple tumor types
Capital Position:
Cash, cash equivalents, marketable securities and restricted cash totaled approximately $287.6 million on March 31, 2024, which excludes the net proceeds of approximately $247.8 million from Biohaven’s public offering completed on April 22, 2024.
First Quarter 2024 Financial Highlights:
Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $156.0 million for the three months ended March 31, 2024, compared to $63.5 million for the three months ended March 31, 2023. The increase of $92.5 million was primarily due to increases in direct program spend for additional and advancing clinical trials, including late Phase 2/3 studies, and preclinical research programs, as well as $16.6 million in common shares associated with the acquisition of Pyramid Biosciences, Inc., including a milestone payment for BHV-1510, and increased non-cash share-based compensation expense. Non-cash share-based compensation expense was $21.3 million for the three months ended March 31, 2024, an increase of $19.1 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the first 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 $27.3 million for the three months ended March 31, 2024, compared to $14.3 million for the three months ended March 31, 2023. The increase of $12.9 million was primarily due to increased non-cash share-based compensation expense. Non-cash share-based compensation expense was $13.6 million for the three months ended March 31, 2024, an increase of $12.1 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the first 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 income of $4.3 million for the three months ended March 31, 2024, compared to a net income of $8.2 million for the three months ended March 31, 2023. The decrease of $3.9 million was primarily due to a decrease of $3.9 million in other income recognized during the three months ended March 31, 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 March 31, 2024, of $179.5 million, or $2.20 per share, compared to $70.5 million, or $1.03 per share, for the same period in 2023. Non-GAAP adjusted net loss for the three months ended March 31, 2024 was $144.6 million, or $1.77 per share, compared to $66.7 million, or $0.98 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. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.