Biohaven Reports Third Quarter 2024 Financial Results and Recent Business Developments
Rhea-AI Summary
Biohaven reported its Q3 2024 financial results and recent business developments. As of October 2, 2024, the company had $642 million in cash and equivalents. Key achievements include positive topline results from a pivotal trial of troriluzole in spinocerebellar ataxia (SCA) and the initiation of a Phase 2 trial for BHV-2100 in acute migraine. Troriluzole met primary and secondary endpoints, showing significant improvements in SCA patients over three years. The company plans to submit an NDA in Q4 2024. Recent public offering raised $269.9 million. R&D expenses increased to $157.6 million, partly due to advancing clinical trials, while net loss widened to $160.3 million. Upcoming milestones include Phase 3 data for taldefgrobep alfa in SMA and continued progress across various programs.
Positive
- Positive topline results from pivotal trial of troriluzole in SCA.
- Planned NDA submission for troriluzole in Q4 2024.
- Initiation of Phase 2 trial for BHV-2100 in acute migraine.
- Raised $269.9 million from public offering.
- Numerous upcoming milestones across various programs.
Negative
- R&D expenses increased to $157.6 million.
- Net loss widened to $160.3 million.
News Market Reaction 1 Alert
On the day this news was published, BHVN declined 2.62%, reflecting a moderate negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
- Cash, cash equivalents, marketable securities and restricted cash as of October 2, 2024 totaled approximately
$642 million - Achieved positive topline trial results from pivotal trial with troriluzole in spinocerebellar ataxia (SCA)
- Troriluzole 200 mg QD dosed orally in patients with SCA met the study's primary endpoint on the change from baseline on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at 3 years in all study population genotypes
- Statistically significant superiority achieved on 9 consecutive, prespecified primary and secondary endpoints
- Both the study protocol and statistical analysis plan were submitted to, and reviewed by, the
U.S. Food and Drug Administration (FDA) prior to topline data analysis - Study designed in discussion with the FDA and utilized Phase 3 data and an external control of matched, untreated SCA subjects from the
U.S. Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) in accordance with the FDA's Guidance on Real-World Evidence (RWE) of effectiveness - CRC-SCA external control included contemporaneous natural history data gathered from 2010-2024
- Planned New Drug Application (NDA) re-submission in 4Q 2024
- Completed clarification meeting with CHMP Rapporteurs in 4Q 2024 and MAA documents are being updated to include the new positive BHV4157-206-RWE study data with broader indication to include all SCA genotypes
- Taldefgrobep alfa, a myostatin-inhibitor, Phase 3 topline data in spinal muscular atrophy (SMA) in 4Q 2024 and Phase 2 trial protocol in obesity expected in 4Q 2024
- Advancing extracellular Molecular Degrader of Extracellular Protein (MoDE) programs
- 3 additional investigational agents expected to enter Phase 1 studies in the next quarter
- Anticipate Phase 1 update for BHV-1300, including subcutaneous formulation, before year-end
- Broad progress with TRPM3 antagonist
- Initiated pivotal Phase 2 trial evaluating BHV-2100, a TRPM3 antagonist, in the acute treatment of migraine
- Initiated separate proof of concept study with BHV-2100 in neuropathic pain
- Patient enrollment continues across 5 Phase 2/3 trials with Kv7 activator, BHV-7000, in epilepsy and mood disorders (bipolar and major depressive disorder (MDD)) with potential for multiple data readouts in 2025
- Continued progress with antibody drug conjugate (ADC) portfolio
- BHV-1510 currently dosing cancer patients in Phase 1/2 study, advancing towards combination dosing of BHV-1510 with Libtayo® in 4Q 2024
Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "The team at Biohaven continues to advance multiple late and early stage assets that have the potential to change the current standard of care treatment paradigm across a number of diseases. Last quarter we announced positive topline results in our RWE trial assessing troriluzole for the treatment of spinocerebellar ataxia. Troriluzole (200 mg dosed orally) met the study's primary endpoint on the change from baseline in the f-SARA at 3 years in all study population genotypes, showed statistically significant superiority after both 1 and 2 years of treatment, and achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints. This was truly a watershed moment for the Company, given the implications of potentially bending the arc of one of the most intractable neurological diseases with no currently approved treatment options. SCA robs patients of their ability to speak, walk, and take care of themselves, and often shortens lifespans and has unfortunate intergenerational implications. We were encouraged by the robust dataset gathered to date and look forward to submitting an NDA to the FDA."
Dr. Coric continued, "We continue to execute across our broad pipeline including the recent initiation of our Phase 2 trial with BHV-2100 in acute migraine. Migraine burden and disability remain high despite advances in treatment; we believe our TRPM3 antagonistic approach has the potential to be a highly-effective, non-sedating, non-opioid treatment for pain and migraine. In the final months of 2024, we expect to report on a number of updates including SMA topline data and data across our MoDE™ platform including a SAD/MAD update and multiple INDs spanning β1AR for the potential treatment of dilated cardiomyopathy, galactose deficient IgA for IgA nephropathy and a further optimized IgG degrader for use in rare diseases."
Third Quarter 2024 and Recent Business Highlights
- Achieved positive topline results in pivotal study of troriluzole in SCA - In September 2024, the Company announced positive topline results from pivotal Study BHV4157-206-RWE demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2. Additionally, troriluzole achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints. SCA patients treated with troriluzole showed a 50
-70% slowing of disease progression, representing 1.5-2.2 years delay in disease progression over the 3-year study period. The Company intends to submit an NDA to the FDA for troriluzole in the treatment of all SCA genotypes in 4Q 2024. The development program for troriluzole has been granted orphan and fast track designations, and is eligible for priority review. European Medicines Agency marketing authorization remains under review and Biohaven completed a clarification meeting with CHMP Rapporteurs in 4Q 2024. MAA documents are being updated to include the new positive BHV4157-206-RWE study data with broader indication to include all SCA genotypes.
- Initiated Phase 2 trial evaluating BHV-2100 in the acute treatment of migraine - In September 2024, the Company initiated a Phase 2 study of an orally administered TRPM3 antagonist, BHV-2100, in the acute treatment of migraine. The study is a randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of two doses (75 mg and 150 mg) of BHV-2100 in the acute treatment of migraine. The trial is designed to support registration with FDA-accepted co-primary endpoints of pain freedom and freedom from most bothersome symptom at 2 hours and is expected to enroll approximately 575 patients across 60 sites in
the United States .
- Public offering - On October 2, 2024, the Company closed its previously announced underwritten public offering of 6,052,631 of its common shares, which included the full exercise of the underwriters' option to purchase additional shares, at the public offering price of
$47.50 $269.9M
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:
- NDA submission to FDA on track for 4Q 2024, following release of pivotal topline results in SCA in September 2024
- Two Phase 3 trials with troriluzole in OCD; expect to conduct interim analysis of the second Phase 3 OCD trial in 4Q 2024 and report topline data from first Phase 3 OCD trial in 1H 2025
Taldefgrobep alfa:
- Report topline data from Phase 3 trial with taldefgrobep in SMA in 4Q 2024
- Initiate Phase 2 trial with taldefgrobep in obesity in 4Q 2024 or early 2025
First-in-class TRPM3 Antagonist:
- Continue advancing enrollment in Phase 2 trial with BHV-2100 in acute migraine and neuropathic pain (laser-evoked potential experimental pain paradigm)
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 subcutaneous BHV-1300, with a further study update in 4Q 2024
Next Generation ADC Platform:
- Advance Phase 1 Trop-2 directed program BHV-1510 in multiple tumor types
Capital Position:
Cash, cash equivalents, marketable securities and restricted cash as of October 2, 2024 totaled approximately
Third Quarter 2024 Financial Highlights:
Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were
General and Administrative (G&A) Expenses: G&A expenses were
Other Income, Net: Other income, net was
Net Loss: Biohaven reported a net loss for the three months ended September 30, 2024 of
Non-GAAP Financial Measures
This press release includes financial results prepared in accordance with accounting principles generally accepted in
In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this news release.
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. The company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical 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; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules and antibody drug conjugates for cancer.
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
BIOHAVEN LTD. | ||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||
(Amounts in thousands, except share and per share amounts) | ||||||||
(Unaudited) | ||||||||
Three Months Ended | Nine Months Ended September | |||||||
2024 | 2023 | 2024 | 2023 | |||||
Operating expenses: | ||||||||
Research and development | $ 157,607 | $ 95,517 | $ 628,398 | $ 238,468 | ||||
General and administrative | 20,561 | 15,030 | 66,782 | 43,872 | ||||
Total operating expenses | 178,168 | 110,547 | 695,180 | 282,340 | ||||
Loss from operations | (178,168) | (110,547) | (695,180) | (282,340) | ||||
Other income, net | 17,805 | 4,686 | 36,288 | 18,757 | ||||
Loss before (benefit) provision for income taxes | (160,363) | (105,861) | (658,892) | (263,583) | ||||
(Benefit) provision for income taxes | (59) | (3,287) | 687 | (171) | ||||
Net loss | $ (160,304) | $ (102,574) | $ (659,579) | $ (263,412) | ||||
Net loss per share — basic and diluted | $ (1.70) | $ (1.50) | $ (7.50) | $ (3.86) | ||||
Weighted average common shares outstanding— basic and diluted | 94,372,159 | 68,320,125 | 87,936,923 | 68,258,757 | ||||
BIOHAVEN LTD. | ||||
CONSOLIDATED BALANCE SHEETS | ||||
(Amounts in thousands, except share amounts) | ||||
September 30, 2024 | December 31, 2023 | |||
(Unaudited) | ||||
Assets | ||||
Current assets: | ||||
Cash and cash equivalents | $ 84,390 | $ 248,402 | ||
Marketable securities | 294,426 | 133,417 | ||
Prepaid expenses | 55,168 | 35,242 | ||
Income tax receivable | 5,318 | 13,252 | ||
Other current assets | 1,198 | 12,133 | ||
Total current assets | 440,500 | 442,446 | ||
Property and equipment, net | 18,276 | 17,191 | ||
Intangible assets | 18,400 | 18,400 | ||
Goodwill | 1,390 | 1,390 | ||
Other non-current assets | 31,957 | 33,785 | ||
Total assets | $ 510,523 | $ 513,212 | ||
Liabilities and Shareholders' Equity | ||||
Current liabilities: | ||||
Accounts payable | $ 19,744 | $ 15,577 | ||
Accrued expenses and other current liabilities | 63,520 | 39,846 | ||
Forward contract liability | 69,030 | — | ||
Total current liabilities | 152,294 | 55,423 | ||
Non-current operating lease liabilities | 25,312 | 27,569 | ||
Derivative liability, non-current | 12,320 | — | ||
Other non-current liabilities | 4,591 | 2,245 | ||
Total liabilities | 194,517 | 85,237 | ||
Shareholders' Equity: | ||||
Preferred shares, no par value; 10,000,000 shares authorized, no shares issued | — | — | ||
Common shares, no par value; 200,000,000 shares authorized as of September | 1,381,699 | 887,528 | ||
Additional paid-in capital | 93,038 | 39,804 | ||
Accumulated deficit | (1,158,871) | (499,292) | ||
Accumulated other comprehensive income (loss) | 140 | (65) | ||
Total shareholders' equity | 316,006 | 427,975 | ||
Total liabilities and shareholders' equity | $ 510,523 | $ 513,212 | ||
BIOHAVEN LTD. | ||||||||
RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL MEASURES | ||||||||
(Amounts in thousands, except share and per share amounts) | ||||||||
(Unaudited) | ||||||||
Three Months Ended | Nine Months Ended September | |||||||
2024 | 2023 | 2024 | 2023 | |||||
Reconciliation of GAAP to Non-GAAP adjusted net loss: | ||||||||
GAAP net loss | $ (160,304) | $ (102,574) | $ (659,579) | $ (263,412) | ||||
Add: non-cash share-based compensation expense | 12,160 | 4,456 | 59,269 | 12,916 | ||||
Add: (gain) loss from change in fair value of derivatives | (15,990) | — | (17,030) | — | ||||
Non-GAAP adjusted net loss | $ (164,134) | $ (98,118) | $ (617,340) | $ (250,496) | ||||
Reconciliation of GAAP to Non-GAAP adjusted net loss per share — basic and diluted: | ||||||||
GAAP net loss per share — basic and diluted | $ (1.70) | $ (1.50) | $ (7.50) | $ (3.86) | ||||
Add: non-cash share-based compensation expense | 0.13 | 0.07 | 0.67 | 0.20 | ||||
Add: (gain) loss from change in fair value of derivatives | (0.17) | — | (0.19) | — | ||||
Non-GAAP adjusted net loss per share — basic and diluted | $ (1.74) | $ (1.44) | $ (7.02) | $ (3.67) | ||||
MoDEs is a trademark of Biohaven Therapeutics Ltd.
Libtayo is a registered trademark of Regeneron Pharmaceuticals, Inc.
Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
+1 (201) 248-0741
Media Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
+1 (312) 961-2502
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SOURCE Biohaven Ltd.
FAQ
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