Biohaven Reports Third Quarter 2024 Financial Results and Recent Business Developments
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.
Biohaven ha riportato i risultati finanziari per il terzo trimestre del 2024 e gli sviluppi aziendali recenti. Al 2 ottobre 2024, l'azienda aveva 642 milioni di dollari in contante e equivalenti. I risultati chiave includono risultati positivi dalla prova pivotale di troriluzolo nell'atassia spinocerebellare (SCA) e l'inizio di una prova di Fase 2 per BHV-2100 nell'emicrania acuta. Il troriluzolo ha raggiunto obiettivi primari e secondari, mostrando miglioramenti significativi nei pazienti con SCA nel corso di tre anni. L'azienda prevede di presentare un NDA nel quarto trimestre del 2024. Un'offerta pubblica recente ha raccolto 269,9 milioni di dollari. Le spese di ricerca e sviluppo sono aumentate a 157,6 milioni di dollari, in parte a causa dell'avanzamento delle prove cliniche, mentre la perdita netta è aumentata a 160,3 milioni di dollari. Tra i prossimi traguardi ci sono i dati di Fase 3 per taldefgrobep alfa nella SMA e progressi continui in vari programmi.
Biohaven informó sobre sus resultados financieros del tercer trimestre de 2024 y desarrollos empresariales recientes. Al 2 de octubre de 2024, la empresa contaba con 642 millones de dólares en efectivo y equivalentes. Los logros clave incluyen resultados positivos de un ensayo pivotal de troriluzole en la ataxia espinocerebelosa (SCA) y el inicio de un ensayo de Fase 2 para BHV-2100 en migraña aguda. El troriluzole cumplió con los objetivos primarios y secundarios, mostrando mejoras significativas en pacientes con SCA durante tres años. La compañía planea presentar una NDA en el cuarto trimestre de 2024. La oferta pública reciente recaudó 269,9 millones de dólares. Los gastos de I+D aumentaron a 157,6 millones de dólares, en parte debido al avance de los ensayos clínicos, mientras que la pérdida neta se amplió a 160,3 millones de dólares. Los próximos hitos incluyen datos de Fase 3 para taldefgrobep alfa en SMA y un progreso continuo en varios programas.
Biohaven는 2024년 3분기 재무 결과와 최근 사업 발전 사항을 보고했습니다. 2024년 10월 2일 기준으로, 회사는 6억 4,200만 달러의 현금 및 현금성 자산을 보유하고 있었습니다. 주요 성과로는 스피노세레벨라 아탁시아(SCA)에 대한 트로릴루졸의 주요 임상 시험에서 긍정적인 topline 결과와 급성 편두통에 대한 BHV-2100의 2상 시험 시작이 포함됩니다. 트로릴루졸은 주요 및 보조 목표를 충족하며, 3년 동안 SCA 환자에서 유의미한 개선을 보여주었습니다. 회사는 2024년 4분기에 NDA를 제출할 계획입니다. 최근의 공모에서 2억 6,990만 달러를 모금했습니다. 연구개발 비용은 1억 5,760만 달러로 증가했으며, 이는 임상 시험 진행에 따른 부분입니다. 순손실은 1억 6,030만 달러로 확대되었습니다. 다가오는 주요 이정표로는 SMA에 대한 탈데프그로베프 알파의 3상 데이터와 다양한 프로그램 전반의 지속적인 진전을 포함합니다.
Biohaven a rapporté ses résultats financiers pour le troisième trimestre 2024 et les développements récents de l'entreprise. Au 2 octobre 2024, la société disposait de 642 millions de dollars en liquidités et équivalents. Les réussites clés comprennent des résultats positifs de l'essai pivot du troriluzole dans l'ataxie spinocérébelleuse (SCA) et le lancement d'un essai de phase 2 pour le BHV-2100 dans la migraine aiguë. Le troriluzole a atteint les objectifs primaires et secondaires, montrant des améliorations significatives chez les patients atteints de SCA au cours de trois ans. La société prévoit de déposer une NDA au quatrième trimestre 2024. Une récente offre publique a permis de lever 269,9 millions de dollars. Les dépenses de R&D ont augmenté pour atteindre 157,6 millions de dollars, en partie en raison de l'avancement des essais cliniques, tandis que la perte nette s'est élargie à 160,3 millions de dollars. Parmi les prochaines étapes, on trouve des données de phase 3 pour le taldefgrobep alfa dans la SMA et des progrès continus dans divers programmes.
Biohaven hat seine Finanzzahlen für das 3. Quartal 2024 und aktuelle Geschäftsentwicklungen berichtet. Zum 2. Oktober 2024 hatte das Unternehmen 642 Millionen Dollar in bar und Barmitteläquivalenten. Zu den wichtigsten Erfolgen zählen positive Topline-Ergebnisse aus einer entscheidenden Studie zu Troriluzole bei spinozerebellärer Ataxie (SCA) und der Start einer Phase-2-Studie für BHV-2100 bei akuten Migräneanfällen. Troriluzole hat die primären und sekundären Endpunkte erreicht und deutliche Verbesserungen bei SCA-Patienten über drei Jahre gezeigt. Das Unternehmen plant, im 4. Quartal 2024 einen NDA-Antrag einzureichen. Eine kürzliche öffentliche Angebotserhöhung brachte 269,9 Millionen Dollar ein. Die Forschungs- und Entwicklungskosten stiegen auf 157,6 Millionen Dollar, was teilweise auf die Fortschritte bei klinischen Studien zurückzuführen ist, während der Nettoverlust auf 160,3 Millionen Dollar ausgeweitet wurde. Zu den bevorstehenden Meilensteinen gehören Daten der Phase 3 für Taldefgrobep-Alfa bei SMA und weiterer Fortschritt in verschiedenen Programmen.
- 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.
- R&D expenses increased to $157.6 million.
- Net loss widened to $160.3 million.
Insights
The Q3 results reveal significant developments and financial positioning. Cash position of
The positive troriluzole trial results in SCA represent a potential breakthrough in an untreated market. With FDA fast track designation and planned Q4 2024 NDA submission, this could translate to accelerated revenue potential. Multiple late-stage catalysts across the pipeline in Q4 2024 and 2025 could drive significant value creation.
The troriluzole SCA trial results are particularly noteworthy, demonstrating
The diversified late-stage pipeline, including taldefgrobep alfa for SMA, BHV-7000 for epilepsy/mood disorders and BHV-2100 for migraine, presents multiple shots on goal in significant market opportunities. The advancement of novel platforms like MoDE and ADC programs adds long-term potential.
- 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.
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