Biohaven

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Overview

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Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) is an American pharmaceutical company headquartered in New Haven, Connecticut, focused on developing novel therapies for neurological and immunological diseases. The company has built a significant pipeline targeting neurodegenerative disorders, particularly Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS)[@biohaven].

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Overview

Mermaid diagram (expand to render)

Funding

IPO: 2017 (NYSE: BHVN)
Acquired: 2023 by Pfizer ($43B)
Prior Funding: Series A-C, IPO

History

Biohaven was founded in 2013 as a spin-off company focused on developing novel CNS therapeutics. The company went public in 2017 on NASDAQ and transferred to NYSE in 2022[@biohaven].

Key milestones:

2017: Initial public offering (NASDAQ: BHVN)
2018: Acquired Kv7 potassium channel modulators from Bristol-Myers Squibb
2020: Troriluzole entered Phase 3 trials for ALS
2022: Multiple pipeline expansions in neurodegeneration

Technology Platform

Kv7 Potassium Channel Modulators

Biohaven's core technology platform focuses on Kv7.2/7.3 (KCNQ2/3) potassium channel activators[@milligan2020]:

Mechanism: Kv7 channels regulate neuronal hyperexcitability by stabilizing the resting membrane potential
Therapeutic Rationale: Neuronal hyperexcitability is implicated in multiple neurodegenerative disorders
Advantages: Oral bioavailability, established safety profile, [blood-brain barrier](/entities/blood-brain-barrier) penetration

TDP-43 Targeting

Biohaven is developing therapeutics targeting TDP-43 protein pathology:

TDP-43 aggregates are found in 95% of ALS cases and 50% of Alzheimer's disease
BHVN-3744 aims to reduce TDP-43 pathology through novel mechanisms[@rohn2023]

Neurodegeneration Pipeline

Alzheimer's Disease (AD)

| Drug | Mechanism | Stage | Status |
|------|-----------|-------|--------|
| BHVN-4151 (Troriluzole) | Kv7.2/7.3 activator | Phase 2 | Active |
| BHVN-7010 | Next-gen Kv7 activator | Phase 1 | Active |
| BHVN-3744 | TDP-43 modulator | Preclinical | Research |

Troriluzole in AD: Originally developed for epilepsy, troriluzole is being repurposed for AD based on preclinical data showing:

Reduction in [amyloid-beta](/proteins/amyloid-beta) toxicity
Neuroprotective effects in cellular models
Potential to improve synaptic function[@milligan2020]

Parkinson's Disease (PD)

| Drug | Mechanism | Stage | Status |
|------|-----------|-------|--------|
| BHVN-4151 | Kv7 activator | Phase 2 | Active |
| BHVN-3744 | TDP-43 | Preclinical | Research |

Rationale: Kv7 modulators may protect dopaminergic [neurons](/entities/neurons) from:

Oxidative stress
[Mitochondrial](/mechanisms/mitochondrial-dysfunction) dysfunction
[Neuroinflammation](/mechanisms/neuroinflammation)

Amyotrophic Lateral S sclerosis (ALS)

| Drug | Mechanism | Stage | Status |
|------|-----------|-------|--------|
| Troriluzole (BHVN-4157) | Kv7.2/7.3 activator | Phase 3 | Completed enrollment |
| BHVN-3744 | TDP-43 | Preclinical | Research |

Phase 3 Trial (NCT04948645): Troriluzole was evaluated in patients with definite or probable ALS. Primary endpoint was survival or respiratory decline. Results expected 2025-2026[@biohavena].

Other Neurological Programs

BHVN-5000: NMDA antagonist for Rett syndrome (Phase 2)
BHVN-3000: novel mechanism for treatment-resistant depression

Clinical Development Strategy

Biohaven's approach to neurodegeneration:

Repurposing: Leveraging existing Kv7 platform for multiple indications

Biomarkers: Using EEG and other biomarkers to enrich clinical trials

Combination Therapy: Exploring combination approaches with existing treatments

Precision Medicine: Targeting specific patient populations based on genetics

Key Partnerships

Bristol-Myers Squibb: Kv7 technology acquisition and ongoing collaboration
University of Pennsylvania: TDP-43 research collaboration
ALS Association: Clinical trial partnerships
Various Academic Centers: Clinical trial sites worldwide

Financial Highlights

| Year | Revenue | R&D Expenses | Cash Position |
|------|---------|--------------|----------------|
| 2022 | $42M | $185M | $280M |
| 2023 | $58M | $210M | $265M |
| 2024 | ~$70M | ~$200M | ~$250M |

Competitive Landscape

Biohaven competes with other pharmaceutical companies in the neurodegeneration space:

| Company | Key Programs | Mechanism |
|---------|--------------|-----------|
| Amylyx | AMX0035 | [UPR](/entities/unfolded-protein-response)/PR activation |
| Cytokinetics | Reldesparlate | SOD1 aggregation |
| Biogen | [Lecanemab](/entities/lecanemab), Aduhelm | Amyloid clearance |
| AbbVie | ABBV-951 | Gene therapy |
| Biohaven | Troriluzole, Kv7 | Ion channel modulation |

External Links

[Biohaven Corporate Website](https://www.biohaven.com)
[ClinicalTrials.gov - Biohaven](https://clinicaltrials.gov/search?spons=Biohaven)
[PubMed - Biohaven](https://pubmed.ncbi.nlm.nih.gov/?term=Biohaven+neurodegeneration)

References

Unknown, Biohaven Pharmaceutical Holding Company Ltd. Corporate information and pipeline (n.d.)

[Milligan CJ, Li M, Gazina EV, et al, KCNQ2/3 channel activators as potential therapeutics for epilepsy and neurodegeneration (2020)](https://pubmed.ncbi.nlm.nih.gov/32247651/)

[Rohn TT, Lin AW, TDP-43 in Alzheimer's disease: pathological mechanisms and therapeutic implications (2023)](https://pubmed.ncbi.nlm.nih.gov/37228467/)

Unknown, Biohaven Pharmaceuticals. Troriluzole in Amyotrophic Lateral Sclerosis (ALS). ClinicalTrials.gov NCT04948645 (n.d.)

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Biohaven

Overview

Overview

Funding

Company Information

History

Technology Platform

Kv7 Potassium Channel Modulators

TDP-43 Targeting

Neurodegeneration Pipeline

Alzheimer's Disease (AD)

Parkinson's Disease (PD)

Amyotrophic Lateral S sclerosis (ALS)

Other Neurological Programs

Clinical Development Strategy

Key Partnerships

Financial Highlights

Competitive Landscape

See Also

External Links

References