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Currently, an estimated 6.7 million Americans live with Alzheimer’s disease (AD), whereas about 44 million people globally live with AD; these numbers are likely to reach 13.8 million in the United States and 131.5 million worldwide by 2050. AD is the sixth leading cause of death in the U.S., with a 146.2% increase in deaths caused between 2000 and 2018. According to World Health Organization projections, the percentage of the world’s population older than 60 is likely to go from 12% to 22% by 2030.
Phanes Biotech is developing two molecules for the prevention and treatment of AD and related neurodegenerative disorders:
- Neuro-regenerative small molecule: PB021, a peptidergic compound proven to prevent the destruction of nerve cells and to regenerate nerve cells undergoing neurodegenerative changes
- Tau immunotherapy: PB43D, a tau monoclonal antibody which reduces both tau and Aβ pathologies
Because of the huge unmet medical need, there is an exponential market opportunity of more than $600 billion for AD. The AD therapeutic market is likely to grow by $3.11 billion from 2020 to 2024 at a compounded annual growth rate of almost 6.9%.
Competitive Advantage and Value Proposition
Phanes Biotech has a unique competitive advantage over other companies working on AD drug development.
| Type of Pathology | Companies | Results | Phanes Biotech |
|---|---|---|---|
| Aβ pathology | Biogen, Eisai, Eli Lilly, Roche, Genentech, Novartis, Amgen | Reduction of Aβ pathology but modest reduction in the rate of cognitive decline | PB021 stimulates regeneration of the brain and inhibits neurodegeneration, both tau and Aβ pathologies, and cognitive impairment.PB43D clears both tau and Aβ pathologies and rescues cognitive impairment. Highly potent and high-affinity monoclonal antibody. |
| Tau pathology | AbbVie, Biogen, BMS, Eli Lilly, Roche, TauRx, AC Immune, Axon Neuroscience | Negative to partially positive | |
| Neuro-regeneration | Cognitive Therapeutics, Athira Pharma | Works on neuro-regeneration but has no effect on Aβ and tau |
Company Milestones
Scientific and Clinical
Neuro-regenerative small molecule: PB021
- 2010: Target identification, lead optimization
- 2012: Patents on the composition and use of PB021 for the treatment of AD and other neurodegenerative disorders
- 2017: Pharmacokinetic-pharmacodynamic studies in mice
- 2018: Stability studies and in vitro receptor affinity studies and successful production of several batches of the molecule
- 2024: Good Manufacturing Practice (GMP) production and pre-investigational new drug (IND) studies
- 2025: Pre-IND studies and approval of IND for PB43D and PB021
- 2026: Generation of GMP production and Phase 1 Clinical trials
- 2028: Phase 2 human clinical trials in AD patients
Tau immunotherapy: PB43D
- 2016: Approval of a patent on epitope and use of PB43D for the treatment of AD and related tauopathies
- 2017: Preclinical studies showing clearance of both tau and Aβ pathologies
- 2018: Inhibition of the seeding and spread of tau pathologies induced by AD hyperphosphorylated tau in an AD transgenic mouse model
- 2023: Successful humanization of PB43D
- 2025: Pre-IND studies and approval of IND
- 2026: GMP production of humanized PB43D and Phase 1 human clinical trials
- 2027: Phase 2 human clinical trials in AD patients
Business
- 2020: Company formation as C corporation
- 2021: Licensing agreement from RFMH for both molecules
- 2022: Awarded NIH/NIA Phase 1 Small Business Innovation Research (SBIR) grant for tau immunotherapy
- 2023: In process of submitting three composition-of-matter claims for immunotherapy molecule; submitted application for patent claim and strategic partnership with Veravas for AD screening test
- 2024: Intend to raise pre-Series A funding to $12 million
Financial Overview
Phanes Biotech has raised $450,000 from self-funding from promoters and $500,000 from NIH SBIR Phase 1 funding. The company is currently seeking to raise $12 million pre-Series A by July 2024. Future funds should raise $150 million to $200 million for completion of Phase 1 and early Phase 2 studies.
Intellectual Property
Phanes Biotech entered into an exclusive licensing agreement with the Research Foundation for Mental Hygiene (RFMH) in 2021. Under this agreement, RFMH granted to Phanes Biotech exclusive worldwide license for PB021 and PB43D to make, use, sell, and offer for sale licensed products and processes that embody the licensed products governed under current or future patents pertaining to the agreement entered that form the basis for development of drug therapies. Phanes Biotech has a strong patent position: eight patents for methods of use and composition of matter. The company is in the process of applying for an additional four patent claims by Quarter 1 of 2024.
Product Development and Regulatory Strategy
Phanes Biotech’s regulatory strategy will be based on the U.S. Food and Drug Administration (FDA) regulatory guidance and recommendations, along with consideration for fast-track application, depending on the stage of our development of P021 and PB43D as a safe and effective drug providing a meaningful therapeutic benefit to millions of AD patients.
- Preclinical development: Includes chemistry, manufacturing, and controls and IND-enabling studies.
- Clinical trial design: Will be based on the FDA guidance issued for selecting ideal patient population biomarkers and endpoints. An ideal target population for PB021 and PB43D includes individuals with mild to severe AD with cognitive impairment. The efficacy will be assessed by measuring the biomarkers in plasma, tau, and phosphorylated tau, along with positron emission tomography/magnetic resonance imaging scans with appropriate endpoints as defined by the FDA.
- Regulatory interactions: We plan to engage with the FDA during every step of the drug development to understand and discuss the regulatory strategy for the preclinical studies and clinical trial design and endpoints. Data compliance and security will be of utmost importance and will be conducted in accordance with applicable regulations and guidelines stipulated by the FDA, including the Health Insurance Portability and Accountability Act, and in the Common Technical Document format.
Commercialization Strategy
The short-term objective is to engage clinical research operations to conduct pre-IND studies, a Phase 1 clinical trial for AD and a Phase 2 clinical trial for AD patients. Phanes Biotech’s long-term objective is to become an independent biopharma company focusing on novel therapies for neurodegenerative diseases.
Phanes Biotech’s revenue strategy predominantly involves revenue from licensing/development collaborations for PB021/PB43D for AD and multiple neurological indications and revenue from the strategic partnership with Veravas. A licensing agreement with a pharmaceutical or biotech company would include an up-front payment, milestone payments based on clinical development and regulatory progress, regulatory approval, and royalties on product sales.
Company Details
Newtown Square, PA
Industry: Therapeutics
Management Team:
- CEO & Founder: Senthil Sambandam, M.D.
- CSO & Co-Founder: Khalid Iqbal, Ph.D.
- Director Drug Development: Manfred Windisch, Ph.D.
Point of Contact:
Senthil Sambandam
senthil@phanesbiotech.com
206-423-5617