Molefy pharma
Molefy Pharma is a clinical-stage Spanish biotech developing small-molecule therapeutics that target TDP-43 proteinopathy in neurodegenerative diseases. Its lead candidate AP-2 is in Phase I trials for ALS, and AP-3 is in preclinical development for frontotemporal dementia.
- Company typePrivate
- Founded2024
- HeadquartersSan Cristóbal De La Laguna, Spain
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What Molefy pharma does
Molefy Pharma is a clinical-stage Spanish biotechnology company founded in 2024 as a spin-off of ARQUIMEA (majority investor) and CSIC (Consejo Superior de Investigaciones Científicas), developing small-molecule therapeutics that target TDP-43 proteinopathy in neurodegenerative diseases. Its lead candidate AP-2 is a CK1δ kinase inhibitor designed to restore TDP-43 cellular homeostasis, with the goal of halting and reversing disease progression in Amyotrophic Lateral Sclerosis (ALS). AP-2 received Orphan Drug Designation from the European Medicines Agency in October 2025 and was authorized by the Spanish Agency of Medicines (AEMPS) to begin Phase I clinical trials in approximately 70 healthy volunteers at Hospital Universitario de La Princesa (Madrid) in April 2026, with a Phase Ib in ~24-28 ALS patients planned from 2027 and a multicenter Phase II from 2028. A second preclinical candidate, AP-3, inhibits the TTBK1 enzyme for frontotemporal dementia and is supported by the Alzheimer's Drug Discovery Foundation.
The company operates a research-driven, grant- and equity-funded business model with no commercial revenue. Capital sources include ARQUIMEA's majority equity stake, a $1 million non-refundable grant from the ALS Association (October 2025), preclinical funding from ADDF, and a planned Phase II investment round targeting institutional investors, VC funds, and strategic partners. The pipeline is built on foundational CSIC patents (CK-1δ filed 2013; TTBK1 filed 2021) licensed to Molefy Pharma at founding, with efficacy demonstrated in patient-derived cellular models and rodent and minipig animal studies. The ~8-person core team is led by CEO Alfonso de Egaña with scientific co-founders Ana Martínez PhD and Carmen Gil PhD (CIB-CSIC researchers), and a Scientific Advisory Board that includes Don W. Cleveland (UC San Diego). The company is headquartered in San Cristóbal de La Laguna, Tenerife, with clinical operations in Madrid.
Molefy pharma firmographics
Firmographics- Name
- Molefy pharma
- Legal name
- Molefy Pharma SL
- Website
- https://molefypharma.com
- Company type
- Private
- Founded year
- 2024
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Molefy Pharma is a clinical-stage Spanish biotech developing small-molecule therapeutics that target TDP-43 proteinopathy in neurodegenerative diseases. Its lead candidate AP-2 is in Phase I trials for ALS, and AP-3 is in preclinical development for frontotemporal dementia.
- Ownership category
- akta.pro rank
Molefy pharma industry classification
Industry- Product category
- Biopharmaceuticals / Neurology Therapeutics
- NAICS
- Pharmaceutical Preparation Manufacturing (325412), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical and Medicine Manufacturing (3254)
- SIC
- Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731), Medicinal Chemicals & Botanical Products (2833)
- akta.pro primary industry
- Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
- akta.pro secondary industry
- Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)
Keywords
Where Molefy pharma is headquartered
LocationHeadquarters
- HQ city
- San Cristóbal De La Laguna
- HQ country
- Spain
- HQ region
- Europe
Offices2 records
Markets served
Molefy pharma business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales
Revenue model
- Pharmaceutical / Biopharmaceutical product revenues: Pre-revenue. The company is developing AP-2 as a disease-modifying therapy for ALS. Upon successful clinical development and regulatory approval, the company aims to commercialize the therapy and generate product revenues. The company is also developing AP-3 for frontotemporal dementia.
- Non-dilutive funding (grants): Molefy Pharma pursues non-dilutive funding from public and private sources including the ALS Association ($1 million awarded in 2025) and the Alzheimer's Drug Discovery Foundation (funding AP-3 preclinical work). These grants support clinical trial and preclinical development without diluting equity.
- Equity investment rounds: The company is currently in an active fundraising phase and plans to launch a Phase II investment round targeting institutional investors, venture capital funds, and strategic partners. ARQUIMEA is the majority investor.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels5 records
Molefy pharma product offering
Product offeringCore offering
Molefy Pharma is a clinical-stage biotech company developing small-molecule therapeutics for neurodegenerative diseases. Its lead candidate AP-2, a CK1δ kinase inhibitor that restores TDP-43 protein homeostasis, is in Phase I clinical trials for Amyotrophic Lateral Sclerosis (ALS). Its second candidate AP-3, a TTBK1 inhibitor, is in preclinical development for frontotemporal dementia (FTD). Both are based on foundational patents licensed from CSIC and validated in patient-derived cellular and animal models.
Product overview
Molefy Pharma is a biotech company with a focused pipeline of two therapeutic candidates for neurodegenerative diseases. The lead product AP-2 is a small molecule in Phase I clinical development for ALS, targeting TDP-43 proteinopathy. The secondary candidate AP-3 is in preclinical development for frontotemporal dementia. The company's scientific approach combines molecular biology research from CSIC with industrial development capabilities from ARQUIMEA.
Differentiator
Problem solved
Functional benefit
Products and services
- AP-2 Small molecule therapeutic candidate designed to restore cellular balance by regulating TDP-43 protein homeostasis via CK1δ kinase inhibition. AP-2 targets the TDP-43 proteinopathy found in over 97% of ALS patients, with potential to not only alleviate symptoms but reverse cellular dysfunction. Demonstrated reversal of cognitive deficits in ALS mouse models and favorable safety in rodent and minipig studies under GMP conditions. Phase I clinical trial for Amyotrophic Lateral Sclerosis (ALS).
- AP-3 Preclinical therapeutic candidate for frontotemporal dementia (FTD). AP-3 is a compound that inhibits the TTBK1 enzyme, which plays a key role in TDP-43 protein alteration and pathology in frontotemporal dementia. Demonstrated significant memory improvement, neuronal preservation, and favorable safety profile in animal models, with strong brain penetration and high selectivity.
Quantifiable outcome
- Phase I clinical trial authorized by AEMPS (April 2026) with ~70 healthy volunteers to assess safety; Phase Ib planned with ~28 ALS patients from 2027; Phase II from 2028
- +2 more outcomes
Companies that use Molefy pharma
Customer profileNamed customers1 record
Segments4 records
Ideal customer profiles3 records
Molefy pharma technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Molefy pharma partnerships and signals
Strategic signalPartnerships
Three partnerships are on record, tiered core and major.
- Hospital Universitario de La Princesa (Madrid)corePhase I clinical trial site for AP-2, authorized by AEMPS. The Clinical Trials Unit at Hospital Universitario de La Princesa (Madrid) will evaluate AP-2 safety in approximately 70 healthy volunteers beginning April 2026. Subsequent Phase Ib will include approximately 28 ALS patients.
- ENCALS (European Network to Cure ALS)majorENCALS is Europe's foremost collaborative platform for ALS research, gathering leading ALS centers, clinicians, and scientists to foster data harmonization, translational science, and collaborative clinical development. Molefy Pharma presented three scientific papers at ENCALS 2025 in Turin, reinforcing strategic European partnerships and scientific credibility.
- CSIC (Consejo Superior de Investigaciones Científicas)coreCSIC is the principal public research institution in Spain and a leading European research body. Provides scientific excellence, cutting-edge molecular biology research, and foundational patents (CK-1º and TTBK1) that underpin Molefy Pharma's therapeutic candidates. Researchers Ana Martínez and Carmen Gil from CIB-CSIC are co-founders. The license transfer from CSIC to Molefy Pharma formally launched the company.
Scale indicators7 records
Recent moves6 records
Expansion highlights6 records
Molefy pharma competitors and assessment
Company assessmentDirect peers
- Prothena: Clinical-stage biotech whose pipeline (PRX012, tau programs) targets proteinopathies including TDP-43-related neurodegeneration. Directly comparable mechanism focus on misfolded protein clearance in neurodegenerative diseases.
- Cytokinetics: Clinical-stage biotech advancing small-molecule ALS therapeutics (reldesemtiv). Directly comparable late-stage clinical ALS competitor developing mechanistically distinct muscle/neuromuscular candidates.
- Amylyx Pharmaceuticals: Clinical-stage biotech developing small-molecule therapies for ALS (AMX0035/Relyvrio). Most directly comparable peer as a small-molecule disease-modifying ALS developer with similar regulatory and clinical-stage profile.
Broad incumbents
- Biogen: Large incumbent in neurodegenerative disease drug development (Spinraza, Tofersen for SOD1-ALS, Aduhelm). Owns the most relevant ALS franchise and a broad CNS pipeline that competes for the same regulatory and commercial slots.
- Ionis Pharmaceuticals: Large antisense oligonucleotide platform leader with multiple ALS programs (partnering with Biogen on Tofersen, internal programs). Sets the innovation benchmark in TDP-43/SOD1-ALS therapeutic development.
Emerging players
- QurAlis Corporation: Early-stage biotech developing antisense oligonucleotides targeting TDP-43 and other ALS mechanisms. Highly comparable focus on TDP-43 proteinopathy from a different (ASO) modality angle.
- Knopp Biosciences: Clinical-stage small-molecule developer focused on ion channel and kinase targets for neuroscience indications including ALS. Comparable as a small-cap small-molecule neuroscience biotech.
- Clene Nanomedicine: Clinical-stage biotech developing CNM-Au8, a gold nanocatalyst for ALS and other neurodegenerative diseases. Comparable emerging clinical-stage ALS therapeutics competitor.
Others
- Ankarpharma SL: Earlier CSIC spin-off that held the CK-1δ patent transferred to Molefy Pharma at founding (2014). Direct lineage comparable as a Spanish neurodegenerative spin-off from the same research group.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat4 records
Key risks7 records
Key highlights7 records
Customer concentration
Molefy pharma social profiles
Digital presenceMolefy pharma compliance and trust
Trust signalCompliance1 record
Molefy pharma financial estimates
Financial estimateRevenue estimate
Valuation estimate
Molefy pharma leadership team
Management profileNumber of profiles
Profiles9 records
Molefy pharma funding detail
Funding detailFunding overview
Funding rounds1 record
Investors1 record
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Molefy pharma M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Molefy pharma
What does Molefy pharma do?
Molefy Pharma is a clinical-stage biotech company developing small-molecule therapeutics for neurodegenerative diseases. Its lead candidate AP-2, a CK1δ kinase inhibitor that restores TDP-43 protein homeostasis, is in Phase I clinical trials for Amyotrophic Lateral Sclerosis (ALS). Its second candidate AP-3, a TTBK1 inhibitor, is in preclinical development for frontotemporal dementia (FTD). Both are based on foundational patents licensed from CSIC and validated in patient-derived cellular and animal models.
Is Molefy pharma a public or private company?
Molefy pharma is a private company. It is classified as corporate owned and is currently operating.
When was Molefy pharma founded?
Molefy pharma was founded in 2024. It employs 1 to 10 people.
Where is Molefy pharma based?
Molefy pharma is headquartered in San Cristóbal De La Laguna, Spain, in the Europe region.
How does Molefy pharma make money?
Three revenue lines are on record. Pharmaceutical / Biopharmaceutical product revenues are the primary driver. The others are non-dilutive funding (grants) and equity investment rounds.
Who are Molefy pharma's main competitors?
Direct peers on record are Prothena, Cytokinetics and Amylyx Pharmaceuticals. Broad incumbents are Biogen and Ionis Pharmaceuticals. Emerging players are QurAlis Corporation, Knopp Biosciences and Clene Nanomedicine. Ankarpharma SL is listed as an others.
Does Molefy pharma have an API?
No public API is recorded for Molefy pharma.
What industry is Molefy pharma in?
Molefy pharma's product category is Biopharmaceuticals / Neurology Therapeutics. Its primary akta.pro industry code is HLAIAIAD, Rare Neurology & Neurodegenerative Disorder Therapies, with a secondary code of HLAIACAC, Neurology & CNS Specialty Pharmaceuticals. Its NAICS code is 325412 and its SIC code is 2834.