What Connecta Therapeutics does

Connecta Therapeutics is a clinical-stage biotechnology company headquartered at the Barcelona Science Park that develops disease-modifying small molecule treatments for unresolved central nervous system (CNS) disorders, with an initial focus on Fragile X Syndrome (FXS). Founded in July 2019 as a spin-off of Prous Institute for Biomedical Research, the company applies a network pharmacology platform that integrates in silico drug design with artificial intelligence techniques to identify and optimize multi-target small molecules acting on neurotrophic factor pathways. Its lead asset CTH120 is a first-in-class small molecule neuroplasticity modulator targeting the tropomyosin receptor kinase B (TrkB) receptor; preclinical studies demonstrated that CTH120 rescues dendritic pathology in FXS models, improves cognitive ability, and restores social abnormalities. A second patented compound, CTH122, has shown preclinical efficacy in both FXS and Rett syndrome models, indicating platform scalability to additional neurodevelopmental disorders.

CTH120 has received EMA Orphan Drug Designation (EU/3/21/2432, granted 2021), conferring 10-year market exclusivity in the EU upon approval and providing protocol and fee benefits during development. Connecta completed a Phase I trial in 76 healthy adults (initiated 2023, positive safety data reported March 2025) and initiated a Phase IIa randomized, double-blind, placebo-controlled multicenter study in 30 adult males aged 18–45 with FXS at Hospital del Mar Research Institute and Parc Taulí in June 2026. A pediatric development program supported by a €2.5M EIC Accelerator grant (September 2025) is underway, including formulation development for an age-appropriate oral dosage form.

The company is pre-revenue and funds operations through a mix of equity (€1.7M seed round led by Inveready in September 2020, with CDTI Innvierte and founders) and non-dilutive grants (AEI Retos-Colaboración 2019, NEOTEC 2021, ENISA participatory loan 2022, NextGenerationEU Phase IIa grant 2024, EIC Accelerator 2025). Total disclosed funding exceeds €5M. Go-to-market will follow the traditional pharmaceutical commercialization pathway through EMA approval, with orphan drug exclusivity providing the primary commercial protection in the EU. Target end users are adult and pediatric patients with FXS and, over time, patients with other neurodevelopmental disorders characterized by impaired neuroplasticity (Rett syndrome, Down syndrome, ASD, ADHD). The core team of 1–10 employees is supplemented by a strong scientific advisory board including Dr. Randi Hagerman (UC Davis MIND Institute) and Dr. Mara Dierssen (CRG).

Name

Connecta Therapeutics

Legal name

CONNECTA Therapeutics, S.L.

Website

https://connectatherapeutics.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

1–10 employees

Short description

Connecta Therapeutics is a Barcelona-based clinical-stage biotech developing first-in-class small molecule neuroplasticity modulators, with lead asset CTH120 in Phase IIa trials for Fragile X Syndrome, targeting adult and pediatric patients with CNS disorders.

Ownership category

akta.pro rank

Product category

CNS Therapeutics — Orphan Drug Development

NAICS

Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industries

Translational & Pharmacodynamic Biomarkers (MOA, target engagement, PD/PK-linked markers) (HLAAAMAD), In Vivo Discovery & Translational Models Platforms (humanized models, PK/PD, efficacy) (HLAAAIAK)

Keywords

• CNS therapeutics
• Fragile X treatment
• Neuroplasticity modulators
• Rare disease drugs
• Orphan drug development

Headquarters

HQ city

Barcelona

HQ country

Spain

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Revenue model

1. Pharmaceutical Development and Commercialization: Pre-revenue clinical-stage biotech company. Revenue will be generated through commercialization of drug candidates upon regulatory approval. CTH120 targets Fragile X Syndrome, a rare disease with orphan drug designation providing 10-year market exclusivity in the EU.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Connecta Therapeutics is a clinical-stage biotechnology company that discovers and develops AI-designed first-in-class small molecule drugs targeting neuroplasticity pathways for the treatment of central nervous system (CNS) disorders, with a primary focus on Fragile X Syndrome (FXS) and other neurodevelopmental conditions. Its lead product CTH120, a neuroplasticity modulator targeting the TrkB receptor, is advancing through Phase IIa clinical trials, with a second compound CTH122 in preclinical development for FXS and Rett syndrome.

Product overview

CONNECTA Therapeutics is a clinical-stage biotech company developing a pipeline of first-in-class small molecule neuroplasticity modulators for CNS disorders. The lead product CTH120 is a novel NCE targeting TrkB receptor for Fragile X Syndrome, currently in Phase IIa clinical trials following successful Phase I completion. A second compound, CTH122, is in preclinical development with demonstrated efficacy in FXS and Rett syndrome models. Both products utilize a network pharmacology-based approach and were designed using AI techniques.

Differentiator

Problem solved

Functional benefit

Products and services

• CTH120 CTH120 is a first-in-class small molecule neuroplasticity modulator and the company's lead clinical asset. It is a novel new chemical entity (NCE) designed using AI techniques to target the tropomyosin receptor kinase B (TrkB) receptor for the treatment of Fragile X Syndrome (FXS). It has demonstrated preclinical efficacy in rescuing dendritic pathology, improving cognitive ability, and completely restoring social abnormalities in FXS models. It has completed Phase I safety/tolerability trials in healthy adult volunteers and is currently advancing through Phase IIa trials in adult males with FXS.
• CTH122 CTH122 is a first-in-class small molecule with neuroplasticity modulation properties in preclinical development. It has demonstrated efficacy in preclinical models of Fragile X Syndrome and Rett syndrome. The compound is protected by a recently granted international patent family and represents a pipeline expansion asset within Connecta's network pharmacology platform.

Quantifiable outcome

• Phase I clinical trial completed with 76 healthy adults demonstrating CTH120 was safe and well tolerated throughout the clinical trial
• +2 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered core and minor.

• Prous Institute for Biomedical ResearchcoreStrategic or Co-development Partner · 1 July 2019Private research organization based in Barcelona, spin-off of Prous Science (acquired by Thomson Reuters in 2007). Founded Connecta Therapeutics in July 2019 as a spin-off. Dr. Josep Prous Jr. serves as Co-Founder and Chief Scientific Officer. Provides expertise in in silico drug discovery and safety technologies.
• Centre for Genomic Regulation (CRG)coreStrategic or Co-development PartnerRenowned international biomedical research centre located in Barcelona with over 400 interdisciplinary scientists. Dr. Mara Dierssen leads the Cellular and Systems Neurobiology group and serves as Research Advisor to Connecta. CRG conducted preclinical studies on CTH120 demonstrating neuroplasticity modulation and cognitive improvement. Under leadership of Mara Dierssen, CRG advances biomarker identification for FXS treatment response.
• Hospital del Mar Research Institute (HMRIB)coreStrategic or Co-development PartnerLeading biomedical research centre in Barcelona with around 700 professionals, among top ten Spanish institutions in scientific impact on healthcare. Dr. Rafael de la Torre serves as Principal Investigator and Study Coordinator. Conducted Phase I clinical trials and currently conducting Phase IIa clinical trial for CTH120 in adult males with FXS.
• Institute for Research and Innovation Parc Taulí (I3PT)coreStrategic or Co-development PartnerCERCA institute accredited by Carlos III Health Institute. Dr. Ana Roche serves as Principal Investigator. Joined Phase IIa clinical trial as strategic partner alongside CRG and Hospital del Mar Research Institute. Conducts clinical research in neurodevelopmental disorders.
• Dr. Randi Hagerman (UC Davis MIND Institute)minorOthersInternationally recognized clinician and researcher in fragile X field. Serves as Clinical Advisor and Medical Director at UC Davis MIND Institute. Distinguished Professor and Endowed Chair in Fragile X Research. Has carried out multiple targeted treatment trials in FXS and autism.

Scale indicators8 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Zynerba Pharmaceuticals: Clinical-stage biotech that developed Zygel (CBD gel) for Fragile X Syndrome and other neurodevelopmental disorders — the most direct historical competitor in FXS drug development. Highly comparable target indication and patient population, providing direct read-throughs on FXS clinical trial design and commercial viability.
• Ovid Therapeutics: CNS-focused biotech developing therapeutics for rare neurodevelopmental disorders including Fragile X Syndrome and Angelman syndrome. Directly comparable in indication focus (FXS), pipeline stage, and strategy of orphan-designated CNS small molecules.
• Marinus Pharmaceuticals: Specialty pharma developing CNS therapies for rare epilepsies and neurodevelopmental disorders (e.g., CDKL5 deficiency disorder). Comparable as a small-cap CNS rare-disease company pursuing orphan indications with similar regulatory and clinical-development profiles.

Broad incumbents

• GW Pharmaceuticals (Jazz Pharmaceuticals): Acquired by Jazz Pharmaceuticals, GW developed Epidiolex (cannabidiol) — approved for seizures associated with FXS-related conditions such as Dravet and Lennox-Gastaut syndromes. Comparable as a CNS specialty company that has reached FDA approval in FXS-adjacent indications, representing both an incumbent and an aspirational benchmark.
• Sage Therapeutics: CNS specialty biopharma developing first-in-class neuroactive therapeutics (e.g., Zurzuvae). Comparable as an established CNS specialty player pursuing novel mechanisms for underserved neurological indications, providing a model for Connecta's potential commercial trajectory.
• Acadia Pharmaceuticals: Commercial-stage CNS specialty pharma (e.g., Nuplazid for Parkinson's psychosis, DAYBUE for Rett syndrome). Highly relevant peer given DAYBUE's approval for Rett syndrome — an explicit expansion indication in Connecta's CTH122 preclinical portfolio.
• Axsome Therapeutics: CNS-focused specialty pharma with FDA-approved products for major depressive disorder and migraine. Comparable as a CNS-specialist commercial-stage company, illustrating the scaling pathway available to Connecta if CTH120 reaches approval.

Emerging players

• Anavex Life Sciences: Clinical-stage biotech developing CNS therapeutics for Rett syndrome, Alzheimer's, and Parkinson's disease. Comparable as a small-cap CNS developer pursuing neurodevelopmental and neurodegenerative indications with novel mechanisms.
• Newron Pharmaceuticals: European-based CNS specialty biopharma developing treatments for rare neurological and neuropsychiatric disorders. Comparable as a EU-headquartered CNS specialty company with a similar scale and orphan-disease strategy.
• Pasithea Therapeutics: Clinical-stage biotech developing CNS therapies for neuropsychiatric and neurodevelopmental conditions. Comparable as a small-cap CNS developer focused on underserved neurological indications, although at a generally earlier development stage.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/connecta-therapeutics
• Xx.com/ConnectaThx

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds5 records

Investors10 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Connecta Therapeutics