Atlas Molecular Pharma closes a EUR 3 million round of funding to advance clinical development of a drug to treat congenital erythropoietic porphyria

Atlas Molecular Pharma (Atlas), a spin-off of the CIC bioGUNE research centre - a member of the BRTA - and set up with the aim of developing new pharmacological chaperones for the treatment of rare metabolic diseases, has completed a EUR 3,000,000 round of funding, with the public body CDTI and the EZTEN Venture Capital Fund of the SPRI Group (Basque Business Development Agency) becoming company shareholders. The round of funding is led by the Donostia-San Sebastián-based asset management company Inveready, with the participation of the company's current partners.

Atlas, a company founded in Bizkaia in 2015, is headed by Óscar Millet, PhD in organic chemistry, and Joaquín Castilla, PhD in molecular biology. Atlas' lead candidate, ATL-001, acts as a pharmacological chaperone by stabilising and recovering the function of UROIIIS, the protein which, when defective due to inherited mutations, causes the accumulation of toxic porphyrins and, consequently, congenital erythropoietic porphyria. ATL-001 has showed promising results in representative animal models and has been granted orphan drug designation by the US Food and Drug Administration (FDA).

Orphan drugs are developed for treating rare diseases. Traditionally, and for financial reasons, such drugs have not been of interest to the pharmaceutical industry, as they are targeted at a small group of patients. However, these drugs address essential public health needs. In this context, the Basque Government has included the study of rare diseases as one of the strategic areas of its smart specialisation programme (RIS3), promoted by the European Union.

Congenital erythropoietic porphyria is an ultra-rare, severe and debilitating pathology, with no therapeutic alternative on the market and no drugs under development. ATL-001 is designed to meet this need as a proposed therapeutic approach that would change the course of the disease at its source.
With this investment, Atlas will now begin a Phase I study and a Phase II clinical trial, both of which will be conducted in the United States, to assess the effectiveness of the drug in patients with congenital erythropoietic porphyria. Following the completion of this study, the aim is to access the FDA's accelerated approval programme to bring ATL-001 to patients and deliver it to market.

Congenital erythropoietic porphyria is a rare disease affecting less than one person per million population. The disease is caused by a deficiency in the activity of the enzyme uroporphyrinogen III synthase, which acts in the heme synthesis pathway, leading to a shortened red blood cell lifespan (anaemia) and extreme photosensitivity, blistering and increased bacterial infections of the skin. Symptoms may appear during childhood or, in less severe cases, in adulthood. Existing treatments only relieve the symptoms and there is no effective curative therapy.

Oscar Millet, CEO of Atlas, explains: “Congenital erythropoietic porphyria is a very rare but also extremely debilitating disease, with a high demand for curative treatments. In addition to the potential direct benefit to patients, the strategy devised at Atlas for this disease can be easily extrapolated to other rare diseases”.
Preclinical results obtained with ATL-001 are promising and significantly improve the disease phenotype. The company will now move on to clinical development with the aim of delivering a therapeutic alternative to the market for patients.
Advisory services for this operation have been provided by Venture Tech Audit, Hoffmann Eitle and RCD.

About Atlas

Atlas Molecular Pharma, with its headquarters in the Bizkaia Science and Technology Park, develops cutting-edge therapies for the treatment of rare diseases, including congenital erythropoietic porphyria, prion diseases and Tyrosinemia type I. The company currently has a staff of eight employees who follow an open innovation model as part of a network of academic collaborators and other companies in the sector. The key challenge for Atlas is to move the congenital erythropoietic porphyria project forward to its clinical phases as soon as possible.

About CIC bioGUNE

The Centre for Cooperative Research in Biosciences (CIC bioGUNE), located in the Bizkaia Technology Park, is a biomedical research organisation conducting cutting-edge research at the interface between structural, molecular and cell biology, with a particular focus on the study of the molecular bases of disease, for use in the development of new diagnostic methods and advanced therapies.

About EZTEN FCR

EZTEN FCR is a venture capital fund managed by Gestión de Capital Riesgo del País Vasco, a company belonging to the SPRI Group - The Basque Business Development Agency.

EZTEN FCR invests in innovative Basque companies, especially in the areas of bioscience, advanced manufacturing and clean energy, with a view to promoting their development.

About Inveready

Inveready, based in Donostia-San Sebastián, is one of the leading venture capital managers in Spain.

Inveready currently invests in technology start-ups through 4 specialised investment vehicles (Digital Technology, Life Sciences, Venture Debt and Hybrid Debt and Equity for listed companies) and has more than EUR 400 million of assets under management. Over the past 11 years, the fund has completed successful divestments in more than 20 companies, some of which have been bought by multinationals such as Intel, Symantec and Red Hat (IBM), whilst others have been listed on public markets such as the IBEX 35, NASDAQ or MAB. Empowering the ambitions of bold entrepreneurs has become part of the DNA of the company. As a consequence of this growth and trajectory, Inveready is proud to have received several awards, including the ASCRI awards for the best venture capital manager in Spain, the best venture capital operation and the best debt operation.

About the CDTI

The CDTI is the organisation of the Spanish government that supports knowledge-based innovation, advising on and offering public support for innovation through subsidies or partially repayable grants. The CDTI also internationalises the R&D and innovation business projects of Spanish companies and organisations, and manages Spanish participation in international R&D&I programmes and networks such as Horizon 2020 and Eureka, and in the Science and Space industries. In addition, through the Innvierte Economía Sostenible initiative, it supports and enables the capitalisation of technology companies.