Global pharma start-up success stories

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From AI to 3D deep-learning research to biologics, Lu Rahman selects some interesting global start-ups pushing the boundaries in drug discovery.

Owkin

French-American startup, Owkin is focussed on using artificial intelligence (AI) to discover and develop better treatments for unmet medical needs, starting with the fight against cancer. Using AI, the company identifies new drug candidates, de-risks and accelerates clinical trials and builds diagnostic tools that improve patient outcomes.

The company was co-founded by Thomas Clozel, a former assistant professor in clinical onco-haematology, and Gilles Wainrib, a pioneer in the field of machine learning in biology, in 2016. Owkin has raised over $300 million and became a unicorn through a $180 million investment from Sanofi in November 2021.

The company recently announced that it has entered into a multi-year, strategic collaboration with Bristol Myers Squibb to apply its AI capabilities to design potentially more precise and efficient clinical trials for Bristol Myers Squibb. The collaboration will initially focus on cardiovascular diseases and has the potential to extend into projects in other therapeutic areas.

As part of the collaboration, data from a network of academic medical centres and machine learning techniques will be used to enhance clinical trial design and execution with AI-powered approaches that optimise endpoint definitions, patient subgroups, and treatment effect estimation with covariate adjustment and external control arms. This new collaboration follows years of collaboration between Owkin and Bristol Myers Squibb, including a number of successful projects to identify biomarkers and improve clinical trial outcomes with covariate adjustment, using real world data.

Gilles Wainrib, Co-founder and Chief Scientific Officer at Owkin, says: “Our collaboration will see cutting-edge machine learning methods used to maximise opportunities for patients to benefit from the latest treatments as quickly and safely as possible. We are excited to start this collaboration with Bristol Myers Squibb, a global leader in cardiovascular with a strong development pipeline which has the potential to change the lives of millions of patients.”

According to Wainrib the collaboration could prove transformational for patient outcomes and scientific progress, making machine learning solutions an integral part of the clinical trial process.

Charm Therapeutics

CHARM Therapeutics is a 3D deep-learning research company discovering and developing transformational medicines. Based on the discoveries of David Baker in protein-folding, CHARM Therapeutics has developed a proprietary technology based on the first rapid, accurate protein/ligand co-folding algorithm. The company’s plan is to use these insights to address challenging targets in cancer and other disease areas and discover and develop new drugs against these targets.

Founded by David Baker and Laksh Aithani (below), the company has just announced a $50M Series A financing and is pioneering end-to-end 3D deep-learning to discover and develop transformational medicines against previously hard-to-drug targets. The company’s proprietary platform, DragonFold, applies expertise in protein-ligand co-folding to deliver transformational medicines targeting challenging molecular targets in cancer and other disease areas.

“3D deep-learning on macromolecular configurations represents a new era in drug discovery. The work of David Baker in predicting highly accurate three-dimensional structures of proteins offers potential to explore new frontiers in medicine, approaching disease targets previously considered undruggable. CHARM Therapeutics has been created to combine these breakthroughs in 3D deep-learning with excellence in drug discovery and development,”says Gary Glick, Executive Chair of CHARM Therapeutics. “Due to the end-to-end nature of DragonFold, it has the potential to simultaneously determine the binding site and model induced fit as well as predict the pose  and affinity of the ligand, thus unlocking the true potential of structure-based drug design,” says Laksh Aithani, Co-Founder. “We are advancing this technology and its application to the hit discovery, hit-to-lead and lead optimisation processes of small molecule drug discovery.” The company is building a robust engine to translate these 3D predictions into novel small molecule therapeutics that modulate challenging targets, initially in cancer, with the potential to transform the lives of patients.

Elektrofi

Boston-based Elektrofi’s founding team of MIT engineers and scientists combined deep expertise in rocket science and biochemistry to focus on the development and delivery of ‘ultra-high’ concentration biologics to meet the unmet need for easier, less burdensome and more cost-effective ways to administer these therapies – and in the process redefine the standard of biologic drug delivery by transforming the patient experience.

Recognising that protein therapeutics have contributed significantly to the treatment of cancer, rare diseases and other chronic conditions, Elektrofi aims to reduce the amount of time biologic drug delivery takes from hours to minutes.

The company recently announced the completion of a $40 million Series B financing round. A portion of the proceeds will be used to establish a centralised GMP manufacturing line in support of multiple upcoming clinical trials. The company will also plan to double headcount over the next 12-18 months to accelerate development across a growing pipeline of licensed programmes with leading pharma and biotech.

Elektrofi’s formulation platform addresses the long-standing and unmet need for stabilised, highly concentrated biologics. Based on a proprietary microparticle technology, the platform enables patient-friendly small-volume injections for monoclonal antibodies, therapeutic proteins, and other large molecule drugs that are of growing importance to human health.

“This financing sets the stage for advancement of multiple lead programmes to human trials, clinical validation of Elektrofi’s high concentration technology, and pipeline expansion in response to significant inbound platform interest,” says Chase Coffman, CEO and Co-founder, Elektrofi. “We view this as a foundational step on our journey to making a meaningful difference for patients and their loved ones.”

Volume 23 – Issue 3, Summer 2022

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