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Daily Brief: Karuna raises $42m for novel CNS approach, Chen exits Roche, Orchard gains GSK board member

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‘Game changing CNS’ company Karuna raises $42m

A Boston-based start-up has raised $42m from investors for its potentially ‘game changing’ approach to developing new treatments for CNS conditions.

Karuna Pharmaceuticals is focused on targeting muscarinic receptors for the treatment of disorders marked by psychosis and cognitive impairment, and has raised the money in a Series A financing round, including the issuance of $22 million in shares upon conversion of debt into equity.

Participants included ARCH Venture Partners, the Wellcome Trust, Steven Paul MD, PureTech Health, and other undisclosed investors.

Karuna will use the financing to advance its lead product candidate, KarXT (Karuna-xanomeline-trospium chloride), including the launch of a phase II trial in patients with schizophrenia in the third quarter of 2018. It also plans to expand into other therapeutic areas, including a non-opiate pain indication.

Its KarXT molecule aims to avoid the safety issues that have given existing antipsychotic medicines a bad name, these include tardive dyskinesia, metabolic dysfunction, weight gain, glucose intolerance, sedation and others problems.

KarXT is designed to improve tolerability and unlock the potential of muscarinic cholinergic receptor agonists that selectively target M1/M4 muscarinic cholinergic receptors in the brain while blocking their activation in tissues outside the brain.

Karuna is currently completing a second phase 1 trial using a proprietary co-formulation of xanomeline, a muscarinic agonist Karuna exclusively licensed from Eli Lilly which showed compelling efficacy in patients with schizophrenia and Alzheimer’s disease but had peripheral cholinergic side effects, combined with trospium chloride, a muscarinic antagonist that acts only in the periphery (outside of the brain and central nervous system).

The initial phase 1 trial demonstrated a significant and clinically meaningful reduction in pre-specified cholinergic side effects with KarXT compared to xanomeline alone. The phase II trial in patients with schizophrenia is designed to replicate and expand on previous efficacy and safety data with xanomeline in schizophrenia and Alzheimer’s disease

“Karuna has all of the characteristics we look for in a game-changing CNS company. Its lead drug candidate has a unique mechanism of action for treating both psychosis and cognition, as well as an exciting non-opiate application for pain. These are therapeutic indications where there is a profound need for new treatments. We are excited about the clinical data that have already been generated, and we look forward to helping to drive the next chapter of growth,” said Robert Nelsen, Co-Founder and Managing Director of ARCH Venture Partners.

Andrew Miller

“We greatly appreciate the support of our investors and their confidence in the potential of KarXT to be the first new and highly differentiated medicine in over 50 years for the treatment of psychosis and cognitive impairment in psychiatric and neurological disorders,” said Andrew Miller, PhD, co-founder and chief executive officer of Karuna (pictured).

“We believe KarXT could be a promising new treatment that safely and effectively addresses the debilitating positive, negative, and cognitive symptoms of patients living with schizophrenia and other central nervous system disorders.”

Karuna is part of a portfolio of companies overseen by the London-listed, Boston-based PureTech Health. Headed up by CEO Daphne Zohar, the group specialises in novel approaches to drug delivery and the emerging science behind what it calls the ‘cross talk’ of the Brain-Immune-Gut Axis.

Orchard

GSK’s licensing head joins Orchard Therapeutics board

UK-based gene therapy company Orchard Therapeutics has added Jon Ellis, Ph.D, GSK’s head of science & technology licensing, to its management board.

Dr Ellis joins the board as GSK’s designated representative following the deal struck earlier this year in which Orchard acquired the big pharma company’s gene therapy portfolio.

“Jon brings a unique combination of business development expertise and deep understanding of emerging technologies in the field of gene and cell therapy. We believe Orchard will greatly benefit from his advice as the company continues to build a world-leading pipeline of gene therapies for rare diseases,” said Mark Rothera, president and CEO of Orchard.

Dr. Ellis has more than 25 years in the pharmaceutical and biotech industry, leading a variety of R&D activities including drug development projects and strategic business development with a particular focus on complex technologies and emerging therapeutic modalities. At GSK, Dr. Ellis led several strategic transactions in the gene therapy field. Dr. Ellis holds Master of Arts and Ph.D. degrees from the University of Cambridge, and an MBA from Henley Management College. He is an inventor of more than 35 patent families.

“GSK conducted an exhaustive process to identify the best company to place its gene therapy programmes for rare diseases,” Dr Ellis commented. “Orchard impressed us with its commitment and capabilities. I am delighted to be joining Orchard’s board of directors and look forward to working with the board and executive team to deliver commercially approved gene therapies to patients across the world.”

Roche’s immunology leader Chen leaves for biotech start-up

Dan Chen

The global head of cancer immunotherapy development at Roche/Genentech, Dr Dan Chen, is to leave the company to join a biotech start up, IgM Biosciences.

Dr Chen (pictured) is one of the industry’s leaders in cancer immunotherapy, and oversaw the development of Roche/Genentech’s checkpoint inhibitor Tecentriq to market.

Now he’s jumping ship to join IGM Biosciences, a new biotech firm focused on developing the next generation of immunoglobulin based monoclonal antibodies therapies.

He becomes chief medical officer at the privately-held firm, and joins alongside ex-Janssen biologics research chief William Strohl, Ph.D., who has been appointed to the company’s board of directors.

“Dan and Bill are both pioneers in novel antibody development, and they each bring a wealth of experience and knowledge that will help direct and accelerate the progress of our groundbreaking IgM antibody platform and help guide our lead candidates into and through the clinic,” said Fred Schwarzer, Chief Executive Officer of IGM Biosciences.

Immunoglobulins, also known as antibodies, comprise a family of proteins that occur in five major forms: IgM, IgD, IgG, IgE and IgA.

Monoclonal antibodies have be based around IgG until now, but the biotech firm believes they can unlock the potential of greater binding and potency in IgM antibodies.

“IGM’s proprietary scientific and technological approach represents an exciting new frontier for the development of antibody treatments for cancer and other serious diseases,” commented Dr. Chen.

“The company’s IgM antibodies have unique advantages over traditional IgG antibodies in that they offer stronger binding to more difficult and rare targets, as well as superior agonist potency.”

He added: “As a clinician who has been immersed in the immuno-oncology space through what could be described as its most dramatic period of growth, I see tremendous potential in the translation of IGM technology to the clinic.”

Andrew McConaghie
2nd August 2018
From: Research
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