First in Human: Venture capital cash boosts Ex-Roche psychiatric assets

This is a recurring column on clinical research in the early stages of development, which is referred to as phase 1. These are treatments being used for the first time in a small number of human patients to determine safety, dosing and general pharmacological activity.

Picking up where Roche left off

Strategic changes at Big Pharma that culminate in a research pivot can yield rich rewards for the canny investor. Take AstraZeneca PLC's olaparib, which was condemned to the laboratory shelf until the arrival of CEO Pascal Soriot in 2012, when the drug was dusted off to become the blockbuster cancer medicine Lynparza.

Keen to snap up other well funded and robustly tested assets that are nevertheless no longer deemed a therapeutic priority, French venture capital group Sofinnova Partners SAS helped establish Noema Pharma AG with seed financing in 2019. Its aim was to capitalize on Roche Holding AG's move away from psychiatry in its neuroscience research to focus on areas of neurodegeneration like Alzheimer's disease and Huntington's, as well as neuroinflammation, notably multiple sclerosis.

An oversubscribed CHF54 million series A fundraising in December allowed Noema Pharma to pursue the development of four in-licensed compounds. The lead product, NOE-101, is set to start phase 2b studies for persistent seizures in tuberous sclerosis complex in the first half of this year, Chief Medical Officer George Garibaldi said in an interview with S&P Global Market Intelligence. A former head of development for neuroscience at Roche, Garibaldi said the Swiss pharmaceutical giant had already completed a number of studies, allowing Noema Pharma to go ahead with a mid-stage trial. The same compound will be tested in severe pain in trigeminal neuralgia, and the other candidates will be developed for Tourette's syndrome and other neurological indications with severe unmet need.

"It's our ambition that first, we show that we can walk and run with what we've been given by Roche," CEO Luigi Costa said. "But our ambition is becoming maybe an engine for the products that Roche has deprioritized but still wants to give a chance to ... in these orphan [central nervous system] indications."

Danish academic spin-out focuses on neuromuscular disease

A 2015 spin-out from Denmark's Aarhus University is working on a novel way of addressing neuromuscular disease focused on an ion channel called CLC-1, which may be applicable across a number of conditions including myasthenia gravis and spinal muscular atrophy. NMD Pharma A/S' research concept was given an early boost after it secured seed funding in 2016 from banner investors including the $55 billion Danish Novo Venture Fund's Novo Seeds. More recently, Roche's venture fund and INKEF Capital lent their support via a €38 million series A financing.

"While we started out asking very fundamental questions around this channel, we realized it might actually be a very useful drug target for patients that have failing communication between the motor nerve and the muscle fibers and hence they have weakness and fatigue and difficulty performing everyday life activities," CEO Thomas Pederson said.

The company's lead compound, NMD670, is a small molecule inhibitor of the muscle-specific CLC-1 that has shown promise in early animal studies. An ongoing phase 1 trial in myasthenia gravis, or MG, will read out within the next couple of months, Pederson told S&P Global Market Intelligence in an interview.

The current treatment for MG, as an autoimmune disease, is two-pronged: an attempt to recover muscle function is first addressed, and then the autoimmune symptoms are tackled. NMD Pharma's approach is to recover muscle function rapidly and efficiently by focusing on a completely new target that is only expressed in skeletal muscle. Companies including Johnson & Johnson's Janssen Biotech Inc. unit are working on addressing the autoimmune symptoms of MG. "It's very complimentary to what's being developed by other people, and also in quite an open space," Pederson said.

NMD Pharma has a portfolio of experimental compounds focused on the CLC-1 inhibitor, and is the only company to take this approach, he said.

"This mechanism could be used also in other neuromuscular diseases. Some of the diseases that we're focusing on are spinal muscular atrophy, Charcot-Marie-Tooth disease, but also diseases where there's absolutely no treatment or very little, such as amyotrophic lateral sclerosis," the CEO said.

PET tracer to diagnose Parkinson's

AC Immune hopes ACI-12589 will become the first tool to track Parkinson's disease. Source: Stockvault

Lausanne, Switzerland-based AC Immune SA announced that the first patient has been scanned with a diagnostic agent that has shown potential to detect and map deposits of a type of protein in the brain known as the hallmark for Parkinson's disease. The company, which researches and develops treatments for central nervous disease conditions including Alzheimer's disease, said ACI-12589 — a PET imaging tracer — showed promise in pre-clinical studies, raising the possibility it might become the world's first diagnostic tool ever for tracking Parkinson's.

"The unmet need for reliable, non-invasive diagnostic tools to enable precision medicine approaches in Parkinson's disease is substantial, which is why our alpha-synuclein PET tracer program is so critical for the field," AC Immune CEO Andrea Pfeifer said.

"The means to accurately detect and quantify pathological alpha-synuclein in the brain has remained elusive," Pfeifer added. "With our next-generation PET tracer candidate, we believe there is potential to change this paradigm."