Cyclerion’s olinciguat flunks sickle cell test; Nimbus nets $60M; Dyno-Roche’s gene therapy pact; RayzeBio debuts with $ 45M to target tumors

Posted on Oct 15, 2020 by Delveinsight

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Cyclerion flunks sickle cell test, prompting pivot to CNS disease

Cyclerion Therapeutics has terminated the development of olinciguat in sickle cell disease after getting a look at data from a phase 2 trial. The setback caused Cyclerion to switch its focus to IW-6463 and to treat central nervous system diseases.

Ironwood Pharmaceuticals spinout Cyclerion took olinciguat into phase 2 in sickle cell disease on evidence strength; it intensifies nitric oxide signaling. By increasing signaling, Cyclerion anticipated olinciguat would cut the proportion of sickled cells while simultaneously improving blood flow and endothelial integrity, thus alleviating pain, fatigue, and other sickle cell symptoms.  

The vascular sGC stimulator flunked. Cyclerion is yet to show data from the 70-subject phase 2 that compared four doses of olinciguat to placebo, declaring that the results did not demonstrate the adequate activity to bolster further internal clinical development.

Nimbus reels USD 60 Million to propel two lead programs through the clinic

Nimbus Therapeutics is reeling in USD 60 million and signing on two new investors, RA Capital Management and BVF Partners.

To prove that Nimbus can go beyond the acetyl-CoA carboxylase inhibitor it developed that was taken up by Gilead Sciences for USD 400 million in 2016, it will have to propel programs into the clinic and maintain a discovery engine, which can keep turning up promising drugs.

The proceeds will push the Nimbus’ lead program, a Tyk2 inhibitor for psoriasis, into a phase 2 study, as well as a second program, an HPK1 blocker, into the clinic in 2021. It will also proceed with several preclinical prospects, including four that Nimbus revealed in June and others it is still keeping under wraps.

The four recently revealed programs include those targeting CTPS1, an enzyme that plays a role in immune activation, and AMPKβ2 regulates metabolic homeostasis in many tissues. The other two aim at WRN and Cbl-b, both immuno-oncology targets.

Dyno, Roche to tackle CNS, liver disease in gene therapy pact  

Gene therapies are the worthy areas of investment in the biotechnology world, as are artificial-intelligence-assisted research methodologies, and “Dyno is at the intersection of these two things,” as said by CEO Eric Kelsic, Dyno. The potential partners have been trying to get in on the action since the company’s founding. Now, Cambridge, Massachusetts-based Dyno shows its third partnership in the last five months: a deal with Roche and its Spark Therapeutics unit to develop a central nervous system (CNS) and liver-directed gene therapies.

The agreement could exceed USD 1.8 billion in clinical and sales milestones, but the companies remained silent about how much Dyno’s getting in an upfront fee and research funding.

A big issue with gene therapies is ensuring the payloads are delivered effectively and safely through their delivery vehicles. Traditionally, adeno-associated virus (AAV) vectors have restrictions as they are challenging to manufacture, can only carry a limited payload, and can only aim at certain tissue types. The gene therapies delivered by AAV may not work in people who have developed a natural immunity to this type of virus. The second challenge lies in creating new capsids, or the protein shell, which coats viral vectors and targets cells. 

This is where Dyno’s AI-powered approach comes in to play. They do experiments to garner information about that space, then use machine learning, build an app, and use those machine learning models to navigate to the most exciting areas of that space.

RayzeBio debuts with USD 45 Million to target tumors 

Radiation is one of the most widely used and effective cancer therapies that is traditionally administered through external beams targeted at a tumor site. Ken Song, M.D., CEO of the newly unveiled company RayzeBio, calls it an incredibly useful mode for killing cells and bets that his San Diego-based startup can make it even better with a more targeted approach.

RayzeBio announced a USD 45 million Series A financing backed by lead investors VenBio Partners and Versant Ventures and joined by Samsara BioCapital, to advance a traditional concept and an older modality of radioisotopes and move it to the forefront. Rayze already has seven active programs underway; all focused on targeted radiopharmaceuticals.

External beam radiation makes eradicating cancer cells difficult. The concept of radiopharmaceuticals is bringing the radiation inside the body and straight to the cell.

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