Life Science Today 079 – Vifor + Sanifit + Inositec, Kura, Takeda, Aadi, BioCryst, USDA

Originally Published as The Niche Podcast

Introduction

 Welcome to The Niche Podcast – Your weekly rundown of the biotech, pharma, clinical research, and life science industries. I’m your host, Dr. Noah Goodson. This week, double acquisitions in Switzerland, FDA holds and what that means, CMV therapy, rare + oncology continues, BioCryst draws millions, and deer. 

Disclaimer

The views expressed on The Niche Podcast are those of the host and guests. They do not necessarily reflect the opinions of any organizations or companies with which they are affiliated. 

Vifor Double Acquisitions

The Swiss company Vifor Pharma has announced two acquisitions. First, they are grabbing the Spanish Startup Sanifit Therapeutics for $231M and a further $192M in milestones. This move strikes a unique cord for a number of reasons. First, Sanifit is a one molecule pipeline company with SNF472 as their sole asset for the treatment of end stage kidney disease patients with calcific uremic arteriolopathy. In basic terms, SNF472 treats calciphylaxis which occurs when calcium deposits build up and block small arteries. Those with the condition have a less than 50% chance of making it a year. Sanifit not only has a really promising therapeutic that could make the fast track through the EMEA and FDA, but you heard correctly that they are a Spanish company. Spain is not well known for their biotech startup infrastructure. However, deals like this may spur more innovators and investors to craft collaborations. 

In a second announcement, Vifor is acquiring fellow Swiss company Inositec for a much more price-conscious ~$22M plus milestones. Inositec’s lead candidate INS-3001 is not unlike SNF472 in that it treats calcification disorders. However, Inositec’s asset is earlier in the clinical development stage with a phase I starting this coming week, and earlier in the treatment paradigm, targeting early-stage calcification disorders. 

The two acquisitions together position Vifor with a strong calcification disorder portfolio in development. 

FDA Holds and What that Could Mean

The FDA has placed a hold Kura Oncology’s Phase IB leukemia study after a patient passed away. The study is treating patients with relapsed or refractory acute myeloid leukemia (AML) with their investigational therapy KO-539. A temporary pause after a participant dies is common practice and does not necessarily indicate the study will be stopped. Normal practice is that an investigation and determination will be made followed by additional recommendation and updated participant consent as needed. In this case, the suspicion is that Differential Syndrome (DS), formally know as retinoic acid syndrome, is at fault. DS happens when therapies lead to cellular migration and release of inflammatory factors. The confusing suite of symptoms include unexplained fever, respiratory distress, pulmonary infiltrates, and capillary leak leading to renal failure. Untreated DS is fatal within a week, but if caught and treated with accessibly therapies like dexamethasone there are good survival curves. The real key here for Kura is to determine if DS is at fault and issue robust guidance to sites, participants, and physicians for close monitoring moving forward. Shares dropped about 30% on the news. I would say provided DS is at fault and occurrence rates remain low this likely represents a delay and not a stop in Kura’s pipeline – but things are not settled yet. 

Takeda CMV Therapy

Cytomegalovirus (CMV) is a common virus carried by 40-100% of the adult population with almost no notice. However, in severely immunocompromised individuals it can represent a major life-threatening condition. For transplant patients who are on strong immunosuppressors is one of the most common infections and a number of common CMV strains are resistant to available antivirals. Last week the FDA approved Takeda’s new oral antiviral to treat CMV, maribavir, which significantly lowered CMV infection rates compared to standard therapies. Maribavir works through suppressing CMVs ability to replicate and encapsulate. This is good news for transplant patients everywhere, and another step in the ongoing battle against evolving viruses. 

Ultra-Rare Cancer Therapy

The FDA has approved a cancer therapy developed by Aadi Bioscience to treat Malignant PEComas. With cases occurring in just 1/1,000,000, this is definitely in the ultra-rare category. The therapy, sold as Fyarro is a protein bound particle injection that acts as an mTOR inhibitor. As with other mTOR inhibitors this one will come with a list of warnings, but with survival rates negligible if untreated Fyarro certainly represents a ray of hope. In the larger context, this is another therapy chalked up to rare or oncology approvals and looking at pipelines, I don’t think this will stop any time soon. 

BioCryst $350M Raise

BioCryst raised $350M by selling $50M in equity to Royalty Pharma, along with $150M for tiered royalties of ORLADEYO. OMERS Capital Markets also purchased $150M for tiered Royalties. We don’t often speak about royalty raises, so as a reminder this capital model has investors return driven by the sales of therapy by providing cash to drive further investment or commercialization. Investors may earn 3% of sales up to 1.5B and tier down from there, or whatever the specific deal stipulates. ORLADEYO is an approved therapy in multiple regions to treat hereditary angioedema (HAE) attacks through a once daily oral therapy. In this case, BioCryst is leveraging this capital to expedite their existing Factor D pipeline and ensure there are further products moving towards approval. 

Virus in Deer 

In case you missed the news, the USDA is also interested in COVID19, but this time it’s in the white-tailed deer population. A pre-print from Penn State University and a publication from the department of agriculture agree that White Tailed deer across the United States are developing COVID19. The studies detected both COVID19 RNA and COVID19 antibodies respectively. Collectively the data suggest that while not homogenous COVID19 has spread to populations and may be linked to human-deer interactions. Now all of this is important because as the global pandemic continues to evolve with variants like Omicron appearing this week, there will be a wide array of COVID19 reservoirs where the virus may move to another species and then migrate back. There is not indication at this time if COVID adversely impacts the deer or if it can travel from deer to humans, but longitudinal data shows it certainly arrived in their population from us. The main take away is that it’s likely COVID in one form or another will be with us for a long time to come. Another take away might be that it’s quite reasonable that COVID arrived in humans from another species. 

Closing Credits

Thanks for joining me on The Niche Podcast; your weekly summary of top news in the biotech, pharma clinical research, and life science industries. You can learn more at thenichepod.com or find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.