Life Science Today 016 – Slow, Sickle-Cell, CRO Mergers, Praxis IPO, Wasp Venom
Originally Published as The Niche Podcast
Introduction
Welcome to The Niche Podcast – Your weekly 5-minute rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, failures in sickle-cell therapy, CRO mergers in Asia, Praxis IPO, and wasp venom.
Calm Before the Storm
It has been a relatively quiet week across the pharmaceutical world. Through the next couple of weeks Q3 reports will be rolling in from major players. Expect that to pair with the market volatility surrounding the election and stimulus talks for a degree of instability that could apply across the globe depending on politics or to specific companies. To date, many of the largest companies have reported good revenue streams and better than expected numbers, despite the pandemic.
Cyclerion Sickle-Cell Failure
Cyclerion is closing down their sickle cell disease therapeutic Olinciguat. The drug was designed to amplify nitric oxide signaling. Working through increased cGMP, the drug was expected to decrease vascular inflammation, cell adhesion, and improve local blood flow. Unfortunately, based on the results of their Phase II clinical trial, Cyclerion has decided to no longer pursue the therapy. In a public announcement Thursday, they stayed that Olinciguat was well tolerated, but did not demonstrate effects sufficient to merit further pursuit.
In a simultaneous announcement, they pivoted into pursuing IW-6463. Early results suggest the drug is well tolerated and crosses the blood brain barrier. However, it is also clear they are not quite sure what it is good for. Their planned trials across the next year include everything from rare mitochondrial disease to Alzheimer’s disease and age-related vascular dementias. Their report is a textbook scientific jargon for “I dunno, seems to do something.”
While IW-6463 may eventually become a disease specific and promising drug candidate, news of the failure of Olinciguat caused Cyclerion shares to drop 57% last week to close at $2.95/share on Friday.
Novotech & PPC Merger
Novotech and PPC Group announced a merger last week. Both companies are small to mid-sized clinical research organizations primarily focused on the Australian and Asian biotech markets. PPC Group (not be confused with the mega-CRO PPD) and Novotech are both majority owned by TPG capital. The merger makes sense combining a range of tools in the Australian and Asian (mainly Chinese) market. This should allow the now ~1500 employee company to compete in the rapidly growing Chinese clinical research industry. The details of the deal are not publicly available. As with most mergers of this kind, expect a “united front” while the companies combine, followed by a shakedown and purge as the number of top spots gets cut in half.
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Praxis Big Week (Year)
Praxis Precision Medicines had a huge week. They planned to offer 7.4 million shares at $16-18, then they offered 10 million at $17-18, then they actually sold at $19, and for a total of $190 million. Then, as soon as they hit the market the rocketed up to close at $27.80 on Friday. Not a bad week considering they pulled in $100M in venture funding in early May then another $110 in late July.
What’s driving the big bank roll? They are moving into a Phase 2/3 trial for their promising GABA A inhibitor PRAX-114. The drug is expected to work on major depressive disorder and perimenopausal depression. If effective this would be a global blockbuster. PRAX-944 is their second candidate which blocks T-Type calcium channels for the treatment of essential tremor. Finally, further back in the pipeline is PRAX-562 which is at Phase 1 trial for the treatment of genetic epilepsy.
None of these therapies are anywhere close to crossing the finish line, but I suspect the $210 million from private equity and venture increased the interest from the broader investor audience resulting in the successful IPO last week. Praxis is not the first company this year to move rapidly through funding rounds and go public and they probably won’t be the last.
Wasp Venom Antibiotics
Starting with the discovery of penicillin in 1928, antibiotics have played a critical role in shaping the last century of public health, saving literally hundreds of millions of lives. However, overuse and the natural tendency of bacteria to evolve has lead to new super-antibiotic resistant strains that can be deadly. Compounding this challenge is the low financial incentive for pharmaceutical companies to pursue new antibiotic development.
Academia has no such tethers. Indeed, studying microbial defense systems is one of the surest pathways to groundbreaking discoveries (listen to last week’s segment on CRISPR). This week we highlight a novel antibiotic discovery, this time made from wasp venom. A paper published in PNAS last week showed that a modified venom from wasps was able to dramatically decrease a bacterial infection in mice, even when compared to extant antibiotics. The finding shows that modified venoms may provide excellent targets for novel antibiotics. While the modified venom is a long way from approved for human treatment, it does suggest that combing genetic engineering with novel sources could produce a series of viable and effective antibiotic solutions. In a war of endless microbial evolution, we will need to think about antibiotics in an evolutionary way, not as a one-and-done therapy.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly 5-minute summary of top news in the biotech, clinical trials, and applied science industries. You can 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.
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Story References
https://novotech-cro.com/news/asia-pacific-has-record-year-clinical-trials-according-novotech
https://investors.praxismedicines.com/press-releases
https://www.pnas.org/content/early/2020/10/07/2012379117
Music by Luke Goodson