The 26-year pipeline, CMDO divestment, oncology, and movement among COVID19 Therapies
Originally Published as The Niche Podcast.
Find out more at
https://LifeScienceTodayPodcast.com
Story References
https://tinyurl.com/Niche-073-1
https://tinyurl.com/Niche-073-2
https://tinyurl.com/Niche-073-3
https://tinyurl.com/Niche-073-4
https://tinyurl.com/Niche-073-5
https://tinyurl.com/Niche-073-6
About the Show
Life Science Today is your source for stories, insights, and trends across the life science industry. You can expect highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It’s news, with a dash of perspective, focused on the life science industry.
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, the 26-year pipeline, CMDO divestment, oncology, and movement among COVID19 Therapies.
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.
Ultra-Rare Disease Win for Enzyvant
Rare diseases do not have a universal definition, instead a range of global institutions provide varying standards from somewhat rare to ultra-rare. These range from diseases that may have only a few individuals globally to just being relatively rare. Some locations include considerations like total disease burden in their qualification of “rareness.” All of this is important because most regulatory agencies have alternative pathways for regulatory approval if a disease qualifies as rare. In the case of the FDA, any disease with a total population of 200,000 or less individuals in the US is considered rare, compared to EU where rareness is a ratio of 5 out of every 10,000 people. In both cases, a rare disease flag qualifies therapies for specialized guidance, reduced approval requirements, and in some cases financial support via grants and other resources.
Rare diseases are actually becoming common targets for biotech companies. This because of a combination of regulatory pathways leading to decreased financial requirements to complete clinical trials, and significant advancements across science that make rare-disease treatments a viable option. It is important to remember that just because a given disease is rare, does not mean rare diseases are. In fact, they are quite common with over 7000 diseases impacting as many as 30M people in the United States alone.
This week the FDA approved Enzyvant’s therapy RETHYMIC for the treatment of the ultra-rare disease Congenital Athymia. Caused by genetic mutations, Congenital Athymia is a deadly disease in which people are born without a thymus. The Thymus is a small gland located between the upper portion of lungs and is critical for the regulation of T-cells in the immune system. People born without a Thymus spend most of their life in the hospital with extensive supportive care and typically die at 2 or 3.
The absolutely incredible efforts to launch RETHYMIC began in 1993. The treatment involves a one-time regenerative therapy used to reconstruct the Thymus in Congenital Athymia patients using engineered human tissue. After more than 25 years and 10 clinical trials, the FDA has granted approval to RETHYMIC as a treatment. The survival curves for this therapy are…. Amazing. Without the treatment the curve hits 0 before the age of 4. With the treatment patients have lived as far as 26 years (the length of the study so far). This is not a cure all, but a 75% chance at a long life is a huge difference from a 0% chance.
Particularly impressive to me is the work over nearly 3 decades to see this therapy become a reality.
Rare Divestment in CDMO Space by Charles River Labs
It is rare to see divestments in the contract manufacturing and development (CDMO) space. In fact, over the last couple of years we’ve covered major investments in Korea and North America as well as consolidations and acquisitions. That’s because securing stable pipelines is a significant and ongoing challenge for therapy manufacturing.
Charles River laboratories is divesting two CMDO facilities. A gene therapy manufacturing plant in Sweden is being sold for $52M and a facility in Japan is being sold for $63M. Why sell two profitable CMDO plants? I think the answer is that it was always part of the plan. Charles River acquired the plants as part of the $875M acquisition of Cognate Bioservices back in March. My guess is that these facilities duplicate existing services covered by Charles River and the redundancy is irrelevant to their long-term growth model. While this does get chalked up to a rare divestment, I wouldn’t say this is part of a trend. More like the normal post-acquisition chopping block.
Merck Continues Roll
I promise this is not a Merck-cast. But the company (MSD outside US) makes top headlines again this week for multiple moves. First, remember that little drug we’ve mentioned once or twice on this show, Keytruda? Well it earned another approval this week. But I’d rank this as a more noteworthy approval. Keytruda with Chemotherapy is now approved by the FDA as a first-line treatment for patients with PD-L1+ cervical cancer tumors. This is the first first-line treatment for this condition approved in 7-years and continues the seemingly endless successes for the monoclonal antibody.
The same week, Merck has submitted their COVID-19 Antiviral therapy, molnupiravir, co-developed with Ridgeback Bio to the FDA for approval. It’s not surprising to see this moving forward quickly and I expect the FDA to have a rapid response for emergency use authorization. There are some concerns around pricing for the medication in the United States. Merck has already agreed to deliver 1.7M doses to the US government at $712/course. Sounds fine for a lifesaving treatment. Except experts are saying it costs less than $20 to manufacture. Now Merck is not all evil Pharma here. They are licensing out manufacturing to several Indian companies who plan to provide a supply globally at close to $12 for the 10 pills taken over 5 days during the course of treatment. Merck themselves says they plan to produce 10M courses by the end of 2021.
Regeneron Stays in the Game
Regeneron was the first company with a meaningful therapeutic to treat COVID19. Their antibody cocktail REGEN-COV has been used significantly under FDA emergency use authorization. Now, Regeneron has a date with destiny, in the form of an April 13th review date for full FDA approval. Regeneron is also delivering 1.4M doses to the US government by the end of 2021 under the EAU but with new products emerging their lead-therapeutic position may not be sustained. If Merck’s new oral treatment is as effective as anticipated, it’s possible that could take a significant chunk out of Regeneron’s long-term profits. But with antibody cocktails infusions generally posing higher risks, switching to an antiviral may not be all bad. I think the most likely course is that Merck’s product because the mild-case at-home standard of care and Regeneron’s antibodies are for more serious cases. But we’ll have to wait and see what the data say.
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.