Life Science Today 047 – FDA, AstraZeneca, BioVie, Pfizer, Vaccitech, Werewolf
Originally Published as The Niche Podcast
Introduction
Welcome to The Niche Podcast – Your weekly rundown of the biotech, clinical research, and life science industries. I’m your host, Dr. Noah Goodson. This week, the FDA slaps wrists and takes names, Farxiga expands FDA approval, BioVie makes cash-light acquisition, Pfizer targets fungus, and one IPO down, another to go.
FDA Out to Slap Wrists
Acceleron Pharma has received a notice of non-compliance from the FDA for failure to post a summary of clinical study data. The warning requires Acceleron to post results within 30 days or face a $10,000 fine (not a big deal) and criminal prosecution (a very big deal). This is critical as it is the first such notice the FDA has dispatched and sends a message of changing tides around this aspect of clinical trial transparency.
Since 2007, when conducting IRB approved clinical trials on human subjects in the US, responsible parties are required to submit a summary of the trial design to clinicaltrials.gov, a national database. Additionally, the federal law requires that studies submit a summary of findings to the same database within 1 year of completing the trial. While the first half, submitting a summary, is pretty well followed, a brief browse through the database will reveal the second half, submitting a summary of results, is rarely ever met with timely compliance.
Last summer, in the midst of about 10,000 other things the FDA was doing, they released guidance (linked in show notes) on their new procedures for identifying and notifying companies of non-compliance with reporting clinical trials results. Multiple companies received pre-notices, but Acceleron is the first to earn an official notice. The nature of this procedure highlights the concerns of the FDA.
Acceleron was running a phase 2 trial on dalantercept, the now abandoned oncological product, combined with axitinib, approved in 2012 for renal cell carcinoma. The trial failed to meet a primary endpoint and dalantercept was abandoned. In fact, the results were published in a 2019 paper in Cancer. So while the failure to submit to clinicaltrials.gov? In this case, I’m not sure, but it may have just fallen through the cracks. This move by the FDA may be just a slap on the wrist, but it is also an indication to the whole field that this “box to check” is also a critical part of trial transparency and companies will be held accountable. The notice was given April 27th. Results from Acceleron were submitted April 28th, according to clinicaltrials.gov.
AstraZeneca Earns FDA Approval to Treat Chronic Kidney Disease with Farxiga
Farxiga has received FDA approval for a critical new indication. It was originally approved in 2014 to help with glycemic control, then last May it was approved to treat heart failure. Farxiga has now received the nod to treat certain types of Kidney disease. The expanded indications come on the back of a placebo controlled clinical trial showing a roughly ~40% reduction in the number of patients progressing to major kidney failure. The same trial showed a significant reduction in hospitalization and death from heart failure or cardiovascular disease.
Farxiga is the first SGLT2 inhibitor to receive approval to treat chronic kidney disease. This approval does not include a variety of genetically generated causes of kidney disease. The approval for heart conditions saw a major increase in sales of Farxiga last year. In fact, AstraZeneca sales of Farxiga rose ~30% to just shy of $2B in 2020. With this latest indication and the intersectionality of its approved treatments, Farxiga will continue to see really robust sales increases across 2021 and beyond.
BioVie Make Cash-light Acquisition
BioVie has acquired all of the assets from NeurMedix, a privately held clinical stage pharmaceutical company. The deal gives NeurMedix $3M at closing and $7.3M after a primary endpoint for a lead drug is met. But the real brunt of the purchase comes from 8.3M new shares of BioVie. At roughly $17/share that’s in the $141M value. This is a very cash-low acquisition for both parties in this space. The really heavy money is all backloaded. In fact, the agreed upon milestones start at $350M for pivotal trial end-points, and pass $1.2B for FDA approval, plus long-run sales milestones. So the deal ends up being a ~$150M upfront, plus ~$3B in milestones. But the structure of it keeps the cash exchange very low for both companies out the gate.
BioVie’s biggest get from the deal is NE3107, which is recruiting patients for a phase III trial to treat Alzheimer’s. While many Alzheimer’s drugs come and go, there are aspects of this specific medication that make me personally excited, for example its potential role in decreasing insulin resistance rather than focused on the consistent dead-end of targeting amyloid plaques. Trial enrollment begins May 3rd, but it’ll likely be end of 2022 or later before we see meaningful results. The biggest surprise to me is that pound-for-pound this is one the cash-lightest deals I’ve seen in the space in a year. On the surface, it’s not a bad deal for either company, but it also reminds me more of the deals I see when markets are tight and companies run out of liquid assets: A distinct contrast to the slues of money we see flying about in capital markets recently.
Sponsor
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Pfizer Wants to Stop the Fun(gi)
Pfizer has acquired Amplyx for an undisclosed amount. Amplyx lead candidate, Fosmanogepix is a potentially first in-class novel anti-fungal. With no new anti-fungal’s on the market in 20 years and fungal evolution continuing, the need for novel therapeutics is a globally significant concern. Amplyx has shown reasonable safety data thus far as both an oral and intravenous formulation. Pfizer was a partner in Amplyx $90M series C in May 2020, and the company had a valuation of $189M at the time. In addition to their lead candidate Amphyx has early-stage antiviral and antifungal’s in their pipeline that likely fall into this portion of Pfizer’s broad strategic approach to the field. Since the deal comes so close after the Series C and Amplyx probably isn’t out of money, my guess is their valuation was higher than in May 2020.
One IPO Complete, Another on the Outsized Docket
Vaccitech was one of the essential partners in the development of AstraZeneca vaccine. Their role in the development of this vaccine has been by and large overlooked, compared to other super-partners, for example BioNTech with Pfizer. From what I can tell, they will benefit relatively little from the global roll-out of the vaccine by AZ. That being said, a good name is hard to come back, and in March they raised $168M series B. Last week they snuck in and closed a $110M IPO. The cash gives them the power to pursue additional products within their pipeline including oncological and infectious disease solutions. If this sounds like a diverse portfolio for a vaccine developer that’s because it is. It’s possible the size and subtlety of this IPO is related to more than just a divergent pipeline. Mixed messages globally on the AZ vaccine may have damped enthusiasms for additional press coverage. Regardless of global opinions or corporate strategy, they’re now in a position to pursue meaningful development of their next product.
Warewolf Therapeutics has announced they are going public in an upsized $120M IPO. Like Vaccitech, this comes close on the heels of the $72M series B from January. Warewolf has a unique pipeline of pro-inflammatory immune modulators aimed at the oncology space. They predominately rely on the conditional activation of Interleukins as well as a solution focused on Interferon alpha which is all very cool. In the flood on oncological approaches out there, they absolutely stand out as having a less common approach with potential unique value to certain tumors. But I think we can agree the most salient and important aspect of Warewolf’s IPO is that they will be listed on the Nasdaq under the ticker HOWL.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly summary of top news in the biotech, clinical trials, 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.
Sponsors
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Story References
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Music by Luke Goodson
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