Regeneron has held high hopes for evinacumab, a rare cholesterol disorder drug that earned a first-in-class nod from the FDA early last year, as a centerpiece of its cardiometabolic franchise. Now, the company is tapping an ultra-rare specialist to take evinacumab to the next level abroad.
Regeneron will receive $30 million in upfront cash and a potential $63 million in downstream milestones from Ultragenyx for ex-US licensing rights to Evkeeza (evinacumab), an ANGPTL3 blocker with an FDA approval to treat a rare cholesterol disorder alongside LDL-C lowering therapy and diet, the companies said Friday.
Ultragenyx, a small pharma known best for its ultra-rare disease drugs, will pick up development and commercialization costs for evinacumab in patients with the condition known as homozygous familial hypercholesterolemia (HoFH), which is believed to affect anywhere between 1 in 160,000 and 300,000 people worldwide.
In addition, Regeneron and Ultragenyx have set preliminary terms for an ex-US licensing deal for one of Regeneron’s investigational drugs, an antibody designed to treat the ultra-rare disease fibrodysplasia ossificans progressiva (FOP). Terms for that potential partnership have yet to be set.
Evinacumab was approved last February at the hefty price tag of an average of $450,000 per year. It was the first drug approved to inhibit ANGPTL3, or angiopoietin-like 3, a protein involved in lipid metabolism. In pivotal data backing the approval, patients taking evinacumab saw a 47% reduction in LDL levels from baseline, compared to a 2% increase in the control. The drug arm also saw significant reductions in levels of apolipoprotein B, an indicator of vascular disease, as well as triglycerides and overall cholesterol.
Evinacumab was envisioned as a sort of natural follow-up for Regeneron’s Praluent, a partnered PCSK9 blocker with Sanofi approved to lower LDL-C. That drug has been locked in a yearslong pricing battle with Amgen’s Repatha in what has effectively become a war of attrition over price decreases in the pricey PCSK9 class.
Ultragenyx, meanwhile, scored its first FDA approval some five years ago with Mepsevii, a drug approved to treat an ultra-rare genetic enzyme disorder called MPS VII. Since then, the drugmaker has earned its stature as an ultra-rare specialist, bringing two additional drugs onto the market in Crysvita, approved to treat two forms of hypophosphatemia, and Dojolvi, an engineered medium-chain fatty acid designed as a supplement for patients with molecularly confirmed long-chain fatty acid oxidation disorders.
Their Staying Power Lies in their Patient-Centricity
Decentralized clinical trials (DCTs) were traditionally utilized in an isolated fashion prior to the COVID-19 pandemic. To continue their research within the constraints of the pandemic, sponsors and clinical investigators pivoted to a decentralized model out of necessity. At the onset, regulatory agencies offered some guidance on the digital approaches that are acceptable to ensure DCT approaches are applied in a way that maintains patient safety, as well as data quality and integrity.
Exscientia CEO Andrew Hopkins and Sanofi CEO Paul Hudson
Drug R&D has for years had an abysmal track record of success, with the vast majority of drug candidates never making it to market. The promise of AI to shorten the discovery time for new drugs and up their chances of success has more big drugmakers buying in — and Sanofi is the latest.
Sanofi will pay $100 million upfront with a potential $5.2 billion in downstream milestones for access to up to 15 small molecule drugs from Exscientia, a red-hot UK deep learning company at the forefront of the so-called ‘AI-discovered’ drug R&D movement, the partners said Friday.
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A district court in Texas will likely tie up the FDA’s FOIA office for months, as the court ruled late Thursday that the agency must release all documents related to its review of Pfizer-BioNTech’s Covid-19 vaccine.
The order from district judge Mark Pittman, handed down late Thursday, notes that while the Court recognizes the ‘unduly burdensome’ challenges that this FOIA request may present to the FDA, there also ‘may not be’ a more important issue at the FDA right now than the pandemic, the Pfizer vaccine, getting every American vaccinated, and making sure to the American public that the process was not rushed.
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Scott Clarke, Ambagon Therapeutics CEO
The saying goes necessity is the mother of invention — and few areas of drug development are in greater need than oncology. A new startup is now repurposing promising technology to tackle the hardest-to-drug proteins, and a suite of big-name backers are buying in.
Ambagon Therapeutics launched Friday with an $85 million A round and a world-class group of researcher-founders building what are known as ‘molecular glues’ to crack the code on a class of elusive cellular proteins, the biotech said.
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Cytokinetics stumbled a bit with its closely watched lead heart drug over the last year or so, losing two pharma partners after missing a key secondary endpoint in a Phase III study. But things are looking up in 2022, as Royalty Pharma is reaching a little deeper into its wallet to bolster that program and another heart candidate.
Royalty Pharma has agreed to lend up to $300 million to support the potential commercialization of Cytokinetics’ lead candidate, omecamtiv mecarbil, and development of its other heart program, aficamten. The cash will come in five tranches, including an initial tranche of $50 million upon closing and four others upon certain regulatory and clinical milestones. Each tranche has an interest-free and payment-free period of six calendar quarters, followed by 34 calendar quarters of installment re-payments totaling 1.9 times the amount drawn.
In the year-plus since Roger Perlmutter left his post leading Merck’s R&D efforts, the Big Pharma’s new leadership has continued to pursue an aggressive dealmaking strategy. On Friday, Merck enlisted a new partner as the spree shows no signs of slowing down.
Merck signed a pair of collaborations with Absci aiming to bolster its AI research capabilities, the companies announced Friday. The first is a relatively modest deal on Merck’s end — there are no details about the upfront or milestone payments but the duo said it could lead to more.
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Eric Easom, CEO of AN2 Therapeutics
After starting up with a $12 million Series A in 2019, California outfit AN2 Therapeutics issued its first press release in over 2 years — and its second ever: The ultra-quiet biotech has raised an $80 million Series B and added a pair of directors to boot.
The biotech is headed by several leaders from Anacor, which was bought out by Pfizer in 2016 for about $5.2 billion. Eric Easom, former head of Anacor’s R&D on neglected diseases, is co-founder and CEO. Other Anacor vets include AN2’s chief development officer, CFO and head of biology.
New year, same Fujifilm Diosynth.
The CDMO giant hit the ground running at full speed this year, announcing it will expand its BioProcess Innovation Center in Research Triangle Park, NC. The move will double its existing laboratory footprint in the Tar Heel State, and add another 145 skilled jobs to the site by 2024. Another 89,000 square feet will be added, which will allow for a more robust commercial process.
Aligos Therapeutics took a beating Thursday after reporting it is stopping development on its lead program for chronic hepatitis B.
The biotech’s shares $ALGS closed down 57% and fell into penny stock territory Thursday, following a morning press release saying Aligos’ ALG-010133 program did not prove efficacious at the dose tested in a Phase I study. Additionally, the company concluded that higher doses were also unlikely to be effective, and ultimately decided to axe the candidate altogether.
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https://endpts.com/regeneron-hands-off-ex-us-rights-for-cholesterol-disorder-drug-to-rare-disease-specialist-ultragenyx
