3 Gene Therapy Biotechs to Watch Out For
The approval of some of the first CAR-T therapies recently, has further exploded the field of immune-oncology and gene therapy. Writing for The Motley Fool, Cory Renauer has taken a look at the top three immune-oncology companies that are likely to increase in value.
The first of which is Gilead Sciences, who had their drug, Yescarta approved by the FDA on 19th October, which was developed by Kite Pharma.
Although its undecided if Yescarta will be able to earn back the $11.9 billion investment, Renauer explained, “New cancer therapies often cost more than $100,000 for a year of treatment, and once you factor in hospitalisation expenses for patients who don’t respond, Gilead’s $373,000 list price for a single Yescarta infusion could save end payers a bundle in the long run. Enough patients are eligible for Yescarta that peak annual sales of the therapy could top out around $2 billion if private insurers and other end payers agree with Gilead’s value proposition.”
If not, the company remains in a good position, as the stock value is likely to increase, even if CAR-T becomes mundane. The company’s market cap is 8.1 times the $13.4 billion in free cash flow its already generated this last year.
In addition, although Bluebird bio has not yet had any approved products, its current development strategy is one to get excited about. Its lead candidate is Lenti-D, a gene therapy to treat cerebral adrenoleukodystrophy (CALD). In trials, 15 of 17 CALD patients receiving the treatment had no major disabilities. Working in its favour the company also has CAR-T compounds in partnership with Celgene. Trials have showing an impressive 100% response rate inn 15 advanced-stage multiple myeloma patients treated with bb2121. Celgene has announced that it will pay for mid- to late- stage clinical development, and Bluebird will receive significant milestones and royalty patments if any of them make it to market.
Renauer commented, “Although Lenti-D might not become a blockbuster drug on its own, it could go a long way toward funding development of a similar gene therapy candidate with far more potential. LentiGlobin could be a huge improvement that allows huge swaths of people with sickle-cell disease and beta-thalassemia. Peak sales estimates for the therapy, which allows patients to produce functional haemoglobin, run as high as $4 billion annually and there’s still more.”
Lastly, AveXis is a gene therapy company that you should definitely have eyes on. On 3rd October is announced that is planned to present top-line data as of 7th August from its Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint Malo, France. An overview suggested that all patients were alive and event-free at 20 months of age. Nine of the 12 patients could sit unassisted for 30 seconds or more, and 92 percent has sustained CHOP-INTEND score more than 40 for a mean of 18.8 months.
Patients with SMA are usually unable to swallow, and mostly lose the ability to breathe unassisted before they turn one year of age. Renauer added, “With results this impressive, Biogen has plenty to worry about. Thr big biotech launched the first ever SMA treatment at the beginning of the year, and sales of the drug are already on pace to reach $1 billion in its first year. AveXis intends to begin a trial designed to support an application before the end of 2017, which means Spinraza sales should be safe for a few more years.”
Renauer suggests that if AVXS-101 continues demonstrating positive data, Beiogen or another company might buy AveXis. However, one limitation for AveXis is that AVX-101 is its sole candidate in clinical trials. In turn, it has a greater potential than Gilead, but if AVXS-101 fails, then the company ultimately won’t be worth much.