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This Week in Biotech: Clinical Success, Plus Wheeling and Dealing

By Sean Williams - Jan 25, 2014 at 11:13AM

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Two positive clinical results, a symbiotic asset deal, and a rumored takeover target are this week's top stories in Part 2 of "This Week in Biotech."

I don't know about you, but to me, between the J.P. Morgan Healthcare Conference two weeks ago and a flurry of earnings and trial data, it seems like an exceptionally furious start to the year for biotech stocks.

This marks our third week in a row that the biotech wrap-up needed to be split into two separate articles because of an inordinate number of investor-pertinent stories.

In Part 1 of "This Week in Biotech," we examined how five regulatory rulings from the Food and Drug Administration and the CHMP, the European Medicines Agency's advisory panel, generally made for a tough week in the sector. In Part 2, though, we're going to look more closely at two unmistakably positive clinical studies, one promising deal, and one rumored deal which helped balance out this week's negative news.

Beaming with BEMA
We had to wait all week for a truly exciting move, but shareholders in BioDelivery Sciences (BDSI) were rewarded for their patience, as it and partner Endo Health Solutions on Friday reported positive top-line results from their late-stage trial of BEMA buprenorphine in opioid-naive patients with moderate to severe chronic pain. What you need to know here is that BEMA buprenorphine met its primary endpoint of a reduction in daily average pain numerical rating scores from the baseline. However, there is still an additional phase 3 trial being run. For BioDelivery Sciences, however, the positive late-stage trial will result in a $10 million milestone payment from Endo Health Solutions, which will help the company with ongoing research and development costs.

Off to a good start
While still wet around the ears, Karyopharm Therapeutics (KPTI 9.07%) delivered positive data at the American Society of Clinical Oncology's 2014 Gastrointestinal Cancers Symposium on two pipeline products. First, Karyopharm reported its phase 1 data of selinexor (previously KPT-330), an experimental metastatic colorectal cancer drug. According to its phase 1 study, selinexor was well tolerated and produced one RECIST partial response, as well as 11 cases of stable disease, out of 35 patients.

Karyopharm's other presentation was based on a novel, oral p21-activated kinase (PAK4) inhibitor being examined as a possible pancreatic cancer treatment in preclinical studies. According to Karyopharm's press release, its PAK4 inhibitor demonstrated "anti-proliferative activity and tolerability," which to me would be the key steps needed to be witnessed prior to filing for an investigational new drug application. While certainly another great week for Karyopharm, I still have my reservations about its current valuation.

A symbiotic deal
With biotech valuations soaring, deal-making has been happening with regular occurrence of late -- and last week was no exception. Life sciences company Repligen (RGEN 1.97%), which once actively pursued a product portfolio of histone deacetylase inhbitors, or HDACis, announced on Wednesday that it will out-license its HDACi portfolio to BioMarin Pharmaceutical (BMRN 0.00%).

Under the terms of the deal, Repligen will get a $2 million payment upfront and have the potential to earn up to $160 million in regulatory and development milestones. In addition, Repligen will get the opportunity to dump its HDACi portfolio off to a company that might be able to do something with it so it can instead focus on expanding its bioprocessing business. For BioMarin, the deal makes perfect sense as it's looking to secure its leading role in rare disease drug development. Best of all, both companies made this deal work with minimal risk for either party.

The rumor mill is working overtime
Finally, the rumor mill put in quite a few hours of overtime late in the week, as Friday saw the highly volatile Ariad Pharmaceuticals (NASDAQ: ARIA) trade a whopping 134 million shares and rise nearly 20% after London's Mail newspaper reported that the company may be a takeover candidate. According to the report, Eli Lilly, GlaxoSmithKline, and Shire are all possible purchasing candidates, with Eli Lilly seen to be in the lead -- and willing to pay as much as $20 per share to boot!

As I noted yesterday, Eli Lilly is desperate to find pathways to growth with its pipeline subjected to a mound of generic competition over the coming years and a number of its late-stage products failing to meet their primary endpoint. Ariad offers attractive points, such as its strong cash position and carry-over tax losses, which could be viewed as a dangling carrot for a larger buyer.

Conversely, though, Ariad's leukemia drug, Iclusig, is reeling since its two-year follow-up study revealed a rise in the number of thrombotic events (i.e., blood clots) in patients taking the drug, compared with patients on Iclusig at the 11-month follow-up. It could be incredibly difficult, even with more stringent labeling, to get physicians to prescribe Iclusig domestically and abroad when high-risk adverse events are a possibility. I would suggest not getting caught up in these rumors and instead listen carefully to how Ariad plans to re-establish confidence in Iclusig, which will be no easy task.

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.

The Motley Fool recommends BioMarin Pharmaceutical. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

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Stocks Mentioned

BioMarin Pharmaceutical Inc. Stock Quote
BioMarin Pharmaceutical Inc.
$95.07 (0.00%) $0.00
BioDelivery Sciences International, Inc. Stock Quote
BioDelivery Sciences International, Inc.
Repligen Corporation Stock Quote
Repligen Corporation
$251.35 (1.97%) $4.86
Karyopharm Therapeutics Inc. Stock Quote
Karyopharm Therapeutics Inc.
$5.41 (9.07%) $0.45

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

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