Date: Feb 26, 2014 Source: bizjournals (
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Feb 26, 2014, 11:52am EST Updated: Feb 26, 2014, 2:49pm EST
Dyax's new long-lasting rare disease drug shows early promise
Gustav Christensen, president and CEO of Dyax Corp.
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Courtesy Dyax
Gustav Christensen, president and CEO of Dyax Corp.
Julie M. Donnelly
Reporter- Boston Business Journal
Dyax Corp. shares were trading up this morning following positive Phase 1 data for its new drug candidate to prevent attacks for patients with the rare disease, hereditary angioedema. The company already sells a drug, called Kalbitor, to combat acute attacks of the disease.
In its first human study, the drug DX2930 was found to be well-tolerated at all four dose levels with no adverse events.
If approved, it could be a game-changer for both the Burlington, Mass.-based company, and for patients with HAE, which causes extreme swelling that can be life-threatening if it affects the larynx. The drug candidate is a monoclonal antibody, which means it forms a tight bond with the target it is attacking, and does little collateral damage to the surrounding tissues.
"If you look at other diseases with good preventive medicines — such as Humira for rheumatoid arthritis or Tysabri for multiple sclerosis — they are monoclonal antibodies," Dyax CEO Gustav Christensen said in an interview. Both of those drugs are blockbusters.
One of the benefits of monoclonal antibodies, Christensen said, is a long half-life. DX2930, if approved, would only have to be given twice per month to prevent HAE attacks. Current preventive products on the market, such as Cinryze (owned by Shire plc) have to be given at least twice per week. In addition, the Dyax product could be given under the skin as opposed to intravenously like Cinryze.
The news is just the boost the company needs. Its approved product, Kalbitor, only brought in around $40 million last year. One reason is that there are only about 6,500 diagnosed cases of HAE. But another is that Kalbitor must be given by a doctor or nurse. Meanwhile, Kalbitor's competitor for acute attacks, Firazyr (also made by Shire) can be self-administered.
Dyax (Nasdaq: DYAX) had $111M of cash on hand as of Sept. 30, 2013 and executives said that was enough money to get past the planned Phase 2 trial for the experimental medicine, which will launch next year and produce data in 2016. Dyax is hoping the FDA will accept the Phase 2 trial as the basis for an application for approval, but won't get a decision on this from the agency until next year.
One factor that may help Dyax win over the FDA is that the disease is not well-controlled on current preventive drugs. For instance, Christensen noted that the Phase 3 trial for Cinryze showed a 52 percent reduction in attacks. This leaves plenty of room for improvement, he said, especially with a disease where an attack can be fatal.
The positive data read-out led Leerink Swann, a boutique Boston life sciences bank, to raise its one-year price target on Dyax to $12 from $8.
Dyax also has two other tricks up its sleeve. Firstly, its second revenue stream, derived from licensing out its drug discovery technology to big pharma companies, is kicking into high gear. A number of drug candidates have reached late stage trials. One experimental oncology therapy made by Eli Lilly & Co., ramucirumab, has been sent to the FDA as a potential medicine for gastric cancer. It has an approval date in the second quarter of this year, and if approved, it could bring in $1 billion per year. If so — and its still a big if — Dyax would stand to make about $25 million per year in royalties for 10 years.
Secondly, it's possible the science behind Dyax's HAE therapies can be expanded to help control swelling and inflammation for patients with other conditions, such as Crohn's disease, ulcerative colitis, psoriasis, diabetic macular edema, and rheumatoid arthritis. Christensen said the company is working on a biomarker diagnostic test which could help identify if the same target causing HAE attacks is causing swelling for these patients with other inflammatory diseases.
Dyax shares were trading at $10.77 just before noon today, up 6 percent from $10.14 at the previous close.
