The American Society of Hematology (ASH) is one of those scientific meetings that usually flies below the radar of the wire services and big newspapers. Still, this year’s event in San Francisco has drawn more than 21,000 physicians, scientists, and intensely interested folks from biotech companies and Wall Street. They are all scouting new treatments for diseases of the blood, like leukemias, lymphomas, myelomas, and every clotting disorder you can shake a stick at.
If you don’t think this sounds like a big deal, check out the sales of anti-clotting drug clopidrogel (Plavix): $4.7 billion worldwide for Bristol-Myers Squibb and Sanofi-Aventis in 2007, even while they faced competition from cheaper generics. Then there’s old ASH standbys like Genentech and Biogen Idec’s rituximab (Rituxan) for non-Hodgkin’s lymphoma, which eclipsed $2 billion in U.S. sales last year and keeps growing.
So, I’ve been keeping an eye on the next big thing for the blood. Here are the newsiest stories we saw cross our biotechnology desk in our three-city network of Boston, San Diego, and Seattle:
The Bothell, WA-based biotech company (NASDAQ: SGEN) caused a stir with its “empowered” antibody drug, which has the tumor-targeting ability of an antibody loaded with a potent toxin to give it extra tumor-killing kick. The drug, SGN-35, was able to completely wipe out aggressive forms of Hodgkin’s disease and related lymphomas with minimal side effects. In a study of 44 patients who were seriously ill and relapsed after a median of three prior rounds of chemotherapy, the Seattle Genetics drug caused tumors to completely disappear or mostly go away in more than one-third of patients (38 percent).
This data is promising enough that Seattle Genetics is preparing to go straight to pivotal studies for Hodgkin’s disease and anaplastic large cell lymphoma in early 2009, which could lead this drug to be FDA approved. Seattle Genetics CEO Clay Siegall said investigators at the ASH meeting are itching to participate in the pivotal studies. “There’s a lot of buzz here,” Siegall said during a break, when he called me yesterday. “The investigators are excited. We’ve never had this much excitement in the 10.5 year history of Seattle Genetics. The doctors really want to get involved in the trial.” He adds that there’s a backlog of patients who could enroll in the next studies, and he’s hopeful that they can be recruited to sign up quickly.
Millennium: The Takeda Oncology Company
Millennium Pharmaceuticals had a breakout performance at last year’s ASH. The Cambridge, MA-based biotech company, now the cancer division of Japan-based Takeda Pharmaceuticals, showed last year that bortezomib (Velcade) could cause complete remissions for 35 percent of patients with multiple myeloma on a combination treatment with its drug, compared with 5 percent who did that well on the combination alone. This year, it showed follow-up data from the trial, called Vista, that showed that early glimpse of promise is translating into an ability to help patients live longer.
This San Diego-based biotech company (NASDAQ: LGND) saw some positive data reported by its partner, GlaxoSmithKline. The drug giant reported that eltrombopag (Promacta) had a long-term benefit for patients with a rare bleeding disorder called immune thrombocytopenic purpura (ITP). This drug is vying for FDA approval in the U.S., and aims to compete with Amgen’s romiplostim (Nplate). The Glaxo/Ligand alternative would be the only one that can be taken as an oral pill, instead of as an injection.
The Glaxo/Ligand drug showed it was able to boost counts of platelets in the blood, which help form clots. This led to less bleeding, and helped wean patients off steroid medications that carry nasty side effects when taken chronically over a long period of time. Patients who got the drug were eight times more likely to have sustained healthy platelet counts over six months than those who got a placebo.
“We believe reduction in the use of other medications is a particularly important finding that will serve to increase its utilization in other indications like HCV, if approved,” said Christopher James, an analyst with Rodman & Renshaw in New York, in a note to clients.
This Cambridge, MA-based company said its experimental drug romidepsin reached its goal in a Phase II clinical trial of patients with cutaneous T-cell lymphoma. Researchers at Stanford Cancer Center found 30 of 72 patients (42 percent) had their tumors completely or partially go away. A second study sponsored by the National Cancer Institute supported the finding with a 40 percent tumor response rate. The company anticipates filing an application by year’s end with the FDA to begin marketing the product. About 1,500 people are diagnosed with cutaneous T-cell lymphoma in the U.S. each year, according to The Leukemia & Lymphoma Society.
Seattle-based ZymoGenetics (NASDAQ: ZGEN) said its sole marketed product was able to be safely given to patients with excess surgical bleeding, without provoking an immune system reaction. The study was of 205 patients who were at increased risk for having reactions against the standard product derived from cow blood, marketed by King Pharmaceuticals as Thrombin-JMI.
The study found that none of the patients developed immune reactions against the ZymoGenetics drug, marketed as Recothrom, which is made differently, through genetically engineered copies of a human clotting protein. The product has gotten off to a slow start in the marketplace, and is expected to generate $7 million in sales this year, the company has said.
Seattle-based Cell Therapeutics (NASDAQ: CTIC) has a drug that’s working well for patients with non-Hodgkin’s lymphoma. The question is whether they can raise enough money to commercialize it, long after most investors have written the company off, driving its stock down to pennies a share.
At the ASH meeting, researchers said ibritumomab tiuxetan (Zevalin) was able to cause long-lasting remissions in a clinical trial of 409 patients. Among patients who had their tumors completely wiped out, patients on the Zevalin group stayed in remission for a median time of at least 67 months, compared with 31 months for patients in a control group.
The world’s largest biotech company, with research and development operations in Seattle and Cambridge, MA, reported some follow-up data on its drug for the bleeding disorder, ITP.
The company found that 160 out of 215 patients (74 percent) were able to boost their platelet counts to a healthy level to stop bleeding. The average treatment period was almost a year and a half (76 weeks). The platelet counts were sustained at that level or higher for at least a year in 41 percent of patients, the company said.
Almost one-third of patients (29 percent) had serious adverse events in the trial, and about seven percent of those were considered drug-related, the company said. The most common side effects were headache, contusions, and fatigue.
“We believe these data support the long-term use of Nplate for adult chronic ITP patients,” said James, the analyst with Rodman & Renshaw. He forecasts it will generate $424 million in sales in 2012, its fourth year on the market.
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