(BioWorld Today Via Acquire Media NewsEdge) Three months after going public on the Italian markets with a $76 million offering - so far the biotech industry's most successful initial public offering in the depressed 2008 markets - MolMed SpA, of Milan, Italy, is well under way with the first pivotal trial of its TK cell therapy in leukemia patients.

TK therapy aims to boost the chances of survival in leukemia patients undergoing stem cell transplantation with partially incompatible family donors. It involves the use of genetically engineered donor T lymphocytes, used in association with haplo-hematopoietic stem cells, to control complications arising from transplant while maintaining the anti-leukemic effects of the procedure.

If it reaches market, TK therapy essentially could increase both the number of patients who can undergo successful stem cell transplants and the number of available donors for those transplants. Right now, only about 40 percent of patients are able to be matched with fully compatible donors, and without full compatibility, patients run a greater risk of graft-vs.-host disease, a potentially fatal transplant complication.

"We're convinced that [TK therapy] dramatically improves immune constitution and protects against overall infections," MolMed President and CEO Claudio Bordignon told BioWorld Today in a telephone interview from Chicago, where he was presenting data on another product candidate at the American Society of Clinical Oncology (ASCO) meeting.

An earlier, nonrandomized Phase I/II study demonstrated "remarkable" results, Bordignon said. In that study, the TK therapy resulted in improvement in survival of patients by promoting rapid and sustained immune reconstitution.

The randomized Phase III study is designed to confirm those promising efficacy data. MolMed's goal is to "have about 200 patients randomized at 20 centers in Europe," Bordignon said, adding that the study's primary endpoint will be overall survival.

The company believes overall survival "is the best representative of benefit," he said, though it requires the lengthiest follow-up, meaning the trial likely will take about three to four years. "We hope to finish the study in 2012," though MolMed is considering adding "additional early primary and secondary endpoints," with hopes of demonstrating efficacy earlier, he said.

The TK therapy is one of the most advanced cell therapy programs aimed at stem cell transplantation, also the first to gain the approval of the conservative Italian regulatory agency AIFA.

In addition to the European Phase III study, MolMed also plans to initiate Phase I/II studies in the U.S., "so we can begin to make the product known" in the states," Bordignon said.

MolMed holds all rights to the product, which has orphan drug status in both Europe and the U.S., with the exception of the Asian market, for which it signed strategic partner Otsu, Japan-based Takara Bio Inc.

The company has planned to take the T-cell therapy all the way through late-stage development and into commercialization on its own. About 30 percent of the transplant centers handle 90 of the transplants, Bordignon said, so it would be feasible to reach those with a modest sales force.

However, the "product and technology has been receiving a lot of attention lately," he said, "so there may be a change in our strategy."

Earlier in its pipeline, the company has Arenegyr (NGR-hTNF-alpha), a vascular-targeting agent that recently generated positive Phase II data in mesothelioma and other cancer types. Data presented at last week's ASCO meeting included Phase II data showing improved overall survival and nearly doubled progression-free survival compared to historical best supportive care in 41 evaluable mesothelioma patients receiving Arenegyr as a second-line therapy.

On Friday, the European Commission granted orphan drug designation for Arenegyr in malignant pleural mesothelioma, a rare form of cancer that nearly always is caused by repeated exposition to asbestos fibers.

ASCO data from a separate Phase II study in heavily pretreated patients with colorectal cancer showed a 50 percent relative improved overall survival at six months compared to historical best supportive care in 31 evaluable patients.

While those are only single-arm studies, "it is our impression that in mesothelioma and colorectal cancer," Arenegyr demonstrated enough efficacy "to justify initiating Phase III," Bordignon said. "It is our intention to start Phase III by the end of this year."

That means the firm could have three Phase III studies up and running in 2009. And it should be well financed for a good start to all of those, thanks to its recent $76 million IPO, the best the industry has seen so far this year.

According to worldwide data from BioWorld Snapshots, only four companies have gone public in 2008, and only two of those, including MolMed, actually raised money. The other was Sunrise, Fla.-based Bioheart Inc., which gained a listing on Nasdaq in conjunction with a meager $5.8 million offering in February.

MolMed, which gained a listing on an Italian exchange, priced its offering at the lowest end of its anticipated ?2.15 to ?2.75 (US$3.39 to $4.30) per share range. Nevertheless, it was an impressive transaction, particularly given the tight European market, where many companies are avoiding the IPO market. Others briefly considered it, like Siena, Italy-based Philogen SpA, which pulled IPO plans in January citing unfavorable market conditions.

Bordignon said it helped that other companies were withdrawing their offerings. "That gave us an advantage." He also attributed MolMed's success to "a combination of different elements," including the reputation of its investors, the promising data from its products and technology and the experience of its management and staff.

Founded in 1996 as a cell therapy joint venture between Boehringer Mannheim, of Mannheim, Germany, and Science Park Raf in Milan, MolMed's ownership later transitioned to a private investment fund, and in 2004, the firm gained support from three large Italian investors - Fininvest, Hercule Finance (now H-Equity Sarl) and La Leonardo Finanziana (now Delfin Sarl).

Bordignon described the overall VC market in Italy as "fairly conservative. It's not an area where you see many high-risk types of investments, but I think the opportunity is there, and I think we'll see more of that in the future," he added. That could be true, if MolMed is any indication, given that the company itself is working in the high-risk, innovative areas of stem cells and gene therapy.

Biotech in Italy also continues to grow, and Bordignon said the number of firms has doubled in the last couple of years.

A 2008 report compiled by Italy's National Biotech Association indicated that there are 228 companies performing research and development in biotech areas, the largest group of those - 168 - working in health care. In 2006, the industry captured ?4.8 billion in revenue from sales in product in-licensing operations and ?1.3 billion in capital investments.

More than 50 biotech-related organizations are expected to attend the upcoming BIO meeting in San Diego. n

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