SAN DIEGO, April 8, 2015 -- Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, today announced it plans to proceed with a Phase 3 clinical study (Study 301) of its investigational new drug PEGPH20 in patients with metastatic pancreatic cancer, using a design allowing for potential marketing application based on either progression free survival (PFS) or overall survival.
The use of PFS as the basis for marketing approval will be subject to the overall benefit and risk associated with PEGPH20 combined with nab-paclitaxel (ABRAXANE®) and gemcitabine therapy, including the:
- Magnitude of the PFS treatment effect observed;
- Toxicity profile; and
- Interim overall survival data.
The study will enroll patients whose tumors accumulate high levels of hyaluronan (HA) – a sugar that is sometimes more prevalent in the areas surrounding cancer cells. Halozyme recently discussed the study as part of a planned Type B meeting with the U.S. Food and Drug Administration (FDA).
Data discussed at the meeting focused on interim results from the company's randomized Phase 2 study in pancreatic cancer, Study 202, which showed a doubling in median PFS in metastatic pancreatic cancer patients with high levels of HA who were treated with Halozyme's investigational new drug PEGPH20 combined with nab-paclitaxel (ABRAXANE®) and gemcitabine (9.2 months vs. 4.3 months in patients treated with nab-paclitaxel ABRAXANE® and gemcitabine alone). The potential risk profile, including rate of thromboembolic events, was also discussed.
Additional takeaways from the meeting include:
- Halozyme affirmed plans to enroll and evaluate high-HA patients using a companion diagnostic test; and
- FDA provided feedback on the current companion diagnostic approach and confirmed that an approved companion diagnostic strategy is required prior to Phase 3 study initiation.
"We are encouraged by the FDA's feedback and their willingness to consider a novel primary endpoint such as PFS given the high unmet medical need in high-HA metastatic pancreatic cancer," said Dr. Helen Torley, President and Chief Executive Officer. "We are committed to rapidly finalizing the Phase 3 protocol and developing the companion diagnostic regulatory filings required to initiate Study 301."
Based on this FDA feedback, the company is targeting the end of first quarter 2016 to initiate the Phase 3 study.
About Study 202
Study 202 (Halo 109-202) is a Phase 2 multi-center, randomized clinical trial evaluating investigational new drug PEGPH20 as a first-line therapy for treatment of patients with stage 4 metastatic pancreatic cancer. The primary outcome of the trial is to measure improvement in progression-free survival in patients receiving investigational new drug PEGPH20 in combination with gemcitabine and nab-paclitaxel compared to gemcitabine and nab-paclitaxel alone. A second primary endpoint will assess the thromboembolic event rate in the PEGPH20 treatment arm. Secondary endpoints also include objective response rate and overall survival. More information may be found at: http://oncologytrials.halozyme.com/pancreatic/
PEGPH20 is an investigational PEGylated form of Halozyme's proprietary recombinant human hyaluronidase under clinical development for the systemic treatment of tumors that accumulate hyaluronan.
FDA granted orphan drug designation to PEGPH20 for treatment of pancreatic cancer and fast track for PEGPH20 in combination with gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. Additionally, the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency, designated investigational drug PEGPH20 an orphan medicinal product for the treatment of pancreatic cancer.
Halozyme Therapeutics is a biotechnology company focused on developing and commercializing novel oncology therapies that target the tumor microenvironment. Halozyme's lead proprietary program, our investigational drug PEGPH20, applies a unique approach to targeting solid tumors, allowing increased access of co-administered cancer drug therapies to the tumor. PEGPH20 is currently in development for metastatic pancreatic cancer and non-small cell lung cancer and has potential across additional cancers in combination with different types of cancer therapies. In addition to its proprietary product portfolio, Halozyme has established value-driving partnerships with leading pharmaceutical companies including Roche, Pfizer, Janssen and Baxter for its drug delivery platform, ENHANZE™, which enables biologics and small molecule compounds that are currently administered intravenously to be delivered subcutaneously. Halozyme is headquartered in San Diego. For more information, visit www.halozyme.com.
Safe Harbor Statement
In addition to historical information, the statements set forth above include forward-looking statements (including, without limitation, statements concerning the possible activity, benefits and attributes of PEGPH20, the possible method of action of PEGPH20, its potential application to improve cancer therapies and statements concerning planned additional studies, regulatory filings and other future actions relating to the development of PEGPH20) that involve risk and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. The forward-looking statements are typically, but not always, identified through use of the words "believe," "enable," "may," "will," "could," "intends," "estimate," "anticipate," "plan," "predict," "probable," "potential," "possible," "should," "continue," and other words of similar meaning. Actual results could differ materially from the expectations contained in forward-looking statements as a result of several factors, including unexpected expenditures and costs, unexpected results or delays in development and regulatory review, regulatory approval requirements, unexpected trial results, unexpected adverse events and competitive conditions. These and other factors that may result in differences are discussed in greater detail in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 2, 2015.