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Spectrum Pharma submits Captisol-enabled Melphalan NDA to US FDA
San Diego | Tuesday, December 30, 2014, 14:00 Hrs  [IST]

Ligand Pharmaceuticals Incorporated, a biopharmaceutical company, announces that its partner Spectrum Pharmaceuticals has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Captisol-enabled Melphalan (CE-Melphalan) HCl for injection (propylene glycol-free) for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma. Spectrum is also seeking approval for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.

According to Spectrum Pharmaceuticals, the Captisol-enabled Melphalan NDA was filed under a 505(b)(2) application and Spectrum expects FDA review to take approximately 10 months. Spectrum plans to launch this drug with its existing hematology/oncology sales force next year pending approval.

“We congratulate the team at Spectrum Pharmaceuticals on their development progress,” said John Higgins, Ligand’s president and chief executive officer. “With a specialty sales force already in place, we look forward to Spectrum’s potential launch and commercial success with this valuable asset.”

Ligand licensed global development and commercialization rights to CE-Melphalan to Spectrum in 2013. Spectrum assumed the responsibility for a pivotal clinical trial and was responsible for filing the NDA. Under the license agreement, Ligand received a license fee and is eligible to receive revenue from Captisol material sales, milestone payments, as well as royalties on net sales following potential commercialization.

According to previous announcements by Spectrum, the phase 2 pivotal trial evaluating CE-Melphalan was a multicenter trial evaluating safety and efficacy. The primary objective of the study was to determine the overall safety and toxicity profile in multiple myeloma patients receiving 200 mg/m2 of CE-Melphalan as myeloablative therapy prior to autologous stem cell transplantation (ASCT). The secondary objectives evaluated the efficacy of CE-Melphalan in this patient population as measured by Multiple Myeloma Response Rate (according to International Myeloma Working Group [IMWG] criteria), and the rates of myeloablation and engraftment. The primary as well as secondary endpoints of this Phase 2 trial were met.

In a previous clinical study, CE- Melphalan met the requirements for establishment of bioequivalence to the currently approved commercial intravenous formulation of melphalan. This demonstrated bioequivalence of CE-Melphalan may facilitate the use of this new more stable, propylene glycol-free Captisol formulation of melphalan following the potential approval of the NDA.

Captisol-Enabled, Propylene Glycol-free Melphalan is a novel intravenous formulation of melphalan being investigated for the multiple myeloma transplant setting, for which it has been granted an Orphan Drug Designation by the FDA. This formulation eliminates the use of propylene glycol, which has been reported to cause renal and cardiac side effects that limit the ability to deliver higher doses of therapeutic compounds. The use of the Captisol technology to reformulate melphalan also improves its stability and is anticipated to allow for slower infusion rates and longer administration durations, potentially enabling clinicians to safely achieve a higher dose intensity for pre-transplant chemotherapy.

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled six FDA-approved products, including Onyx Pharmaceuticals’ Kyprolis®, Baxter International’s Nexterone and Merck’s NOXAFIL IV. There are more than 30 Captisol-enabled products currently in clinical development.

Ligand is a biopharmaceutical company with a business model that is based upon the concept of developing or acquiring royalty revenue generating assets and coupling them to a lean corporate cost structure.

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