Welcome to your Update from the Clinic for the month of January. Pluristem and OncoMed reported preliminary efficacy data from early-stage trials, while the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) granted a number of Orphan Drug Designations for various cancer indications being developed by OncoMed, Stemline Therapeutics, and Kite Pharma.
Pluristem (PSTM), which is investigating its placenta-derived (PLX-PAD) cells as a means to repair muscle injury following total hip arthroplasty, posted some promising results. The cell therapy easily hit its primary endpoint, boosting the force of gluteal muscles in the operated leg by a remarkable 40 times relative to placebo. Patients in the treatment group received 150 million PLX-PAD cells. The study was conducted in Germany between the Musculoskeletal Surgery & Julius Wolff Institute and Berlin-Brandenburg Center for Regenerative Therapies.
OncoMed (OMED) presented preliminary safety and efficacy data from its Phase 1b ALPINE clinical study in frontline metastatic pancreatic cancer. Tarextumab, which targets the Notch 2/3 receptors, was administered in combination with gemcitabine and Abraxane®. Patients had median progression-free survival (PFS) and median overall survival (OS) of 5.6 and 11.6 months, respectively. Clinical impact appears to be slightly more positive in patients with elevated Notch expression, who received an incremental benefit in PFS and OS of one and three months, respectively. The company announced dosing of its first patient with a bi-specific antibody (anti-DLL4/VEGF) in a Phase 1 in patients with solid tumours and enrollment of its first biomarker-selected patient in a Phase 1 investigating an anti-Notch 1 antibody in solid tumours.
2014 was a rough year for OMED, which saw nearly 40% of its market cap evaporate. The stock surged early in the year after its announcement of a multi-billion deal with Celgene in December 2013, which provides Celgene co-marketing rights for six of OMED’s cancer stem cell programs in the United States and a lead role in commercialization elsewhere. However, the share price slowly slipped away in subsequent months and was hit further on news, in June, that the company would have to halt clinical studies investigating vantictumab and a fusion protein, FZD8-Fc, due to safety concerns. The studies were re-initiated in the fall but the company’s share price has failed to recover.
Orphan Drug Designations are all the rage these days. While the markets are much smaller, pricing and reimbursement are much more favourable (for the time being), especially for rare genetic disorders and uncommon cancers where few, if any, treatment options are available. This past month the FDA granted an orphan designation to Tarextumab for the treatment of pancreatic and small cell lung cancer and another to Stemline Therapeutic’s (STML) SL-401 for glioma. Kite Pharma (KITE), which is developing engineered T cells for cancer indications, had one of its products, KTE-C19, designated an orphan medicinal product by the EMA for the treatment of diffuse large B cell lymphoma.
Keep an eye out for clinical data from Fate Therapeutics (FATE), which is expecting to have clinical read-outs from its adult PUMA study and pediatric PROMPT study, both investigating PROHEMA (ex vivo programmed hematopoietic cells) for hematological malignancies. The company should also have top line data from its pediatric PROVIDE trial this year investigating PROHEMA in rare, inherited metabolic disorders.
Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit http://ccrm.ca/industry-consortium.