Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of June 17, 2013

DelMar Pharmaceuticals reported results from a phase I/II trial of VAL-083 (dianhydrogalactitol) for the treatment of recurrent malignant glioma and progressive secondary brain tumors. Of the three cohorts of patients currently enrolled, all have failed standard therapies and have no viable treatment options. The subjects received VAL-083 at a dose of 1.5mg/m; 3.0mg/m and 5.0mg/m. At these doses, 25% (2/8) of GBM patients and 17% (1/6) of secondary-progressive brain cancer patients showed stable disease or tumor regression in response to treatment. VAL-083 therapy is well tolerated in glioblastoma multiforme (GBM) and secondary-progressive brain tumor patients with no drug-related serious adverse events at doses studied to date. DelMar plans to split the protocol into two separate studies: one focusing solely on refractory glioblastoma and the other focusing on metastatic brain cancers.

Halozyme Therapeutics reported results from a phase Ib trial of PEGPH20 (PEGylated Recombinant Human Hyaluronidase) for the treatment of stage IV metastatic pancreatic cancer. The single-arm phase Ib trial enrolled 28 stage IV treatment naïve pancreatic ductal adenocarcinoma patients. The overall response rate (complete response + partial response) by RECIST 1.1 criteria was 42% (95% CI 22-62) for those treated at therapeutic dose levels of PEGPH20 (1.6 and 3.0μg/kg) as assessed by an independent radiology review (n=24). Additionally, in subjects with high levels of hyaluronan (HA), a substance found in a protective matrix that surrounds many pancreatic cancers, the overall response rate was 64% (seven of 11 subjects with available biopsy). Moreover, 43% (six of 14 subjects) saw a reduction of at least 70% in serum carbohydrate antigen 19-9 (CA 19-9), a biomarker that often correlates with tumor cell burden. Treatment was generally well tolerated, and the adverse event profile was consistent with those seen in previous studies of PEGPH20 as a single agent and as previously reported for gemcitabine alone. The most common adverse events were muscle spasms, myalgia and arthralgia (all grade 1/2). There was no evidence of new toxicities when used in combination with gemcitabine. The results support the dosing regimen at 3μg/kg, being used in the phase II trial.

Immune Response BioPharma reported results from a phase II trial of IR103 REMUNEX for the treatment of HIV. This randomized, single-blind, controlled, comparative study was conducted over 52 weeks at three clinical sites; one in Canada and two in the U.K. A total of 180 participants were randomized into four treatment groups. Groups one and four had a combined total of 80 individuals who received HAART-inclusive of NNTRI, a class of antiretroviral drug that inhibits the action of reverse transcriptase, and had an undetectable viral load. Groups two and three were each comprised of 50 individuals who had no more than seven days of antiretroviral treatment at any time prior to study entry (antiretroviral-naïve), with a viral load between 1,000 and 60,000 copies/ml. The combination of Remune with Amplivax was well tolerated with no serious adverse events reported. The study results support further evaluation of this combination as a therapeutic HIV vaccine.

Rigel Pharmaceuticals and AstraZeneca reported results from OSKIRA-2 and OSKIRA- 3, phase III trials of fostamatinib for the treatment of rheumatoid arthritis. In the OSKIRA-2 study of patients inadequately responding to disease modifying anti-rheumatic drugs (DMARDs), fostamatinib in combination with DMARDs showed statistically significant improvements in ACR20 response rates at 24 weeks in both the 100mg twice daily (bid) group and the group receiving 100mg bid for four weeks followed by 150mg once daily (qd) compared to placebo. In the OSKIRA-3 study of patients inadequately responding to methotrexate (MTX) and a single TNF-alpha antagonist, fostamatinib in combination with MTX showed statistically significant improvements in ACR20 response rates at 24 weeks in the 100mg bid group, but not in the group given 100mg bid for four weeks followed by 150mg qd compared to placebo. Fostamatinib continues to be well tolerated by patients. Based on the totality of the results, AZ has decided it will not proceed with regulatory filings and will return the rights to the compound to Rigel.