The Clinical Catch-Up for Monday, July 1

syringe depositing liquid into petri dishes

Exiting the last week of June and heading into July, a number of companies announced results from ongoing clinical trials. Here’s a look.

Bristol-Myers Squibb announced that its Opdivo (nivolumab) did not meet its primary endpoint in unresectable hepatocellular carcinoma (HCC). The drug was being compared to Bayer’s Nexavar (sorafenib). The drug failed to meet statistical significance for overall survival (OS). However, the company is putting a positive spin on it, saying it showed a clear improvement trend in OS for patients treated with Opdivo compared to sorafenib. Sorafenib is the current standard of care for HCC.

Krystal Biotech announced positive results from its Phase II trial of KB103 in patients with severe generalized dystrophic epidermolysis bullosa (RDEB). DEB is an incurable skin blistering disease caused by a lack of collagen in the skin. KB103 is a replication-defective, non-integrating viral vector engineered using the companys’ STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. Five out of six wounds treated with the therapy closed 100% during the trial. The remaining one had been a deep wound open for more than four years. It closed 35% at the 30-day measurement s and 42% at the 90-day measurement.

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Axsome Therapeutics launched GEMINI Phase III trial of AXS-05 in major depressive disorder. It enrolled the first patient in the trial. The drug is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. Topline results are expected in the second of this year. The drug received the FDA’s Breakthrough Therapy Designation in March. About 300 patients with a confirmed diagnosis of moderate to severe MDD will be randomized 1:1 to AXS-05 or placebo for six weeks.

Alnylam Pharmaceuticals released new results from the Global Open-Label Extension (OLE) trial of Onpattros (patisiran) for polyneuropathy of hereditary ATTR (hATTR) amyloidosis. They presented the data at the 2019 Peripheral Nerve Society (PNS) Annual Meeting in Genoa, Italy. Serum transthyretin (TTTR) levels decreased by about 80% in six months in the placebo arm of the APOLLO trial who started treatment with patisiran and were maintained over time. As of September 24, 2018, the Global OLE showed a mean of 20.5 months of patisiran, with more than 6,000 doses administered.

Aldeyra Therapeutics reported that its reproxalap failed to meet its primary or secondary endpoints in noninfectious anterior uveitis. Uveitis is an inflammation of the eye, which can cause vision loss. It causes about 10% of blindness in the U.S. Noninfectious anterior uveitis is a severe inflammatory eye disease that often occurs along with autoimmune diseases. It is marked by pain, sensitivity to light, redness, loss of vision, and other ocular symptoms.

The company did not provide data, but did state, “Statistical significance was not achieved for the primary or secondary endpoints, due to high rates of disease resolution in vehicle-treated patients, but the activity of reproxalap was consistently greater than that of vehicle.” In this case, “vehicle” refers to placebo.

Eli Lillys Trulicity hit its primary endpoint of significantly reducing A1C from baseline compared to a once-a-week dose of Trulicity. Trulicity (dulaglutide) is approved for type 2 diabetes in a 1.5 mg dose. In the Phase III AWARD-11 trial, patients received doses of 3.0 mg and 4.5 mg. At 36 weeks, the patients receiving higher doses had better A1C reduction. The trial also hits its secondary efficacy endpoints for superiority on weight reduction.

AstraZeneca announced a scheduled interim analysis of its Phase III CASPIAN clinical trial. The trial is evaluating Imfinzi (durvalumab) in first-line extensive-stage small cell lung cancer (SCLC). The Independent Data Monitoring Committee decided the trial had met its primary endpoint, showing statistically significant and clinically meaningful improvement in overall survival in patients receiving Imfinzi in combination with standard-of-care etoposide and platinum-based chemotherapy options compared to chemotherapy alone.

“The Phase III CASPIAN results offer new hope for patients who are facing the devastating diagnosis of small cell lung cancer, and for whom new medicines are urgently needed,” stated Jose Baselga, AstraZeneca’s executive vice president, Oncology R&D. “This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options.”

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