Clinical Catch-up from Last Week: May 5-10

Almost every week, biopharma companies release results from ongoing clinical trials. Here’s a roundup of some of the top clinical trial news from the previous week.

• AveXis, a Novartis company, released interim data from the Phase I STRONG trial, additional data from the STR1VE trial, and the first data from the Phase III SPR1NT trial for Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1. Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMA gene. SMA is a severe neuromuscular disease noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis.
• Neurocrine Biosciences presented data from two Phase III clinical trials of opicapone, a once-daily, oral catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s disease. They found that patients receiving opicapone 50 mg with levodopa, the standard of care, had a significant and sustained improvement in symptoms. Neurocrine reported on two Phase III studies, BIPARK-1 and BIPARK-2. BIPARK-1 looked at about 600 patients with Parkinson’s and motor fluctuations. BIPARK-2 looked at about 400 patients with Parkinson’s and motor fluctuations. They received once-daily doses of opicapone of either 25 mg or 50 mg or placebo for 14 to 15 weeks. The data from these two Phase III trials showed that adding once-daily opicapone to levodopa significantly increased ON time without troublesome dyskinesia.
• Urovant Sciences presented Phase III data at the 2019 American Urological Association Annual Meeting (AUA). The drug is being evaluated in adults with overactive bladder. The EMPOWUR trial showed statistical significance with the drug over placebo in reducing daily urge urinary incontinence (UUI) episodes and reduction in daily micturitions. The company expects to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) by early 2020.
• Eli Lilly presented results from the Phase III EVOLVE-1 and EVOLVE-2 studies in Emgality (galcanezumab-gnlm) in monthly migraine headaches at the Annual Meeting of the American Academy of Neurology (AAN). Emgality was approved by the FDA in September 2018 for prevention of migraine in adults. These two studies stratified patients by frequency of migraine and evaluated two different doses of the drug for its ability to reduce the different subgroups’ headache frequency.
• UroGen announced results from a secondary analysis of its Phase III OLYMPUS trial of UGN-101 (mitomycin gel) in patients with endoscopically unresectable low-grade upper tract urothelial cancer (UTUC). The results were presented at the American Urological Association Annual Meeting. Patients in the study showed a 59% complete response rate, at least in a subset of patients. Of the evaluated complete responses, 27 patients have had a six-month evaluation, and 24 out of 27, or 89%, are disease-free at six months. Overall, 5 out of 41 patients who achieved a complete response have relapsed at any time during the study.
• Biogen presented data at the AAN meeting showing that its Spinraza (nusinersen) had a durable treatment effect in patients with SMA. Babies receiving the drug were followed up to four years. The infants showed additional or new motor function improvements during that period. These were the results of two trials, SHINE and NURTURE.
• Merck & Company announced it had positive results in a Phase III clinical trial evaluating Belsomra (suvorexant) for insomnia in patients with mild-to-moderate Alzheimer’s disease dementia. Merck presented the data at the AAN Annual Meeting. The trial looked at the efficacy and safety of Belsomra 10 mg, which could be increased to 20 mg based on patient’s response, compared to placebo in patients with mild-to-moderate Alzheimer’s dementia who also had insomnia. The trial met both primary and secondary efficacy endpoints. Belsomra improved mean total sleep time (TST) by 28.2 minutes compared to placebo at 4 weeks, which was the primary endpoint. This compared to a mean increase from baseline of 73.4 minutes with Belsomra and 45.2 minutes with placebo.
• Genentech, a Roche company, presented data from its FIREFISH clinical trial of risdiplam in Type 1 spinal muscular atrophy (SMA) at the AAN meeting. The drug is an oral survival motor neuron-2 (SMN2) splicing modifier and is part of a collaboration with the SMA Foundation and PTC Therapeutics. FIREFISH is a two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation trial in 21 infants. In FIREFISH Part 1, infants showed key motor milestones after one year of treatment with risdiplam. Among the babies receiving the dose chosen for the confirmatory Part 2 of the trial, seven, or 41.2%, were able to sit without support for at least 5 seconds. Also, 11 (64.7%) were able to sit with or without support while nine (52.9%) had upright head control after 12 months of treatment. One infant hit the milestone of standing by the 12-month mark.
• AstraZeneca reported positive data for its Phase III clinical trial of Calquence (acalabrutinib) in previously-treated patients with chronic lymphocytic leukemia (CLL). The trial was stopped early after the drug hit its endpoint at an interim analysis, showing the drug demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to a combination therapy of rituximab and a doctor’s choice of idelalisib or bendamustine.
• GW Pharmaceuticals reported that Epidiolex met the primary endpoint in its Phase III trial for seizures associated with tuberous sclerosis complex (TSC), a rare and severe type of childhood-onset epilepsy. The company indicated that the two doses of the drug, 25 mg and 50 mg, had similar results, providing seizure decreases of 48.6% and 47.5% from baseline, respectively. The placebo group had a seizure reduction of 26.5%.
• AstraZeneca and Daiichi Sankyo announced that their trastuzumab deruxtecan (DS-8201) hit its primary endpoint in HER2-positive, unresectable and/or metastatic breast cancer that had previously been treated with trastuzumab emtansine. The Phase II DESTINY-Breast01 clinical trial is an open-label, global, multicenter, two-part trial looking at the safety and efficacy of trastuzumab deruxtecan in patients with HER2-positive unresectable and/or metastatic breast cancer who had received previous treatment with Genentech’s trastuzumab emtansine (Kadcycla). The primary endpoint was objective response rate (ORR). Secondary objectives included duration of response, disease control rate, clinical benefit rate, progression-free survival and overall survival.
• Merck and Co. announced that its investigational 15-valent pneumococcal conjugate vaccine was "noninferior" to a currently available 13-valent pneumococcal conjugate vaccine in healthy babies 6 to 12 weeks of age. Phase II study, called V114-008, met its primary endpoint, showing noninferiority for the 13 serotypes seen in both vaccines. V114 also caused an immune response in infants for two additional disease-causing serotypes, 22F and 33F, which aren’t in PCV13. Pneumococcal disease is caused by Streptococcus pneumoniae, and includes non-invasive illnesses like pneumonia, sinusitis, and middle ear infection. It also causes invasive diseases such as bacteremia, bacteremic pneumonia, and meningitis.
• Bristol-Myers Squibb announced that its checkpoint inhibitor Opdivo failed to meet its primary endpoint in patients with newly diagnosed O6-methylguanine-DNA methyltransferase (MGMT)-unmethylated glioblastoma multiforme (GBM), a type of brain cancer. The drug was being evaluated in the Phase III CheckMate -498 trial with radiation compared to temozolomide plus radiation in this patient population. Temozolomide is an oral chemotherapy drug. The primary endpoint was overall survival (OS).
• Moderna announced the publication of results from two Phase I clinical trials of mRNA vaccines against two strains of influenza, H10N8 and H7N0. They were both published in the journal Vaccine. Both strains of influenza virus have shown high fatality rates, but neither has received vaccine approval. "Both seasonal and pandemic influenzas are serious public health problems, and there is a clear need for effective vaccines that can be quickly developed and deployed," stated Mike Watson, a study co-author and senior vice president of vaccine partnerships and health impact at Moderna. "Production of current flu vaccines takes significant time and requires virus or antigen production in cell-culture or eggs and in dedicated facilities. These Phase I data highlight the potential of Moderna’s mRNA platform to demonstrate similar or better immunogenicity than existing vaccines, which can be rapidly produced in a multi-use facility."

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