Amyris announced that it has received a successful GRAS notification from the FDA for its zero calorie sweetener. With this notification, the FDA has accepted the unanimous conclusion of a panel of food-safety experts, the company announced on August 30, that Amyris’s zero calorie sweetener made from sugarcane is safe for use as a sweetener.
Wednesday, December 12, 2018
Galmed, Intercept, Viking, Madrigal initiated at B. Riley FBR
B. Riley FBR rolls out coverage on NASH players with three Buy ratings. B. Riley FBR analyst Mayank Mamtani last night initiated coverage of four companies working on developing drugs to treat nonalcoholic steatohepatitis. The analyst started Galmed Pharmaceuticals (GLMD) with a Buy rating and $28 price target, Intercept Pharmaceuticals (ICPT) with a Buy rating and $155 price target, Viking Therapeutics (VKTX) with a Buy rating and $16 price target, and Madrigal Pharmaceuticals (MDGL) with a Neutral rating and $124 price target. NASH disease burden is “poised to create the next liver transplant crisis” and is drawing significant investments from the biotech industry, Mamtani writes in a research note. He points out that Intercept’s obeticholic acid is the only NASH drug to have the FDA’s breakthrough therapy designation for strong anti-fibrotic efficacy and be previously approved by the FDA, for an orphan liver indication. His proprietary safety analysis suggests the Street should gain comfort in drug’s cardiovascular profile going into pivotal readout in Q2 of 2019. Mamtani believes Viking’s lead drug, VK2809, has an “excellent” safety and tolerability profile that assuages concerns of any off-target effects. Further, his comparator analysis suggests efficacy in high-dose of Galmed’s Aramchol drug “represents a compelling clinical profile on both regulatory acceptable endpoints.”
MeiraGTx announces publication of new research on AAV-GAD gene therapy
MeiraGTx announced publication of research assessing the mechanism underlying the motor improvement observed in patients with Parkinson’s disease who were treated with adeno-associated virus, or AAV, encoding glutamic acid decarboxylase, or AAV-GAD, an investigational gene therapy product candidate, in a completed Phase 2 study. In the new research, which was published in the November 28 issue of Science Translational Medicine, patients treated with AAV2-GAD gene therapy expressed a distinct treatment-related metabolic brain network, providing a clinical benefit by inducing the formation of new polysynaptic pathways connecting the STN to cortical motor regions. Rather than act through conventional motor pathways involving certain regions of the brain, AAV-GAD gene therapy was observed to co-opt adjacent “nonmotor” regions in the treated patients. The research published in Science Translational Medicine showed that patients treated with AAV-GAD gene therapy developed a unique treatment-dependent metabolic brain network, which reflected the formation of new functional pathways linking the STN to motor cortical regions. GADRP was characterized by increased metabolism in certain brain regions along with relatively reduced metabolic activity in other brain regions. The research also showed that the GADRP correlated with clinical improvement in the gene therapy-treated subjects as measured by changes in the Unified Parkinson’s Disease Rating Scale motor ratings. In contrast, the clinical correlation with sham surgery-related pattern was not significant.
XBiotech bermekimab study meets primary, secondary endpoints
XBiotech announced that its open label, proof of concept, multicenter study using bermekimab to treat patients with moderate to severe atopic dermatitis, or AD, has completed and the study met all primary and secondary endpoints. Thirty eight patients in two treatment groups received a low or high dose of bermekimab once weekly for either a four or seven-week treatment regimen, respectively. Statistically significant improvement was seen for all efficacy endpoints in the high dose group; and a significant dose response for the high dose compared to low dose group was observed for key endpoints, including the Eczema Area and Severity Index, or EASI, Global Individual Sign Score, or GISS, Patient Oriented Eczema Measure, or POEM, Hospital Anxiety and Depression Scale, or HADS, and SCORing Atopic Dermatitis, or SCORAD. While clinically and statistically significant improvement was seen for all clinical endpoints in the high dose group, also notable was the speed, magnitude, and trajectory of responses seen. In the high dose group, for example, after only four weeks of treatment, 61% of patients achieved a 4-point improvement in the Pruritus Numerical Rating Scale, or NRS, a key method used to measure itch in clinical trials for atopic dermatitis, and 75% of patients achieved a 4-point improvement by week 7. For the only biological therapy currently approved to treat atopic dermatitis, dupilumab, which was granted breakthrough designation by the FDA, only 16%-23% of patients achieved a 4-point NRS improvement after 4 weeks of therapy; and only 36-41% of patients achieved a 4-point improvement by week 16. Another key measure of efficacy in the XBiotech study was the EASI. In the study, 39% of high dose patients achieved 75% improvement in EASI score after four weeks of therapy and 71% of patients achieved EASI-75 at week seven. Of note, participants were not allowed to use concomitant topical corticosteroids during the study and thus these improvements were most likely due to the study drug alone. The only approved biological therapy, dupilumab, reports only 44-51% of patients achieved EASI-75 by week 165.
FDA posts tenative approval for Taro Pharmaceutical’s Dapsone
https://thefly.com/landingPageNews.php?id=2836085
Janssen says Phase 3 data demonstrate superiority of Tremfya vs. Consentyx
The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the ECLIPSE study demonstrating that Tremfya was superior to Cosentyx in treating adults with moderate to severe plaque psoriasis for the primary endpoint assessed at week 48. Data from the multicenter, randomized, double-blind head-to-head Phase 3 study demonstrated that 84.5% of patients treated with guselkumab achieved at least 90% improvement in their baseline Psoriasis Area Severity Index score at week 48, compared with 70.0% of patients treated with secukinumab. “These data, presented at the 3rd Inflammatory Skin Disease Summit in Vienna, 12-15 December, mark the first-ever results from a head-to-head study comparing an interleukin (IL)-23-targeted biologic therapy with an IL-17 inhibitor. ECLIPSE is Janssen’s fourth Phase 3 study for guselkumab in plaque psoriasis2-4 and is part of a comprehensive clinical development program that also includes ongoing Phase 3 studies in psoriatic arthritis and Crohn’s disease,” the company stated.
Neurocrine price target lowered to $97 from $127 at JPMorgan
After Neurocrine announced that the phase 2b T-Force GOLD study of Ingrezza for pediatric Tourette syndrome failed its primary endpoint, JPMorgan analyst Anupam Rama called the update “a clear disappointment” and removed the Tourette opportunity out of his model given this and other previous trial setbacks. Given the removal of the indication, he cut his price target on Neurocrine shares to $97 from $127. However, he keeps an Overweight rating on the shares, stating that he expects investor focus to return to Ingrezza launch progress in tardive dyskinesia, the CAH data for NBI-74788 expected in the first quarter and the opicapone NDA filing in Parkinson’s Disease in Q2.
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