“The first quarter of 2019 was one of focused activity in the clinical development arena. We continued to identify and initiate new sites and enroll participants in our Company-sponsored Phase 3 trial in relapsed or refractory AML patients. We also worked closely with our two consortia partners to expand our late-stage uproleselan program, culminating in our announcement that the NCI consortium dosed its first patient in its trial in late April. During the same period, we worked with clinical collaborators at Duke Cancer Institute to plan our next trial for GMI-1359, a dual antagonist of E-selectin and CXCR-4, and defined individuals with breast cancer and bone metastases as our initial target study population,” said CEO Rachel King.
Thursday, May 2, 2019
Kaleido Biosciences reports Q1 EPS ($1.56), consensus (67c)
Cash and cash equivalents were $121.3M as of March 31. “The first quarter of 2019 was historic for Kaleido. During the four years since our inception, we built a library of over 1,000 Microbiome Metabolic Therapy candidates, completed seven non-IND human clinical studies, and established a pipeline with five programs spanning multiple disease areas. This rapid advancement of our platform and portfolio culminated with our Initial Public Offering and the initiation of our first human studies in two different target patient populations in the first quarter,” said Alison Lawton, president and CEO of Kaleido. “I am exceptionally proud of our talented and hard-working team, and we are grateful to everyone who has supported us along the way, including investigators, clinical study participants and investors. Looking ahead, we anticipate additional important milestones during 2019, including the initiation of our Phase 2 clinical trial under our cleared IND for KB195 in patients with urea cycle disorders (UCD) – as well as data from non-IND clinical studies we recently initiated for KB195 in UCD patients and KB174 in patients with cirrhosis.”
Kiniksa reports Q1 EPS ($1.27), consensus (80c)
“The first quarter of 2019 was marked by the advancement of our clinical-stage pipeline,” said Sanj K. Patel, CEO and Chairman of the Board of Kiniksa. “Progress included the initiation of our Phase 2a trial for KPL-716 in prurigo nodularis as well as the continued enrollment of our pivotal Phase 3 trial for rilonacept in recurrent pericarditis and our global Phase 2 trial for mavrilimumab in giant cell arteritis. Going forward, we expect to build upon this momentum with focused investment behind our product candidates, including commercial readiness activities for rilonacept, and continued execution.” Kiniksa is enrolling RHAPSODY, a global, randomized-withdrawal design, pivotal Phase 3 clinical trial of rilonacept in subjects with recurrent pericarditis. The primary efficacy endpoint is time-to-first-adjudicated pericarditis-recurrence in the RW period. Top-line data are expected in the second half of 2020. Kiniksa is enrolling a global Phase 2 proof-of-concept clinical trial of mavrilimumab in subjects with GCA. The primary efficacy endpoint is time-to-first-flare. Top-line data are expected in the second half of 2020. Kiniksa is enrolling a Phase 2a clinical trial of KPL-716 in subjects with prurigo nodularis. The primary efficacy endpoint is percent change from baseline in weekly average Worst-Itch Numeric Rating Scale. Top-line data are expected in the first half of 2020. Kiniksa plans to initiate an exploratory Phase 2 clinical trial in diseases characterized by chronic pruritus in the first half of 2019. The trial is designed to identify chronic pruritic conditions where interleukin-31 and/or oncostatin M may be playing a role and to investigate the efficacy, safety and tolerability of KPL-716 in reducing the moderate-to-severe pruritus experienced by these subjects. Top-line data are expected in the second half of 2020. Kiniksa is progressing its preclinical activities with KPL-404, a monoclonal antibody inhibitor of the CD40 co-stimulatory receptor, in T-cell-dependent, B-cell-mediated disorders. Kiniksa expects to file an investigational new drug application with the FDA in the second half of 2019 and initiate a Phase 1 clinical trial in the first half of 2020. Kiniksa expects that its cash, cash equivalents and short-investments will fund its current operating plan into 2021.
Abiomed: FDA approval of STEMI DTU Pivotal Randomized Controlled Trial
Abiomed announces that, on April 26, the FDA approved initiation of the ST-Elevation Myocardial Infarction Door-to-Unloading, or STEMI DTU, Pivotal Randomized Controlled Trial. The prospective, multi-center, two-arm trial plans to enroll 668 patients undergoing treatment for a STEMI heart attack. Half the patients will be randomized to receive delayed reperfusion after 30 minutes of left ventricular unloading with the Impella CP. The other half will receive immediate reperfusion, the current standard of care.
Aronex Pharmaceuticals Inc initiated at Oppenheimer
Aronex Pharmaceuticals Inc initiated with an Outperform at Oppenheimer. Oppenheimer analyst Kevin DeGeeter started coverage of Aronex Pharmaceuticals Inc with an Outperform rating and $20 price target. The analyst cited the expectation for positive Oraxol Phase III breast cancer data in Q3, proprietary survey results suggesting potential for meaningful uptake of KX2-391 for treatment of actinic keratosis, and strength of the company’s commercial channel for specialty oncology products and opportunity for operating leverage. DeGeeter views Aronex Pharmaceuticals Inc’s Orascovery oral chemotherapy platform as offering potential for important efficacy and tolerability benefits compared to traditional IV chemotherapy, and views partnering as providing important catalysts over the next 12-24 months.
Dicerna submits updated IND application for DCR-PHXC
Dicerna Pharmaceuticals announced the submission of an updated investigational new drug, or IND, application to the FDA for DCR-PHXC, the Company’s lead GalXC product candidate for the treatment of all forms of primary hyperoxaluria, or PH. The update reflects agreement on the primary endpoint for the PHYOX 2 pivotal clinical trial, which is enrolling patients with PH type 1, or PH1, and PH type 2, or PH2, and alignment with the FDA regarding the path to full approval for the treatment of patients with PH1, as conveyed during a recent FDA Type A meeting. The Company will continue its ongoing dialogue with the FDA regarding endpoints for studies involving patients with PH2 and PH3, as part of the PHYOX clinical development program for DCR-PHXC.
Rocket Pharma presents preclinical data of RP-A501 for Danon disease
Rocket Pharmaceuticals presents preclinical data of RP-A501 at the American Society of Gene and Cell Therapy 2019 Annual Meeting in Washington, D.C. RP-A501 is the Company’s adeno-associated viral vector-based gene therapy for the treatment of Danon disease. The data is included in an oral presentation by Annahita Keravala, Ph.D., Associate Vice President, AAV Platform, entitled, “Systemic Delivery of AAV9.LAMP2B for the Treatment of Danon Disease: Toxicology Studies in Mice and Cynomolgus Monkeys.” Investigational New Drug application-enabling toxicology studies were conducted in wild-type mice and non-human primates. Three dose levels were tested in mice, including 3X10 13 vg/kg, 1X10 14 vg/kg, and 3X10 14 vg/kg. The highest dose level from the murine study, 3X10 14 vg/kg, was tested in non-human primates. No dose-related adverse events were observed at all tested doses in both mice and non-human primates. Vector genomes, mRNA and protein expression were widely distributed across key tissues with high levels of transduction, transcription and translation detected in the heart, skeletal muscle, diaphragm and liver. The ASGCT presentation also highlights previously reported preclinical efficacy data of RP-A501 in LAMP-2 knockout mice which showed dose-dependent improvements and restoration of cardiac function, with responses observed in both older and younger KO mice.
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