Arrowhead Pharmaceuticals announced that it has filed an application for approval to begin a Phase 1 clinical trial of ARO-APOC3, an RNAi-based investigational medicine targeting Apolipoprotein C-III being developed for the treatment of hypertriglyceridemia. Pending approval, Arrowhead intends to proceed with AROAPOC31001, a Phase 1 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in adult healthy volunteers, hypertriglyceridemic patients, and patients with familial chylomicronemia syndrome. The study is designed to enroll up to 63 subjects.
Monday, January 7, 2019
Invitae sees FY19 revenue of more than $220M, consensus $200M
Looking ahead to 2019, Invitae anticipates test volume of more than 500,000 samples generating more than $220M in revenue for the year.
Allergan to establish R&D presence in Cambridge, Massachusetts
Allergan announced it will establish an R&D presence in the Kendall Square section of Cambridge, Massachusetts, a hub of science, innovation and business opportunities. The site will be led by Don Frail, PhD, Senior Vice President of Research and External Scientific Innovation and Non-Clinical and Translational Sciences.
Adaptimmune gets endorsement for dose escalation in ADP-A2AFP study
Adaptimmune announced that the Safety Review Committee, or SRC, has endorsed dose escalation in the ongoing ADP-A2AFP study in patients with hepatocellular carcinoma to the second dose cohort. The SRC has also endorsed moving to the expansion phase of the ADP-A2M10 lung cancer study. Across both studies, most adverse events have been consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies with no evidence of alloreactivity or toxicity related to off-target binding. In the ADP-A2AFP study, two patients have received 100M transduced SPEAR T-cells targeting AFP in the first dose cohort, and there was no evidence of hepatotoxicity. The SRC endorsed dose escalation after evaluating the first two patients and taking into consideration the benefit:risk profile observed across programs in Cohort 1. In the ADP-A2M10 lung cancer study, ten patients have been treated in the first three cohorts and the expansion phase will allow for doses of up to 10B transduced cells.
Rigel Pharmaceuticals provides business update
In April 2018, TAVALISSE was approved in the U.S. for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. Following the product launch of TAVALISSE at the end of May 2018, the company has continued to execute on its commercial strategy. From May 29, 2018 through December 31, 2018, Rigel sold 1,794 bottles of TAVALISSE, of which, 1,556 were shipped directly to patients and clinics. There has been encouraging early demand for TAVALISSE, including use as the first or second choice after steroids. Based on experience to date, continuity of TAVALISSE therapy has been positive as well. The company estimates that over 45% of eligible patients have continued on treatment for the fourth consecutive month. Rigel remains on track with the execution of its global commercial strategy for fostamatinib. In early 2019, Kissei Pharmaceuticals, Rigel’s partner in Asia, plans to meet with Japan’s Pharmaceuticals and Medical Devices Agency. In parallel, the European Medicines Agency is progressing with its review of the marketing authorization application for fostamatinib for the treatment of adults with chronic ITP in Europe. The company anticipates a final decision on the MAA by the end of 2019. Discussions with potential partners to commercialize the product in Europe are ongoing and an agreement is expected during 2019. Based upon preliminary estimates, Rigel expects to end 2018 with approximately $128.5M in cash, cash equivalents, and short-term investments, which it believes will be sufficient to fund its operations into 2020. These operations include continued expansion of its commercial programs for TAVALISSE in the U.S., the launch of a Phase 3 clinical trial for fostamatinib in warm AIHA, and continued support of on-going research and development programs. In 2019, the company also expects incremental contribution to its cash position from revenue generated by its commercial business.
KemPharm: FDA approves Apadaz sNDA for two additional strengths
KemPharm announced today that the FDA has approved a supplemental new drug application, or sNDA, for two additional strengths of Apadaz, an immediate release combination of KemPharm’s prodrug, benzhydrocodone, and acetaminophen, or APAP. The approval of these new dosage strengths, 4.08 mg benzhydrocodone/325 mg APAP and 8.16 mg benzhydrocodone/325 mg APAP, follows the NDA approval on February 23, 2018 of the 6.12 mg benzhydrocodone/325 mg APAP dosage strength of Apadaz.
Axsome Therapeutics price target raised to $15 from $10 at H.C. Wainwright
H.C. Wainwright analyst Raghuram Selvaraju raised his price target for Axsome Therapeutics to $15 after the company announced that the Phase 2 Ascend trial of AXS-05, its proprietary combination of dextromethorphan and bupropion, in major depressive disorder had met its primary endpoint. The analyst views these data as “highly encouraging, particularly given the relatively small size of the trial.” AXS-05’s clear separation from bupropion underscores the differentiated profile of Axsome’s candidate, says Selvaraju. He keeps a Buy rating on Axsome Therapeutics.
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