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.
Monday, January 7, 2019
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.
Celgene says ozanimod regulatory submissions on-track for Q1
Overview of Key Milestones Expected Over the Next 12 Months: REVLIMID: Approval expected by the U.S. Food and Drug Administration on the supplemental New Drug Application for REVLIMID in combination with rituximab in relapsed/refractory indolent lymphoma. Approval expected by the European Medicines Agency’s Committee for Medicinal Products for Human Use for REVLIMID in combination with bortezomib and dexamethasone in newly diagnosed multiple myeloma. Data expected from the phase III ROBUST trial with REVLIMID in combination with rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate and prednisone in patients with first-line ABC-subtype diffuse large B-cell lymphoma. POMALYST/ IMNOVID: Approval expected by the EMA CHMP and Japan Pharmaceuticals and Medical Devices Agency for POMALYST/IMNOVID in combination with bortezomib and dexamethasone in relapsed/refractory multiple myeloma. OTEZLA: Approval expected by the U.S. FDA for the sNDA in Behet’s disease with a Prescription Drug User Fee Act action date of July 21, 2019. Approval by the PMDA in Japan is expected in H2:2019. Submission of the sNDA with label update for moderate to severe scalp psoriasis to the U.S. FDA expected in Q2:2019. ABRAXANE: Data from the phase III apact trial with ABRAXANE as adjuvant therapy in patients with surgically resected pancreatic cancer. PDUFA action date of March 12, 2019 for the supplemental Biologics License Application submission filed by Roche of Tecentriq in combination with ABRAXANE for the initial treatment of patients with PD-L1-positive, metastatic triple-negative breast cancer. Milestones Expected for Key Pivotal Assets: Ozanimod: U.S. NDA and EU Marketing Authorization Application submissions in relapsing multiple sclerosis on-track for Q1:2019. Phase III TRUE NORTH trial in ulcerative colitis expected to complete enrollment in H1:2019. Fedratinib: U.S. FDA approval expected by year-end 2019. EU MAA submission planned in H1:2019. Phase I/II combination trial with luspatercept planned. Luspatercept: U.S. and EU regulatory applications for transfusion-dependent, lower-risk myelodysplastic syndromes with ring sideroblasts and transfusion-dependent beta-thalassemia planned for H1:2019. Data expected from the phase II myelofibrosis trial in H2:2019. Liso-cel: Data from the pivotal TRANSCEND trial in relapsed/refractory DLBCL expected in 2019. U.S. BLA submission expected in H2:2019. Pivotal phase II trial in relapsed/refractory chronic lymphocytic leukemia to be initiated in H1:2019. bb2121: Data from the KarMMa pivotal trial in RRMM expected in H2:2019: Phase II trial in NDMM to be initiated in H2:2019.
CytoReason, Pfizer sign collaboration agreement for machine learning model
CytoReason announced that it has entered into a collaboration agreement with Pfizer that will leverage CytoReason’s cell-centered models of the immune system. CytoReason will receive from Pfizer payments potentially equaling up to low double digit millions of US$ for technology access fees, research support and certain success-based payments. CytoReason’s proprietary platform helps rebuild lost cellular information from gene expression data and associates genes to specific cells. This information is then integrated with additional omics and literature data to create a cell-based model of the trial-specific immune response. Integration with the CytoReason disease model empowers the study analytics and allows the model to learn and improve, leading to robust target discovery, drug response biomarkers and indication selection
BioTime enters into research option agreement with Orbit Biomedical
BioTime announced that it has entered into a research and option agreement with Orbit Biomedical Limited. Orbit Biomedical, based in London, UK and Ambler, PA, was founded in 2018 to develop a surgical device and training platform for the delivery of cell and gene therapies to the sub-retinal space. Under the terms of the agreement, BioTime and Orbit Biomedical will collaborate on the use of Orbit Biomedical’s proprietary injection technology to deliver OpRegen for the treatment of dry age-related macular degeneration. Under the terms of the exclusive 12 month agreement, BioTime and Orbit Biomedical plan to investigate the safety and utility of Orbit Biomedical’s surgical delivery technology in the ongoing Phase I/II clinical study of OpRegen for the treatment of dry-AMD.
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