Stifel analyst Paul Matteis said each epileptologist he talked to at the American Epilepsy Society meeting said that patients are frequently asking for GW Pharmaceuticals’ Epidiolex. Additionally, the doctors he spoke to said they are already writing prescriptions for the drug. Early headaches with GW’s specialty pharmacy hub, which were cited by a few physicians, appear to have been addressed by the company, added Matteis, who reiterated his Buy rating on GW Pharmaceuticals shares following the AES meeting.
Monday, December 3, 2018
Eiger BioPharmaceuticals: FDA accepts IND application for lonafarnib
Eiger BioPharmaceuticals announced that the U.S. FDA has accepted the Investigational New Drug application for lonafarnib in the treatment of both Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies. Eiger is collaborating with The Progeria Research Foundation and plans to submit a new drug application to the FDA in 2019. There is no approved treatment for progeria or progeroid laminopathies. Lonafarnib is a first-in-class prenylation inhibitor in development for hepatitis delta virus infection and also progeria and progeroid laminopathies.
Gemphire Therapeutics announces review of strategic alternatives
Gemphire Therapeutics announced that its Board of Directors is conducting a review of a range of strategic alternatives focused on maximizing stockholder value. The Company has engaged Ladenburg Thalmann & Co. Inc. to act as its strategic financial advisor for this process. The Board of Directors has established a committee to oversee this review. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, in-licensing, or other strategic transaction involving the Company. There can be no assurance that this process will result in Gemphire pursuing any transaction or that any transaction, if pursued, will be completed. The Company does not intend to discuss or disclose further developments regarding the strategic review process unless and until its Board of Directors has approved a specific action or otherwise determined that further disclosure is appropriate or required by law. “As we have previously disclosed, the recent request by the U.S. FDA for additional preclinical data on gemcabene means that our planned Phase 3 programs, initially focused in hypertriglyceridemia, are expected to start later than originally planned,” said Steven Gullans, Ph.D., CEO of Gemphire. “We continue to be encouraged by the results from 25 clinical trials in nearly 1,200 adult patients in which gemcabene demonstrated statistically significant signs of efficacy with no severe adverse events or drug-drug interactions. We remain confident that we will be able to meet the FDA’s requests to enable it to reconsider lifting the partial clinical hold on gemcabene. However, we believe it is prudent to fully leverage our resources by exploring strategic alternatives.” The Phase 2a study investigating gemcabene in Familial Partial Lipodystrophy disease recently completed enrollment. This open-label investigator-led study, being conducted by Dr. Elif Oral at the University of Michigan, is assessing gemcabene’s efficacy, including its effects on plasma triglyceride and inflammatory markers, as well as liver fat determined by MRI-PDFF. To date, no drug-related toxicities have been detected, and two patients have completed the 24 week gemcabene treatment regimen. Top-line results are expected in mid-2019. The Company continues to make progress with the ongoing preclinical studies recently requested by the FDA with the goal of providing the FDA with the data it requires to lift the partial clinical hold on gemcabene. Results are expected to be provided to the FDA in the second half of 2019.
Onconova presents rigosertib with azacitidine data at ASH
Onconova Therapeutics announced the presentation of the efficacy and safety results of oral rigosertib in combination with azacitidine in patients with HR-MDS reported at an oral presentation during the American Society of Hematology, or ASH. Rigosertib, the company’s lead compound, is being evaluated in both intravenous and oral forms. Seventy-four patients were treated with a median age of 69 years at nine clinical sites and received either 840 mg or 1,120 mg of oral rigosertib daily divided into two doses, in combination with a standard dose of injectable azacitidine. Of the 55 evaluable patients, 29 patients were treated with a daily dose of 1,120 mg of oral rigosertib, either 560 mg twice daily or 840 mg in the a.m. and 280 mg in the afternoon. Twenty-six patients were treated with 560 mg in the AM and 280 mg in the PM for the first three weeks of a four-week cycle. All patients also received 75 mg/m2/day SC or IV azacitidine during the second week of the four-week cycle. The median duration of treatment for the HMA naive and HMA failed patients was 7.8 and 4.9 months respectively. The median duration of response in these groups was 12.2 and 10.8 months, respectively. The overall response rate, or ORR, using the IWG 2006 criteria, in 29 HMA naive patients was 90%, including 10 patients with complete remission, or CR. Among the 26 evaluable HMA-failed patients the ORR was 54% including 8% CR or PR. The median time to initial and best response were 1 and 4 cycles in the HMA naive group and 2 and 5 cycles in the HMA failed group. The safety population received at least 1 dose of oral rigosertib. The combination was well tolerated. Other than genitourinary adverse events, or AEs, the AE profile was similar to those described for azacitidine alone in this patient population. Genitourinary AEs, including hematuria and dysuria were observed. A safety optimization strategy was implemented for the higher dose cohort of 1,120 mg of oral rigosertib. These strategies included earlier in the day administration of the PM dose, oral hydration, monitoring of urinary pH and mandatory bladder emptying at night. Collectively these strategies resulted in mitigation of the target genitourinary AEs, including reduction of genitourinary grade 3 AEs reported from an earlier cohort despite receiving a higher dose of oral rigosertib. In conclusion, oral rigosertib in combination with azacitidine was well tolerated in HMA naive and HMA failed HR-MDS patients. The combination produced an encouraging rate of overall response and complete remission in both groups. The safety optimization strategies and increased dose exploration of oral rigosertib in the combination is leading to the development of a pivotal Phase 3 trial in HMA and chemotherapy naive patients. Onconova plans to meet with the FDA to discuss the results of the Phase 2 trial and the planned Phase 3 trial, and to seek a special protocol assessment. The company has partnered rigosertib with SymBio Pharmaceuticals, for Japan and Korea, and with Pint Pharma for Latin American countries. Both partners have indicated their interest in participating in the proposed new pivotal Phase 3 trial by enrolling patients in their respective territories. SymBio is currently conducting Phase 1 studies with oral rigosertib in Japan and also participating in the Phase 3 global INSPIRE trial. The company is also actively seeking additional collaborations for rigosertib in other geographies.
Gamida Cell: NiCord recipients showed immune reconstitution in study
Gamida Cell reported translational data showing that recipients who received NiCord, an investigational cell therapy in Phase 3 clinical development for allogeneic hematopoietic stem cell transplant had rapid and robust reconstitution of key immune cells. Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant. Despite the curative potential of bone marrow transplants, it is estimated that more than 40% of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor. While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells. The poster presentation, “Rapid and robust CD4+ and CD8+ T-, NK-, B- and monocyte cell reconstitution after nicotinamide-expanded cord blood transplantation”, described early, in-depth immune reconstitution data from the completed Phase 1/2, multicenter clinical study of NiCord as a stand-alone graft after myeloablative therapy in patients with high-risk hematologic malignancies. A random subgroup of 27 patients from this study had extensive immune monitoring evaluated throughout the first year after transplant. The primary endpoint was the probability of achieving CD4+ immune reconstitution within the first 100 days, and the secondary endpoints included the recovery of B cells, CD4+ T cells and natural killer cells during the first year after transplantation. These data were compared to cohorts of adolescent and young adults with hematologic malignancies receiving unmanipulated cord blood transplantation or unrelated bone marrow transplantation. Key findings from the analysis include the following: 91% of patients achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation with NiCord. Immune reconstitution of T cells was similar in the NiCord group compared to the younger cohorts receiving unmanipulated cord blood and unrelated bone marrow. Immune reconstitution of B cells and NK cells was significantly faster after transplantation with NiCord compared to the other groups. Immune reconstitution after NiCord transplantation was associated with recovery of a broad spectrum of T cell, B cell and NK cell subsets representing a range of effector functions similar to that observed with other graft sources.
Ocular Therapeutix to hold a conference call
Management holds a conference call to discuss the FDA approval of DEXTENZA for treatment of ocular pain following ophthalmic surgery on December 3 at 8:30 am.
https://thefly.com/landingPageNews.php?id=2831101
Casi Pharmaceuticals announces China market approval for EVOMELA
CASI Pharmaceuticals announces that it has received National Medical Products Administration approval of Melphalan Hydrochloride For Injection for: use as a high-dose conditioning treatment prior to hematopoietic progenitor cell transplantation in patients with multiple myeloma, and the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.
https://thefly.com/landingPageNews.php?id=2831103
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