The FDA set a December 21, 2018, PDUFA Date for Shire/Janssen’s prucalopride. There are no priority designations for the drug.
Acceleron and Celgene announced that in the 48-week, 336 patient, Phase III BELIEVE trial, 21.4% of patients treated with luspatercept had at least 33% reduction in red blood cell (RBC) transfusions compared to 4.5% with placebo in patients with beta thalassemia.
Celgene and Juno announced that in a 16 patient, Phase I trial, treatment with lisocabtagene maraleucel led to an overall response of 81% and a complete response in 43% in patients with refractory or resistant chronic lymphocytic leukemia.
Apellis announced interim data from 10 patients in the Phase 1, PADDOCK trial, where APL-2 patients achieved transfusion independence with an average hemoglobin increase of 4.2 g/dL by day 85 in patients with paroxysmal nocturnal hemoglobinuria. Apellis announced interim data from autoimmune hemolytic anemia patients in the open label, Phase II, PLAUDIT trial where APL-2 increased hemoglobin by 3.4 g/dL at day 56 in 12 patients with cold agglutinin disease and by 2.0 g/dL in warm antibody autoimmune hemolytic anemia.
NewLink announced that in a Phase II trial, of 102 patients with advanced melanoma, indoximod monotherapy or in combination with a checkpoint inhibitor (pembrolizumab, nivolumab or ipilumumab), 70 of the patients had an ORR of 56%.
Novartis has the ongoing Phase III STRIVE study evaluating intravenous onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy Type 1, 2 & 3 and Phase 1 STRONG study evaluating onasemnogene abeparvovec in spinal muscular atrophy Type 2. Onasemnogene abeparvovec will compete with nusinersen. Novartis bought AveXis in April 2018. The FDA accepted the BLA for onasemnogene abeparvovec in December 2018 and set a PDUFA Date of 5/3/19. AveXis indicated it may price onasemnogene abeparvovec at $4 million or more for the single dose treatment of spinal muscular atrophy.
Biohaven announced that in a 1,351 patient, Phase III trial, 21.2% of patients with acute migraine treated with rimegepantin an orally dissolving tablet (ODT) formulation were pain-free and 35.1% were free of their most bothersome symptoms at two hours compared to 10.9% and 26.8% that received placebo.
Onconova announced that in a 74 patient, Phase II trial, treatment with the combination of rigosertib and azacitidine demonstrated an overall response rate of 90% and complete remission in 34% of patients with myelodysplastic syndrome.
TG Therapeutics announced that in a 10 patient, Phase I/II trial, a combination of umbralisib, ublituximab and pembrolizumab resulted in a complete response in one patient and a partial response in 8 patients in patients with relapsed/refractory chronic lymphocytic leukemia. TG Therapeutics announced interim results from a Phase I/Ib trial where a combination of umbralisib, ublituximab and bendamustine resulted in an overall response in 12/25 patients with diffuse large B-cell lymphoma and 11/13 patients with follicular lymphoma.
Redhill announced that in the 455 patient, Phase III, ERADICATE Hp2 trial, the combination of amoxicillin, omeprazole and rifabutin (RH-105) demonstrated an 84% eradication of H. pylori compared to 58% eradication with amoxicillin and omeprazole in dyspepsia patients with confirmed H. pylori infection. Redhill plans to file an NDA in the first half of 2019.
Legend and J&J announced interim results from 57 patients in a phase I Chinese trial where JNJ-68284528 demonstrated an overall response rate of 88% and complete response of 74% in patients with relapsed multiple myeloma. 90% of patients in the interim analysis developed cytokine release syndrome (CRS) with 4 developing severe CRS. All but one CRS patient recovered. Pyrexia was the most common ADR. Only one (out of 57) has developed mild neurotoxicity.
Bluebird announced that in a 21 patient, Phase I trial, treatment with bb2121 resulted in complete remission in 56% of patients with relapsed or refractory multiple myeloma and 9-months progression-free survival of 71%. Bluebird announced that in a 43 patient, Phase I trial, relapsed multiple myeloma patients were treated with a lymphodepleting regimen of fludarabine and cyclophosphamide followed by bb2121. Half of the patients that received a higher dose (>150,000,000 cells) of bb2121 had a PFS of 10.8 months, a CR of 50% and an ORR ion 95.5%. Bluebird announced interim results from 12 patients in a Phase I trial where treatment with bb21217 (a longer lasting version of bb2121) resulted in an 83% objective response rate in heavily pretreated multiple myeloma patients.
Karyopharm announced interim results from the first 115 or 127 patients in the Phase IIb, SADAL trial, where treatment with selinexor resulted in a 29.6% Overall Response Rate in patients with relapsed or refractory diffuse large B-cell lymphoma. Karyopharm announced interim results from two arms of the Phase Ib/II STOMP trial. In one arm, selinexor in combination with daratumumab achieved an overall response rate of 73% in patients with relapsed or refractory multiple myeloma. In the second arm, selinexor in combination with pomalidomide achieved an overall response rate of 50% in patients with relapsed or refractory multiple myeloma. Karyopharm announced that in the 122 patient, Phase IIb, STORM trial, treatment with selinexor in combination with dexamethasone resulted in a 26% overall response rate in patients with highly resistant multiple myeloma.
Geron announced interim results from 59 patients in the 24-week, Phase II, IMbark trial, where 10% of patients treated with imetelstat achieved a ≥35% reduction in spleen volume and 32% achieved a ≥50% reduction in Total Symptom Score, at 24 weeks in Intermediate-2 or High-risk myelofibrosis patients who are relapsed or refractory to a Janus Kinase inhibitor.
Global Blood Therapeutics announced that in the 24-week, 154 patient Phase III, HOPE trial, 65% of voxelotor 1,500 mg patients and 33% of the 900 mg patients had a 1 g/dL increase in hemoglobin compared to 10% with placebo in patients with sickle cell disease.
AbbVie announced it was stopping enrollment in the Phase III TAHOE small cell lung cancer trial after an independent interim review found that treatment with rovalpituzumab resulted in worse overall survival than the comparator topotecan. AbbVie will continue development of rovalpituzumab for other indications but will not seek accelerated approval.
Cerenis announced that in the 24-week, Phase III, TANGO trial, CER-001 did not reduce mean vascular wall area compared to placebo in patients with Familial Hypoalphalipoproteinemia.
Conatus announced that in a 24-week, 263 patient, Phase IIb trial, emricasan did not improve hepatic venous pressure gradient compared to placebo in patients with nonalcoholic steatohepatitis.
Genfit announced that in a 12-week, 45 patient, Phase II trial, treatment with elafibranor resulted in a 48% decrease in serum alkaline phosphatase with 80 mg and a 41% decrease with 120 mg compared to a 3% increase with placebo in patients with primary biliary cholangitis that had an inadequate response to ursodeoxycholic acid.
Immunomedics announced that in a 108 patient, Phase II clinical trial, treatment with sacituzumab govitecan resulted in an ORR of 33% and a duration of response of 7.7 months in patients with triple-negative breast cancer.
Spectrum announced that in 237 patient, Phase III RECOVER trial, eflapegrastim was non-inferior to pegfilgrastim in reducing the duration of severe neutropenia during four cycles of treatment in patients with breast cancer.
Tralokinumab failed to decrease annual exacerbation in severe asthmatics compared to placebo in the 52-week, 1,000 patient, Phase III STRATOS-1 trial and a 454-patient, Phase IIb trial. AstraZeneca announced topline data from the Phase III, STRATOS 2 trial of asthma patients with increased IL-13 activity, where treatment with tralokinumab did not reduce exacerbations compared to placebo. In the 12-week, 224 patient, Phase II, MESOS trial, treatment with tralokinumab did not lower bronchial eosinophil count compared to patients with moderate to severe asthma that was inadequately controlled on inhaled corticosteroids. In the 40-week, 140 patient, Phase III, TROPOS trial of asthma patients with increased IL-13 activity, treatment with tralokinumab did not reduce the need for corticosteroids compared to placebo.
AstraZeneca announced that in the Phase III, open-label, EAGLE trial, neither durvalumab monotherapy or durvalumab plus tremelimumab improved overall survival compared to standard-of-care chemotherapy in patients with platinum-resistant head and neck squamous cell carcinoma.
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