Pipeline News and Updates
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We just added 16 new drugs to the knowledgebase:
Regulatory Update
The FDA approved tirzepatide (Mounjaro, Lilly), on 5/13/2022, as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Tirzepatide was approved with a black box warning regarding an increased risk for thyroid c-cell tumors and is contraindicated in patients with medullary thyroid carcinoma Multiple Endocrine Neoplasia syndrome type 2. The FDA rejected bimekizumab for the treatment of moderate to severe plaque psoriasis due to inspection observations that need to be corrected. Biogen and Eisai completed a rolling submission for lecanemab for the treatment of early Alzheimer's disease. The FDA delayed the PDUFA dates for Amicus’ combination of cipaglucosidase alfa and miglustat by three months to allow more time to review new information. The new dates are 8/29/2022 for the miglustat NDA and 10/29/2022 for the cipaglucosidase alfa BLA. Green Valley discontinued a Phase III Alzheimer’s trial in May 2022 evaluating oligomannate due to insufficient funding to support the trial. The trial had enrolled 257 Alzheimer’s patients in North America, 118 in Europe and 64 in China. KemPharm bought Orphazyme in May 2022 and took over development of arimoclomol. KemPharm plans to refile an NDA for arimoclomol in 1Q23. Announced Research Updates Roche announced interim results from the 534 patient, Phase III, SKYSCRAPER-01 trial, where adding tiragolumab to atezolizumab did not improve PFS compared to atezolizumab alone in patients with PD-L1-high locally advanced or metastatic NSCLC. J&J announced that in the 295 patient, Phase Ib, TRIMM-2 trial, treatment with teclistamab plus daratumumab improved the overall response rate (ORR) compared to daratumumab alone in heavily pretreated patients with multiple myeloma. Gamida announced that in a long-term extension of a 125 patient, Phase III trial, patients who received an umbilical cord blood transplant (UCBT) and were treated with omidubicel did not achieve a statistically significant improvement in non-relapse mortality or survival one year after transplant compared to UCBT alone. BMS announced that in the 1,221 patient, open-label, Phase III, POETYK PSO-LTE trial, a two-year extension of the Phase III, POETYK PSO-1 and POETYK PSO-2 trial, efficacy at 60-weeks with deucravacitinib was maintained, with 77.7% of patients maintaining PASI-75 and 58.7% maintaining a Physicians Global Assessment score of 0 or 1 (clear/almost clear skin). Published Research Updates In the 36 patient cohort 1 of the 15-day, 72 patient, Phase II/III, CAPELLA trial, 88% (21/24) of patients that had oral lenacapavir added to a failing HIV-treatment regimen achieved at least a 0.5 log10 copies/mL reduction in viral load compared to 17% (2/12) that received the failing regimen only in patients with multidrug resistant HIV-1 infection. In the 178 patient, open-label, Phase III, ATTAIN trial, treatment with etirinotecan pegol did not improve overall survival compared to physician’s choice of eribulin, ixabepilone, vinorelbine, gemcitabine, paclitaxel, docetaxel, or nab-paclitaxel in patients with breast cancer and brain metastases. In a 12-week, 147 patient, Phase II trial, treatment with BI 1015550 resulted in a 5.7 ml increase in Forced Vital Capacity (FVC) compared to a 81.7 ml decrease with placebo in patients with idiopathic pulmonary fibrosis (IPF) who were not receiving an antifibrotic drug. Among IPF patients receiving an antifibrotic drug, there was a 2.7 ml increase in Forced Vital Capacity (FVC) compared to a 59.2 ml decrease with placebo. In a 52-week, 24 patient, Phase III trial, treatment with Oxalobacter formigenes did not decrease plasma oxalate levels compared to placebo in patients with PH. In a 24-week, 463 patient, Phase III SEED trial, treatment with dorzagliatin reduced HbA1c 1.07% compared to a 0.50% decrease with placebo, in Chinese patients with type 2 diabetes. In a 24-week, 767 patient, Phase III trial, adding dorzagliatin to metformin lowered HbA1c 1.02% compared to a 0.36% decrease with metformin monotherapy in Chinese patients with type 2 diabetes. In the 4-week, 115 patient, Phase III, URIROX-1 trial, treatment with reloxaliase reduced the 24-hour urinary oxalate excretion by 22.6% compared to a 9.7% decrease with placebo in patients with enteric hyperoxaluria. Regulatory Update
The FDA approved vonoprazan in combination with amoxicillin and clarithromycin (Voquenza Triple Pak, PhathomPharmaceuticals) and in combination with amoxicillin alone (Voquenza Dual Pak, Phathom Pharmaceuticals), on 5/3/2022, for the treatment of Helicobacter pylori infections. Announced Research Updates Astellas announced that in the 12-week, 501 patient, Phase III, SKYLIGHT 1 trials, patients treated with fezolinetant reduced the severity of moderate to severe VMS by 0.15 points at 4 weeks and 0.24 points at 12 weeks with 30 mg and 0.19 points at 4 weeks and 0.20 points at 12 weeks with 45 mg compared to placebo in patients with moderate to severe VMS. The frequency of moderate to severe VMS events per day versus placebo was reduced by 1.87 episodes at 4 weeks and 2.39 episodes at 12 weeks with 30 mg and 2.07 episodes at 4 weeks and 2.55 episodes at 12 weeks with 45 mg compared to placebo. Regulatory Update
The FDA approved mavacamten (Camzyos, Bristol Myers Squibb), on 4/29/2022, to improve functional capacity and symptoms in adults with symptomatic New York Heart Association (NYHA) class II to III obstructive hypertrophic cardiomyopathy.
The FDA rejected Hutchmed’s surufatinib for the treatment of advanced pancreatic and extrapancreatic neuroendocrine tumors and recommended a multiregional trial using a population that is similar to the U.S. population. The FDA rejected Junshi’s and Coherus’ toripalimab, in combination with gemcitabine and cisplatin, for the treatment of nasopharyngeal carcinoma and recommended a change in a manufacturing quality process. In December 2021, the FDA placed a hold on Pfizer’s fordadistrogene movaparvovec trials after the death of patient in a Phase Ib trial. The FDA allowed the trial to resume in April 2022. Sage and Biogen initiated a rolling NDA for zuranolone for the treatment of major depressive disorder. CHMP recommended approval of Amryt Pharma’s oleogel-S10 for the treatment of dystrophic and junctional epidermolysis bullosa. The FDA rejected oleogel-S10 in February 2022. Brickell sold marketing and development rights for sofpironium to Botanix Pharmaceuticals. Announced Research Updates Lilly announced that in the 72-week, 2,539 patient, Phase III, SURMOUNT-1 trial, adding tirzepatide to a reduced-calorie diet and increased physical activity resulted in a 16% weight loss with 5 mg, 21.4% with 10 mg and 22.5% with 15 mg compared to 2.4% reduction with placebo plus diet and increased activity in adults without type 2 diabetes who have obesity, or overweight with at least one of the following comorbidities: hypertension, dyslipidemia, obstructive sleep apnea or cardiovascular disease. At least a 5% reduction in body weight was achieved with tirzepatide in 89% with 5 mg and 96% with 10 mg and 15 mg compared to 28% with placebo. Novartis announced that in the 649 patient, Phase III, RATIONALE 306 trial, adding tislelizumab to chemotherapy improved overall survival compared to chemotherapy only in patients with advanced or metastatic esophageal squamous cell carcinoma. Novo announced that in the 26-week, 526 patent, Phase IIIa, ONWARDS 2 trial, once-weekly insulin icodec lowered HbA1c 0.93% compared to a 0.71% decrease with once-daily insulin degludec in patients with type 2 diabetes switching from daily insulin. Stealth announced that in the 48-week, 176 patient, Phase II, ReCLAIM-2 trial, treatment with elamipretide did not improve low luminance visual acuity nor geographic atrophy progression compared to placebo in patients with geographic atrophy secondary to dry age-related macular degeneration. Milestone announced that in the 169 patient. Phase III NODE-302 trial, a long term extension of the 431 patient, NODE-301 trial, 105 patients experienced 188 PSVT episodes with a conversion rate of 60.2% and a median time to conversion of 15.5 minutes with self-administered nasal etripam. In a 12-week, 302 patient, Phase III trial, treatment with CNTX-4975 did not reduce the Weekly Pain With Walking score in the index knee compared to placebo in patients with moderate-to-severe osteoarthritis knee pain. The results have not been announced, presented or published and are only available on clinicaltrials.gov Published Research Updates In the 52-week, 36 patient, Phase II/III, ASCEND trial, compared to placebo, treatment with olipudase resulted in an improvement in DLco (22% vs 3.0%), spleen volume (39% decrease vs 0.5% increase) and decrease in liver volume (28% vs 1.5%) in adult patients with acid sphingomyelinase deficiency (Niemann-Pick Disease). However, treatment with olipudase did not improve the Splenomegaly Related Score compared to placebo. In an interim analysis of a 1,153 patient, Phase IIb trial, vaccination with RSVpreF elicited an antibody response with transplacental transfer in pregnant women and their infants. In a ten patient, open-label, Phase II trial, treatment with clazakizumab for at least 2.5 years stabilized eGFR at 41.6 ml/min per 1.73 m(2) at 12 months and 38.1 ml/min per 1.73 m(2) at 24 months in kidney transplant patients with chronic active antibody-mediated rejection. Regulatory Update
The FDA granted Fast Track status to ADI-001 for the treatment of relapsed or refractory B-cell Non-Hodgkin’s lymphoma. PLOS One has retracted five articles describing research evaluating simufilam for the treatment of Alzheimer’s disease (AD). According to the New York Times (NYT), the studies were retracted due to questions regarding the validity of the results. NYT interviewed nine AD expert and all expressed concern over the design of the trials and the accuracy of the results. The FDA accepted the NDA for daprodustat for the treatment of anemia of chronic kidney disease (CKD) and assigned a PDUFA date for 2/1/2023 The FDA accepted the resubmitted BLA for daxibotulinumtoxinA for the treatment of moderate to severe glabellar lines and set a PDUFA date for 9/8/2022. The FDA accepted the BLA for tremelimumab, given with durvalumab, for the treatment of unresectable hepatocellular carcinoma suggesting a PDUFA date for 10/25/2022. CHMP recommended approval of mosunetuzumab for the treatment of relapsed or refractory follicular lymphoma after failure of at least two prior systemic therapies. Announced Research Updates Mirati announced results from a Phase I/II trial, evaluating the combination of sitravatinib plus nivolumab in the treatment of advanced clear cell renal cell cancer. Published Research Updates Results were published from a Phase II trial evaluating the outcome from adding cilofexor and/or firsocostat to semaglutide in patients with NASH. The Phase III, Chinese RATIONALE 302 trial results were published describing a comparison of treatment with tislelizumab to investigator’s choice of paclitaxel, docetaxel or irinotecan (in the treatment of advanced or metastatic esophageal squamous cell carcinoma. Results from a Phase II trial were published evaluating pixantrone in the treatment of refractory HER2-negative metastatic breast cancer. The SURPASS-3 CGM sub-study results were published, comparing tirzepatide to insulin degludec, for maintainingblood glucose in the target range of 71-140 mg/dL in patients with type 2 diabetes. Regulatory Update
TG Therapeutics voluntarily withdrew the pending BLA/sNDA for the combination of ublituximab and umbralisib for the treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma due to lower overall survival compared to obinutuzumab plus chlorambucil in the UNITY-CLL trial. TG Therapeutics also voluntarily withdrew umbralisib (Ukoniq) from the market. Umbralisib had been approved in February 2021, under an accelerated approval, for the treatment of relapsed or refractory marginal zone lymphoma or refractory follicular lymphoma. The FDA designated celiprolol a Breakthrough Therapy for the treatment of vascular Ehlers Danlos Syndrome. Coeptis Therapeutics bought marketing and development rights for entolimod from Statera Biopharma. Announced Research Updates Nektar and BMS announced results from the PIVOT 09 and PIVOT 10 trials, along with a decision on future development of the combination. Published Research Updates ICER released a draft review of betibeglogene autotemcel for the treatment of beta thalassemia. Results were published for a Phase I/II trial, evaluating rilzabrutinib in the treatment of patients with immune thrombocytopenia (ITP). Regulatory Update
The FDA accepted the resubmitted BLA for eflapegrastim for the management of chemotherapy-induced neutropenia and set a PDUFA date for 9/9/2022. The FDA removed the hold on trials evaluating magrolimab and azacitidine, but left the hold on the magrolimab monotherapy trials in place. Saniona paused two Phase IIb trials evaluating tesofensine and metoprolol in the treatment of hypothalamic obesity and Prader-Willi syndrome in order to reduce operating expenses. Reata resubmitted an NDA for omaveloxolone for the treatment of Friedreich’s ataxia. Announced Research Updates Coherus BioSciences and Junshi Biosciences announced results from the JUPITER-02 trial, which evaluated the addition of toripalimab to gemcitabine plus cisplatin in the treatment of recurrent or metastatic nasopharyngeal carcinoma. Merck announced results from the cervical cancer cohort of the KEYVIBE trial, which was evaluating the combination of vibostolimab plus pembrolizumab. Lilly announced ADhere trial results, which evaluated adding lebrikizumab to topical corticosteroids in the treatment moderate-to-severe atopic dermatitis. Published Research Updates Two Phase III trials evaluating an investigational oral drug to an established parenteral drug were published. The ASCEND-ID trial compared oral daprodustat to parenteral darbepoetin alfa on maintenance of hemoglobin levels in CKD patients receiving incident dialysis. The ADAPT-PO trial compared oral tebipenem to intravenous ertapenem in the treatment of complicated urinary tract infection or acute pyelonephritis. Last week we added 15 new drugs to the knowledgebase:
Regulatory Update
The FDA rejected vadadustat due to cardiovascular, thromboembolic and hepatotoxicity safety concerns and recommended new trials to assess safety. The FDA extended the PDUFA date for vutrisiran by three months to 7/14/2022, for the treatment of transthyretin-mediated (ATTR) amyloidosis, to allow time to review information on a new packaging and labeling facility. The FDA extended the PDUFA date for ublituximab for the treatment of CLL and small lymphocytic lymphoma (SLL) by three months to allow additional time to review new data. The new PDUFA date is 6/25/2022. The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 to recommend the FDA reject AMX0035 or the treatment of ALS and recommended an additional trial to evaluate efficacy. The FDA accepted the NDA for futibatinib for the treatment of locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions and set a PDUFA date for 9/30/2022. ImmunoGen submitted a BLA for mirvetuximab soravtansine for the treatment of folate receptor alpha (FRα)-high platinum-resistant ovarian cancer that has been previously treated with one to three prior systemic treatments. The FDA placed a partial hold on leronlimab HIV trials and a full hold on leronlimab COVID-19 trials in the United States. At the same time, CytoDyn decided to place a hold on its leronlimab COVID-19 Brazilian trial pending review of two cardiac events by the data safety monitoring board. Announced Research Updates Boehringer Ingelheim announced 12-week data for the spesolimab Effisayil 1 trial. Roche announced from the SKYSCRAPER-02 trial, evaluating the addition of tiragolumab to atezolizumab, carboplatin, and etoposide in patients with extensive-stage small cell lung cancer. Cytokinetics announced results from cohort III of the REDWOOD-HCM trial, evaluating aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy. BMS announced results for the VALOR-HCM trial evaluating the effect of mavacamten on the need for septal reduction therapy in patients with symptomatic obstructive hypertrophic cardiomyopathy. BMS also announced long-term-extension results from the EXPLORER-LTE trial, evaluating mavacamten in the treatment of patients with NYHA Class II or III obstructive hypertrophic cardiomyopathy. Published Research Updates H. pylori eradication with vonoprazan compared to lansoprazole results were published. Harvard researchers discuss the estimated cost effective prices for aducanumab and donanemab in a new analysis. A comparison of anetumab ravtansine to vinorelbine in the treatment of unresectable locally advanced or metastatic disease overexpressing mesothelin was published. Results were published for the GOAL trial evaluating pixantrone plus obinutuzumab in the treatment of diffuse large B-cell lymphoma. The efficacy of CTP-543 in the treatment of moderate to severe alopecia areata was published Results were published for the ROCKIES trial, comparing oral roxadustat to parenteral epoetin alfa in patients with dialysis-dependent CKD. Regulatory Update
The FDA approved lutetium Lu 177 vipivotide tetraxetan (Pluvicto, Novartis), on 3/23/2022, for the treatment of prostate-specific membrane antigen–positive metastatic castration-resistant prostate cancer. Lutetium Lu 177 vipivotide tetraxetan was formerly referred to as 177Lu-PSMA-617. The FDA has also approved gallium Ga 68 gozetotide injection (Locametz, Novartis), a radiolabeling diagnostic agent used to identify PSMA-positive lesions through a positron emission tomography (PET) scan. The FDA followed the ODAC’s recommendation and rejected sintilimab and recommended a multiregional trial that uses a non-inferiority design with an overall survival endpoint before re-submitting the drug. The FDA rejected zandelisib and recommended a Phase III trial to support safety and efficacy. The FDA accepted the re-submitted BLA for teplizumab for the delay of clinical type 1 diabetes in at-risk patients and set a PDUFA date for 8/17/2022. An FDA review of AMX0035 found weak evidence to support efficacy due to loss of patients, the potential for unblinding from adverse effects and only a modest p-value using non-preferred analysis methods. The FDA did not find the open-label, long-term extension data to support a survival benefit. Announced Research Updates Protalix announced results from the Phase III BRIGHT trial, evaluating the impacet of switching Fabry disease patients from agalsidase alfa or beta to pegunigalsidase alfa ImmunoGen announced results from the SORAYA trial, evluating mirvetuximab soravtansine in the treatment of platinum-resistant ovarian cancer. Sangamo announced one-year interim results for four Fabry disease patients treated with a single infusion of the gene therapy, isaralgagene civaparvovec. Pfizer announced results from the ELEVATE UC 12 trial, evaluating etrasimod in the treatment of ulcerative colitis in patients who had failed or were intolerant to at least one conventional biologic, or JAK therapy. UCB announced open-label, long-term extension data from the Phase IIIB, BE RADIANT trial, bimekizumab in the treatment of moderate-to-severe chronic plaque psoriasis. Lilly announced the Phase III, ADvocate 1 & 2 trials, evaluating lebrikizumab in the treatment of moderate-to-severe atopic dermatitis. Published Research Updates The results from the Phase III, NRG-GY004 trial were published describing the effect of olaparib on the treatment ofplatinum-sensitive relapsed ovarian cancer. Long-term results from the explorer4 and explorer5 trials were published evaluating the effect of treatment with concizumab on the annualized bleeding rate in hemophilia A. |
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