Pipeline News and Updates
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Regulatory Update
The FDA approved teprotumumab (Tepezza, Horizon Therapeutics) on 1/21/2020, for the treatment of thyroid eye disease. Horizon has set WAC for teprotumumab at $14,900 per vial. A full 6-month course of treatment will require 23 vials for a total treatment WAC of $343,000 and an estimated price after discount of $200,000. Teprotumumab was approved 6 weeks early. The original PDUFA date was 3/8/2020. The FDA approved tazemetostat (Tazverik, Epizyme) on 1/23/20, for the treatment of metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. Tazemetostat is given orally twice a day. Epizyme set WAC at $15,500 for a one-month supply of tazemetostat. GlaxoSmithKline’s belantamab mafodotin was granted a priority review for the treatment of heavily pre-treated patients with relapsed or refractory multiple myeloma. The drug has a projected summer 2020 PDUFA date. The FDA designated AstraZeneca’s tremelimumab an Orphan Drug when given in combination with durvalumab for the treatment of hepatocellular carcinoma. GlaxoSmithKline licensed the M72/AS01E tuberculosis vaccine to the Bill & Melinda Gates Medical Research Institute (MRI). Gates MRI will assume responsibility for development of the vaccine for use in low-income countries with high TB rates. Announced Research Updates Roche announced that in the 12-month, 41 patient, Phase III open-label FIREFISH trial, treatment with risdiplam increased the number of infants sitting without support for at least five seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) in patients with Type 1 spinal muscular atrophy. BeiGene announced interim results from a 360 patient, open-label, Phase III trial, in which progression-free survival was improved in patients treated with tislelizumab plus paclitaxel and carboplatin or tislelizumab plus nab-paclitaxel and carboplatin compared to paclitaxel and carboplatin alone in patients with squamous non-small cell lung cancer. Ipsen paused two fibrodysplasia ossificans progressiva (FOP) studies when an independent data monitoring committee concluded the current protocol was unlikely to achieve its objective and the protocol and efficacy analysis may need to be modified. Halozyme announced that in a 494 patient, Phase III trial, treatment with pegvorhyaluronidase alfa plus gemcitabine and nab-paclitaxel did not improve overall survival compared to gemcitabine and nab-paclitaxel (11.2 months vs 11.5 months) alone in patients with metastatic, hyaluronan-high pancreatic ductal adenocarcinoma. Published Research Updates In a 50 patient, Phase II trial, veliparib added to cisplatin and gemcitabine did not improve the response rate compared to cisplatin and gemcitabine alone in patients with BRCA/PALB2+ pancreatic ductal adenocarcinomas. Regulatory Update
Oxycodegol has reduced permeability across the blood-brain barrier, so it may cause less euphoria and sedation. In July 2019, the FDA put review of NKTR-181 on hold and canceled an advisory committee review of the drug. On 1/14/2020 the FDA's Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee voted 27-0 against approval, due to abuse potential of the drug and lack of adequate efficacy studies. Nektar Therapeutics withdrew the NDA and ceased development of oxycodegol due to the decision. Roche indicated they plan to price risdiplam lower than nusinersen and onasemnogene abeparvovec when the drug is approved. Risdiplam has a PDUFA date of 5/24/2020. The EMA approved osilodrostat (Isturisa) in January 2020 for the treatment of endogenous Cushing’s syndrome. Sarepta initiated a rolling NDA submission for casimersen for the treatment of Duchenne muscular dystrophy amenable to exon 43 skipping. TG Therapeutics initiated a rolling NDA submission for umbralisib for patients with previously treated marginal zone lymphoma and follicular lymphoma. Published Research Updates In the 12-month, 362 patient, Phase III, TULIP 2 trial, 47.8% of patients treated with anifrolumab had a decrease in disease activity (BICLA response) compared to 31.5% with placebo in patients with systemic lupus erythematosus. In two randomized, crossover, dose-escalation studies, treatment with gefapixant in doses of 30 mg or > decreased cough frequency and severity compared to placebo in patients with refractory chronic cough. Regulatory Update
The FDA approved avapritinib (Ayvakit, Blueprint Medicines Corp) on 1/9/20 for the treatment of unresectable or metastatic gastrointestinal stromal tumors (GIST) that harbor a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. Incyte licensed tafasitamab from MorphoSys and will co-promote the drug in the U.S. and exclusively market the drug outside the U.S. Announced Research Updates Apellis announced that in the 16-week, 80 patient, Phase III, PEGASUS trial, patients treated with pegcetacoplan had a 2.4 g/dL increase in hemoglobin compared to a 1.5 g/dL decrease with eculizumab in patients with paroxysmal nocturnal hemoglobinuria. Blueprint announced that in the 80 patient, Phase I/II, open label, ARROW trial treatment with pralsetinib resulted in a 61% ORR in patients with RET fusion-positive non-small cell lung cancer previously treated with platinum-based chemotherapy. Pralsetinib is available in an expanded access program through NCT04204928. DBV Technologies announced that in the 3-year, 141 patient, open-label PEOPLES trial extension of the PEPITES trial, 75.9% of patients that received a Viaskin peanut patch increased the amount peanut protein they tolerated and 51.8% could tolerate at least 1,000 mg peanut protein. Published Research Updates In the 26-week, 59 patient, Phase II REAL 3 trial, height velocity achieved with weekly somapacitan (doses 0.04, 0.08 and 0.16 mg/kg/week) was 8 to 12.9 cm/year compared to 11.4 cm/year with daily growth hormone (0.034 mg/kg/day) in children with with growth hormone deficiency. In a 26-week extension (52 weeks total) of the REAL 3 trial, height velocity achieved with weekly somapacitan was 4.72 to 8.6 cm/year compared to 7.41 cm/year with daily growth hormone. In October 2019, I completed an Ironman in Louisville Kentucky. It was a day that started in the 40’s, got to 70 and dropped back into the 50’s. Finishing an Ironman involves a lot of the same skills that allow us to be successful in our professions. Each day you put out your best effort and stay consistent with your plan. You can’t skip a training day due to rain, just like you can’t forget when that report is due. You must be ready to make changes in order to deal with obstacles. In addition to weather issues, you may have to find ways to overcome a mechanical, health or nutrition issue while in business you may need to deal with data loss, resource allocation or logistics. Once you start the race (or project) you don’t stop until you reach the finish.
There are no problems, there are only opportunities looking for solutions. And always enjoy the journey on the way to the finish. Race Overview (1 minute) Race Highlights (3 minutes) Regulatory Update
The FDA approved seven new drugs during December 2019, bringing the total new approvals for the year to forty-four. During the month the agency took another nineteen actions. There are two investigational drugs with PDUFA Dates in January: Aimmune Therapeutics’ AR101 for Peanut Allergy and Epizyme’s Tazemetostat for Epithelioid sarcoma. AR101 has three FDA Priority Designations: Orphan Drug, Breakthrough Therapy, and Fast Track. For a complete review of January’s potential blockbusters order Prescribe Right’s Pharmacy & Therapeutics Committee Forecast (PDF document and slides). The current forecast coverage period is January 1, 2020 through March 31, 2020. Each forecast contains a review of investigational drugs with PDUFA Dates during each calendar quarter. Contents include generic name, brand name (if available), mechanism of action, ADRs, route, indication(s), PDUFA Date, FDA Priority Designations (if any), efficacy and a general comment from our Founder and CEO Scot Walker, PharmD, MS, BCPS, BCACP. Click here for ordering details Seven New December Approvals
Regulatory Update
Spectrum withdrew the BLA for eflapegrastim in March 2019 to provide more information to the FDA on the chemistry, manufacturing and controls process. The FDA accepted the BLA for eflapegrastim for the management of chemotherapy-induced neutropenia in December 2019 and set a PDUFA date of 10/24/2020. The FDA followed the recommendation of the advisory committee and rejected vernakalant due to safety concerns. The FDA requested a trial to assess the cardiovascular risk and that Correvio develop a process to identify patients at low risk of adverse cardiovascular adverse events. Melinta Therapeutics filed for bankruptcy in December 2019. Melinta is developing solithromycin, a drug for macrolide resistant bacteria. The FDA rejected solithromycin in December 2016. Urovant submitted an NDA for vibegron for the treatment of patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency. Announced Research Updates Orphazyme announced that in an open-label extension of the Niemann-Pick disease trial, 41 patents completed an additional 12-months of treatment. Patients switched from placebo to arimoclomol saw a reduction in NPC-CSS, while patients that continued on arimoclomol had a progression in NPC-CSS, primarily from patients < 4 with aggressive disease. Incyte announced that in a 28-day, 439 patient, Phase III trial, treatment with itacitinib and corticosteroids did not improve overall response rate compared to placebo (74% vs 66%) in patients with treatment-naive acute graft-versus-host disease. Published Research Updates A meta analysis of serelaxin heart failure trials concluded that while serelaxin reduced the risk of 5-day worsening heart failure and changes in renal function markers, it did not decrease length of stay, cardiovascular death, heart or renal failure. In the 72-week, 318 patient, Phase IIb, ENCORE-NF trial, emricasan did not improve the CRN fibrosis stage without suffering a worsening of steatohepatitis compared to placebo in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH) and liver fibrosis. In a 3-year, 15 patient, Phase I/II, open-label trial, treatment with valoctocogene roxaparvovec eliminated annualized treated bleeding events and factor VIII infusions in patients with hemophilia A. |
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