The FDA approved bempedoic acid (Nexletol, Esperion) on 2/21/20 as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD). Esperion plans to price bempedoic acid around $10 a day. Bempedoic acid will be commercially available on 3/30/2020 and Esperion will offer a prescription reduction program that will reduce the copay to $10 for up to a 3-month supply.
The FDA approved eptinezumab (Vyepti, Lundbeck) on 2/21/20 for migraine prevention in adults. Eptinezumab is administered as a 30-minute IV infusion every 3 months. Lundbeck plans to have eptinezumab available in April 2020.
The FDA approved meloxicam injection (Anjeso, Baudax Bio) for the treatment of moderate to severe pain in adults, as monotherapy or concomitantly with other non-NSAID analgesics. Meloxicam injection is given as a once-daily intravenous injection.
The FDA accepted the BLA for valoctocogene roxaparvovec and set a PDUFA date of 8/21/2020. BioMarin indicated it may change between $2 million and $3 million for a dose of valoctocogene roxaparvovec.
The FDA accepted the NDA for lurbinectedin for the treatment of small cell lung cancer that relapsed after platinum-based therapy and set a PDUFA date of 8/16/2020.
The FDA accepted the NDA for berotralstat for the prevention of hereditary angioedema (HAE) attacks and set a PDUFA date of 12/3/2020.
An FDA advisory committee will review Viaskin Peanut patch on May 15.
Bluebird delayed its launch of beta beglogene darolentivec in Europe until February 2020 in order to finalize large scale manufacturing. The FDA requested additional information on beta beglogene darolentivec; Bluebird has delayed submission of a BLA to the end of 2020.
Announced Research Updates
BrainStorm is evaluating mesenchymal stem cells neurotrophic growth factors (MSC-NTF) in a 200 patient, Phase III trial as a treatment for faster-progressing amyotrrophic lateral sclerosis (ALS) and an open-label Phase II, progressive multiple sclerosis (MS) trial. BrainStorm expects topline results from both trials to be available in 4Q20.
NGM announced that in a 24-week, 78 patient, Phase II trial, treatment with aldafermin resulted in a 39% decrease in liver fat content compared to 13% decrease with placebo in patients with biopsy-confirmed NASH with F2-F3 liver fibrosis.
Published Research Updates
The 204 patient, Phase III, ENDEAVOR trial was discontinued after an average follow-up of 6.7 months when mortality was higher with revusiran than placebo (12.9% vs 3.0%) in patients with cardiomyopathy caused by hereditary transthyretin-mediated amyloidosis.
In a 4-week, 70 patient, Phase II trial, treatment with nemolizumab reduced the pruritus scale score by 4.5 points compared to a 1.7 point decrease with placebo in patients with moderate-to-severe prurigo nodularis and severe pruritus.
In a 78 patient Phase II trial, tivantinib plus cetuximab did not improve the overall response rate compared to cetuximab monotherapy in patients with cetuximab-naive, platinum-refractory head and neck squamous cell carcinomas.
In the 4-week, 71 patient, Phase II, PROGRESS-HF trial, treatment with elamipretide did not improve left ventricular end systolic volume compared to placebo in patients with heart failure with reduced ejection fraction (HFrEF).
In a 42-day, 227 patient, Phase IIb trial, treatment with sofpironium resulted in at least a one-grade improvement in the Hyperhidrosis Disease Severity Measure-Axillary score in 70% of patients treated with 5% gel, 79% with a 10% gel, 76% with a 15% gel compared to 54% with placebo in patients with axillary hyperhidrosis.
In the 12-week, 1,518 patient, Phase III, EMPOWUR trial, treatment with vibegron reduced micturitions per 24 hours by 1.8 episodes/day compared to a 1.3 episode decrease with placebo and a 1.6 episode decrease with tolterodine (non-significant difference) in patients with overactive bladder.
In a 24-month, 275 patient, Phase IIb trial, treatment with nelipepimut-S plus sargramostim did not improve disease-free survival compared to sargramostim monotherapy in patients with HER2 low-expressing breast cancer who were receiving trastuzumab.
In the 12-week, 888 patient, Phase III, PROMISE-1 trial, eptinezumab reduced monthly migraine days by 4.0 days with 30 mg, 3.9 days with 100 mg and 4.3 days with 300 mg compared to a 3.2 days reduction with placebo in patients with episodic migraine.
In a 113 patient, Phase I, open-label trial, treatment with mirvetuximab soravtansine and bevacizumab resulted in an ORR of 39% and median PFS of 6.9 months in patients with FRα-positive, platinum-resistant ovarian cancer.
In a 42 patient, Phase II trial, 52.4% of patients treated with lurbinectedin achieved progression free survival at 12 weeks in patients with progressive malignant pleural mesothelioma.
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The FDA approved lactitol (Pizensy, Braintree Laboratories) on 2/13/2020 for the treatment of chronic idiopathic constipation in adults.
The FDA accepted the NDAs for:
Trevena resubmitted an NDA for oliceridine for the management of moderate-to-severe acute pain.
Announced Research Updates
Lilly announced that in the 5-year, 194 patient, Phase II/III, DIAN-TU trial, neither solanezumab nor gantenerumabslowed the loss of cognition compared to placebo in patients at risk for dominantly inherited Alzheimer's disease.
Abeona announced interim results from 3 patients enrolled in the Phase I/II, open-label, Transpher A trial, where treatment with ABO-102 resulted in neurocognitive development within normal age equivalent parameters 18-24 months post treatment in patients with Sanfilippo syndrome type A (MPS IIIA).
Abeona announced interim results from 7 patients enrolled in the Phase I/II, open-label, Transpher B trial, where treatment with ABO-101 resulted in improvements in biomarkers associated with abnormal accumulation of glycosaminoglycans in the brain and throughout the body neurocognitive development within normal age equivalent parameters 18-24 months post treatment in patients with Sanfilippo syndrome type B (MPS IIIB).
Published Research Updates
In a 6-month, 76 patient, Phase II, open-label trial, treatment with bevacizumab plus buparlisib resulted in progression free survival of 4 months and an overall response rate of 26%, which is similar to historical bevacizumab monotherapy, but with a higher rate of adverse effects in the treatment of patients with relapsed/refractory glioblastoma.
In a 27 patient, Phase Ib/II, open-label trial, treatment with tivozanib resulted in a progression-free interval of 24 weeks and an overall response rate of 21% in patients with inoperable hepatocellular carcinoma.
In a 4-week, 375 patient, Phase IIb, dose-ranging trial, treatment with ensifentrine improved FEV1 compared to placebo by 146mL with 0.75mg, 153 mL with 1.5 mg, 200 mL with 3 mg and 139 mL with 6 mg in patients with COPD.
In a 7-week, 450 patient, Phase II, dose-ranging trial, mirogabalin did not improve the average daily pain score compared to pregabalin or placebo in Asian patients with diabetic peripheral neuropathic pain.
In the 253 patient, Phase II, INCREASE trial, treatment with icotinib 250 mg TID resulted in a progression-free survival of 12.9 months compared to 9.2 months with icotinib 125 mg TID in patients with non-small cell lung cancer harboring 21-L858R mutation.
The FDA accepted the BLA for Revance’s daxibotulinumtoxinA for the treatment of moderate to severe glabellar lines and set a PDUFA date of 11/25/2020.
Mesoblast completed a rolling BLA for remestemcel-L in February 2020 for the treatment of pediatric steroid resistant graft versus host disease.
CorMedix initiated a rolling NDA for taurolidine in February 2020.
The EMA accepted the MAA for GlaxoSmithKline’s belantamab mafodotin to treat multiple myeloma.
Announced Research Updates
Roche announced that in the 12-month, 180 patient, Phase III SUNFISH trial, treatment with risdiplam increased the Motor Function Measure 32 (MFM32) score by 1.59 points compared to placebo in patients with Type 2 or 3 spinal muscular atrophy.
Lilly announced that in the 16-week, Phase III, BREEZE-AD5 trial, 29.5% of patients treated with baricitinib 2 mg achieved a 75% or greater change from baseline in their Eczema Area and Severity Index (EASI-75) compared to 8.2% with placebo in patients with moderate to severe atopic dermatitis. Improvement with a 1 mg dose of baricitinib did not reach significance.
Biohaven Pharmaceutical announced that in an 8-week, 402 patient, Phase III trial, treatment with troriluzole was no different that placebo in improving the Hamilton Anxiety Rating Scale (HAM-A) in patients with Generalized Anxiety Disorder (GAD).
Published Research Updates
In the 34-week, 301 patient, Phase III REAL 1 trial, treatment with somapacitan reduced the truncal fat percentage an additional 1.53% compared to placebo in adults with growth hormone deficiency. In a 52-week, open-label extension of REAL 1, truncal fat reduction was maintained with somapacitan and growth hormone at 86 weeks.
In a 6-month, 54 patient, Phase III, open label trial, 70.4% of patients treated with remestemcel-L had an overall response to the treatment at day 28 compared to a historical rate of around 45% in children with acute graft-versus-host disease that was refractory to steroids. At 180 days, the overall survival rate was 78.9% in responders at day 28 compared to 47.1% in non-responders.
In the Phase Ib, open-label TATTON trial, treatment with osimertinib plus savolitinib resulted in 79/138 patients with a mixed treatment history achieving an objective partial response and 23/42 patients that had not previously received a third-generation EGFR TKI and were Thr790Met negative achieving an objective partial response in patients with locally advanced or metastatic, MET-amplified, EGFR mutation-positive non-small-cell lung cancer, who had progressed on EGFR TKIs.
In a 112 patient Phase I, open label trial, treatment with enfortumab vedotin resulted in a 43% objective response rate in patients with Nectin-4-expressing urothelial carcinoma who progressed on at least 1 prior chemotherapy regimen and/or anti-PD-(L)1 therapy.
In the 462 patient, Phase III ADAPT trail, treatment with Rocapuldencel-T in combination with sunitinib did not improve overall survival compared to sunitinib monotherapy in patients with metastatic clear cell renal cell carcinoma.
In a 6-month, 73 patient, Phase II trial, 28% of patients treated with rindopepimut plus bevacizumab achieved progression-free survival at 6-months compared to 16% with bevacizumab in patients with recurrent EGFRvIII-positive glioblastoma.
In the 48-week, 203 patient, Phase IIb, BE ACTIVE trial, ACR50 was achieved in 26.8% of patients treated with 16 mg of bimekizumab, 41.5% with 160 mg, 46.3% with 160 mg after a 320-mg loading dose, and 24.4% who received 320 mg compared to 7.1% with placebo in patients with psoriatic arthritis. Patients from the 16 mg and placebo groups were then randomized to one of the higher doses for 36 weeks. At 48 weeks, ACR50 had been achieved by 55% with 160mg, 57% with 160 mg after a 320 mg loading dose and 63% with the 320 mg dose with improvements seen in the 16 mg and placebo patients randomized to higher doses of bimekizumab.
A published BioMarin funded cost-effectiveness analysis found the lifetime cost to treat hemophilia A as $23.5 million with prophylactic factor VIII and $16.7 million with valoctocogene roxaparvovec for a savings of $6.8 million per patient. ICER is currently working on an analysis of valoctocogene roxaparvovec compared to emicizumab and factor VIII. SPK-8011, another hemophilia gene therapy and fitusiran, an RNA interference drug for the treatment of hemophilia A and B were not included in the current scoping document draft.
Sanofi announced that in a 52-week, 36 patient, Phase II/III trial, treatment with olipudase resulted in a relative improvement of 22% in the predicted diffusing capacity of carbon monoxide (DLco) and a 39.5% decrease in spleen size compared to a 3% DLco improvement and a 0.5% decrease in spleen size with placebo in adult patients with acid sphingomyelinase deficiency (Niemann-Pick Disease). However, treatment with olipudase did not improve the Splenomegaly Related Score compared to placebo. Sanofi announced that in a 52-week, 20 patient, open-label, Phase II trial, treatment with olipudase resulted in a relative DLco improvement of 33% and a 49% decrease in spleen size in patients < 18 years old with acid sphingomyelinase deficiency.
The NIH announced that it halted a trial after interim data from 5,383 patients enrolled in a 5,407 HIV-negative patient, Phase IIb/III trial, showed that 129/2,694 patients who received the Sanofi Pasteur vaccine with the GSK immunity-boosting adjuvant became infected with HIV compared to 123/2,689 patients that received placebo.
Published Research Updates
In a 12-week, 111 patient, Phase IIb trial, several dosing regimens of tirzepatide lowered HbA1c more than placebo in patients with type 2 diabetes.
In a 13-week, 42 patient, Phase II, open-label trial, treatment with bermekimab resulted in 61% of patients naive to anti-TNF treatment and 63% of patients that failed anti-TNF treatment achieving Hidradenitis Suppurativa Clinical Response (HiSCR), which was defined as a greater than or equal to 50% reduction in inflammatory lesion count and no increase in abscesses or draining fistulas.
In an 8-week, 204 patient, Phase II trial, 33.3% of patients treated with serlopitant achieved a 4 point or > worst-itch numeric rating scale (WI-NRS) score compared to 21.1% with placebo in patients with pruritus associated with psoriasis.
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