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Pipeline News and Updates
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Prescribe Right Pharmaceutical Pipeline Update for September 26

9/26/2018

 
Thirty-one citations were added to our knowledgebase bringing the total to 809. Each citation is accessible from the single drug look-up monograph via a URL link. There are now 612 investigational drugs in the Knowledgebase.

Two Updates on products with PDUFA Dates and/or Priority Designations
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended approval of Lilly’s galcanezumab. The FDA has Fast-Tracked the drug with a review date next week, September 30, 2018.

J&J announced that in a 4-week, 346 patient, Phase III trial, esketamine nasal spray added to a new anti-depressant did not improve Montgomery-Asberg Depression Rating Scale (MADRS) total score compared to monotherapy with a new antidepressant in patients with treatment-resistant depression. No PDUFA date but the drug has a Breakthrough Therapy Priority Designation.

Five additional updates:
  • A pooled analysis of 3,492 patients in 8 trials involving baricitinib (Lilly, Incyte) found mortality, cardiovascular events and serious infection rates similar to placebo. Brand name: Olumiant.
  • Astellas announced topline results from a 52-week, Phase III trial in which roxadustat achieved a hemoglobin response rate in the first 24 weeks and hemoglobin change from baseline at Weeks 28 to 52 compared to placebo in CKD patients with anemia. 
  • Novo Nordisk announced that in a Phase III trial, daily oral semaglutide lowered HbA1c 1.8% with 14mg, 1.4% with 7mg and 0.7% with 3 mg compared to a 1.3% reduction with weekly dulaglutide 0.75 mg in Japanese type 2 diabetic patients. The effect on weight with semaglutide was a 1.9 kg loss with 14 mg, 1 kg loss with 7mg and a 0.1 kg weight gain with 3 mg compared to a 1.1 kg gain with dulaglutide.
  • In a 390-patient, open label trial involving dengue naive US adults, use of a compressed vaccination schedule (0-2-6 months) was comparable to the standard scheduled (0-6-12 months) given with or without concomitant yellow fever vaccine. Patients that received the compressed schedule were 73.4% seropositive for ≥3 serotypes and 50% seropositive for all four serotypes compared to 82.4% and 42.6% for the standard schedule.
  • In a 37-month, 1,147 patient, Phase III trial, treatment with Novartis’ buparlisib plus Astra Zeneca’s fulvestrant (FDA approval date 11/15/2017) was no better than fulvestrant monotherapy in overall survival of the total population or patients with PIK3CA-mutant ctDNA.

You can access up-to-date information on all drugs in the late stages of development with the 
Prescribe Right Pharmaceutical Pipeline Tracker.

Prescribe Right Pharmaceutical Pipeline Update for September 20

9/20/2018

 
Update on investigational drugs with recent and upcoming PDUFA dates:
  • The FDA approved Teva’s fremanezumab (Brand name: Ajovy) for the prevention of migraines, on 9/14/18, two days ahead of its PDUFA date. Fremanezumab will be available in pre-filled syringes dosed as 225mg monthly or 675mg (3 x 225mg doses) every 3 months. Teva has set the Wholesale Acquisition Cost (WAC) at $575 per syringe. A coupon program to reduce the co-pay is also being launched. The drug had a Fast Track Priority Designation.
  • Jazz announced that in patients with narcolepsy or obstructive sleep apnea, solriamfetol-treated patients in Phase III Trials had an improvement in wakefulness and a decrease in sleepiness compared to placebo. Jazz also announced a subset of 280 patients participated in a 2-week withdrawal trial, where patients switched from solriamfetol to placebo had a decrease in wakefulness and an increase in sleepiness. PDUFA date Dec 20, 2018, Orphan Drug Priority Designation.
  • Acacia Pharma’s amisulpride for post-operative nausea and vomiting, has a PDUFA date of 10/5/18. Trade name: Barhemsys
Plus news about nine other drugs in development:
In a Phase II trial, bimagrumab (Novartis) treated patients with sporadic inclusion body myositis had an increase in thigh muscle volume, but not 6-minute walk distance. Bimagrumab has a Breakthrough Therapy Priority Designation.

Patients with rheumatoid arthritis that had achieved sustained control with 4 mg baricitinib (Lilly, Incyte) when given a reduced dose of 2mg showed a low disease activity rate in 67% vs 80%, remission in 33% vs 40%, relapse in 37% vs 23% and need for rescue medication in 18% vs 10%.

 In a Phase III trial, postmenopausal women were treated with romosozumab (Amgen, UCB) or placebo for 1 year, then denosumab for 2 years. After 36 months 1% of romosozumab patients had a vertebral fracture compared to 2.8% with placebo. In another Phase III trial, patients were treated with either romosozumab or alendronate for one year, then all patients received alendronate for an additional year. After 24 months. romosozumab patients had an incidence of vertebral and clinical fractures of 6.2% and 9.7% compared to alendronate patients’ incidence of 11.9% and 13%. In a complicated extension trial with multiple treatment combinations, there was evidence to suggest further increase in BMD, in patients that received 12 months of denosumab after receiving 24 months of romosozumab. In a 245 male patient, Phase III trial, romosozumab increased BMD in the spine and hip compared to placebo in men with osteoporosis. 

In a Phase III trial, more patients with moderate-to-severe rheumatoid arthritis treated with filgotinib (Galapagos, Gilead) 200 mg achieved AR20 (69 vs 55%), AR50 (46 vs 35%) and AR70 (32 vs 20%) vs placebo.

In a 12-week, dose ranging, Phase II trial, of patients with moderate-to-severe psoriasis, treatment with daily doses of BMS-986165 resulted in lower Psoriasis Area and Severity Index score than placebo.

Multiple updates for risankizumab (AbbVie, Boehringer Ingelheim):
  •  In Phase II trial, 24% to 37% of patients with Crohn’s Disease treated with risankizumab reached remission (CDAI score < 150) compared to 15% with placebo. 
  • In a Phase II trial, 77% of patients with moderate-to-severe plaque psoriasis, treated with risankizumab had a 90% reduction in their PASI compared to 40% treated with ustekinumab. After 9 months, 69% of patients with moderate-to-severe plaque psoriasis maintained clear or almost clear skin in the higher dose risankizumab group compared to 30% of patients in the placebo group. 
  • In a Phase III, 75% of risankizumab treated patients achieved PASI 90 compared to 42% treated with ustekinumab and 5% with placebo in patients with moderate-to-severe chronic plaque psoriasis. 
  • In a Phase III trial, of patients with moderate-to-severe chronic plaque psoriasis, 75% of risankizumab-treated patients achieved PASI 90 compared to 47% treated with ustekinumab and 2% with placebo. Pooled data from trials found that at 12-months, 56% of patients treated with risankizumab reported being symptom free measured with the patient reported psoriasis symptom scale (PSS) compared to 30% with ustekinumab. 
  • In another Phase III trial, 72% of risankizumab-treated patients achieved PASI 90 compared to 47% treated with adalimumab. 
  •  A presentation at ACR 2017 described 16-week results from a Phase II psoriatic arthritis trial where 60% of risankizumab patients achieved ACR20 compared to 36% with placebo. A reduction in psoriatic arthritis severity as measured by PASI75 was achieved in 67%-75% of risankizumab patients compared to 10% with placebo. 
  •  In a Phase III trial of patients with active ankylosing spondylitis, patients treated with risankizumab did not have at least a 40% improvement in the Assessment in SpondyloArthritis International Society (ASAS40) compared to placebo.
  • In a Phase III trial, Vital Therapies’ VTL C3A, with an Orphan Drug Priority Designation, failed to demonstrate a benefit in survival compared to placebo in patients with severe alcoholic hepatitis.
Multiple updates for upadacitinib (AbbVie):
  • AbbVie announced that in a phase II trial, upadacitinib, with a Breakthrough Therapy Priority Designation, reduced endoscopic remission in Crohn's patients more than placebo. 
  • In a Phase III trial of patients with moderate-to-severe rheumatoid arthritis that had an inadequate response to conventional DMARDs, ACR20 was achieved by 64-66% of patients for the 15 mg and 30 mg doses of upadacitinib compared to 36% with placebo. 
  • AbbVie announced that in a Phase III trial, complete remission was reached by 41% of rheumatoid arthritis patients that received upadacitinib 30 mg compared to 8% continuing on methotrexate. 
  • AbbVie announced that in a Phase III trial, ACR20 was achieved by 71% of rheumatoid arthritis patients that received upadacitinib compared to 63% on adalimumab and 36% on placebo. Clinical remission was reached by 71% in upadacitinib patients compared to 29% on adalimumab and 14% on placebo. 
  • AbbVie announced interim results at 32 weeks in a Phase IIb trial, where patients with moderate or severe atopic dermatitis patients treated with upadacitinib had a 48% (7.5mg dose), 44% (15mg dose) and 68% (30mg dose) improvement in their Eczema Area and Severity Index score compared to 34% with placebo. 
  • AbbVie announced that in a Phase III trial, ACR50 was achieved by 60-66% of rheumatoid arthritis patients that received upadacitinib compared to 33% that received methotrexate.​
Karyopharm announced that in a Phase IIb trial, treatment with selinexor in combination with dexamethasone resulted in a 26% overall response rate and a median survival of 8.6 months in patients with highly resistant multiple myeloma. Karyopharm initiated a rolling NDA in July 2018 and plans to file an MAA in early 2019. Selinexor is also being studied in combination with bortezomib in the treatment of resistant multiple myeloma. Karyopharm has an ongoing Phase III study evaluating selinexor, combined with bortezomib and dexamethasone in the treatment of resistant multiple myeloma with topline results available in 2019. Selinexor has an Orphan Drug Priority Designation.

Colucid announced a study of 1,545 migraine patients, in which 28-32% of patients treated with lasmiditan were migraine pain-free at 2 hours compared to 15% with placebo and 41% had resolution of their most bothersome symptoms vs 30% with placebo.
Lilly announced that in a Phase III trial, 31-39% of patients with an acute migraine treated with lasmiditan were pain-free at 2 hours compared to 21% with placebo. 44-49% of lasmiditan patients were free of their most bothersome symptoms at 2 hours.
​

You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.

Prescribe Right Pharmaceutical Pipeline Update for September 11

9/11/2018

 
Update on investigational drugs with recent PDUFA dates:
  • In a 1,064 patient, Phase III, CAPSTONE-1 trial, a single dose of baloxavir marboxil (Genentech, Shionogi) reduced the time to alleviation of symptoms of influenza infection by 26.5 hours compared to placebo and was similar to 5 days of oseltamivir. Baloxavir marboxil also reduced viral load more rapidly than oseltamivir or placebo. The drug’s PDUFA date is December 24, 2018, but has no Priority Designations at present. 
  • A phase II study showed a decrease in tumor volume and 4-5-month progression-free survival with talazoparib in resistant BRCA1/2 mutation breast cancer patients. Pfizer announced that in a 20 patient, Phase II trial of patients with BRCA+ breast cancer, patients received 6 months of talazoparib prior to surgery. Before surgery, 63% of patients had a residual cancer burden score of RCB0 or RCB1, which indicates the same positive expected outcome. In the 287 patient, Phase III, EMBRACA trial, patients treated with talazoparib had a PFS of 8.6 months compared to 5.6 months with standard chemotherapy and an ORR of 63% vs 27% in patients with advanced breast cancer who carry the BRCA1/2 mutations. PDUFA Date: December 15, 2018. No Priority Designations.
Ablynx’ Caplacizumab (Cablivi) is now approved in Europe. No PDUFA date, however, it has an Orphan Drug Priority Designation.

The FDA rejected Sunovion Pharmaceuticals’ dasotraline on 8/31/18 and requested additional data on efficacy and tolerability of dasotraline for the treatment of ADHD.

In a Phase III trial of 32 patients with heart failure and renal impairment, Orion Pharma’s levosimendan compared to dobutamine had similar increases in renal blood flow (22% and 26%, respectively), better glomerular filtration rate, and filtration fraction was not affected by levosimendan but decreased by 17% with dobutamine.

Biocryst announced topline results from the high dose cohort of the 95 patient, ZENITH-1 Phase II trial, where 64% of patients with Hereditary Angioedema, treated with BCX7353 had mild or no symptoms compared to 32% with placebo. BCX7353 reduced the need for rescue drugs by 32%. BCX7353 has a Fast Track Priority Designation but no PDUFA date.

China approved fruquintinib (Chi-Med, Lilly) in Sept 2018 for the treatment of metastatic colorectal cancer.

Bluebird Bio announced Interim results from the phase II/III Starbeam Study which demonstrated that 15 of 17 childhood cerebral adrenoleukodystrophy patients treated with Elivaldogene tavalentivec were free of major functional disabilities and no graft versus host disease almost 30 months after treatment. Twenty-nine patients had at least 4.2 months of follow-up and none had experienced grade 2 or higher acute graft-versus-host disease. The drug has a Breakthrough Therapy Priority Designation but no PDUFA date.

After failure in the CONCERTO and ARPEGGIO trials, Teva stopped development of laquinimod for multiple sclerosis. After the failure of a Phase II Huntington's disease trial, Teva ceased all development projects for laquinimod and returned all rights to Active Biotech.

Gilead and Galapagos announced that in the 12-week, 116 patient Phase II, TORTUGA trial, patients treated with filgotinib had a 1.5-point decrease in their Ankylosing Spondylitis Disease Activity Score (ASDAS) compared to a 0.6 decrease with placebo.

J&J has an ongoing Phase III trial testing esketamine for treatment-resistant depression and major depressive disorder with imminent risk of suicide. J&J filed an NDA for esketamine in September 2018 and plans to file an MAA before the end of 2018. 

Tezepelumab (AstraZeneca, Amgen) has been granted breakthrough status. No PDUFA date.


You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.

Erenumab Competitors Coming This Month

9/6/2018

 
September may be the month we see competition in the Anti-CGRP monoclonal antibody drug class. Two Investigational drugs in the Pharmaceutical Pipeline Tracker’s knowledgebase have PDUFA Dates during September. This will bring competition for erenunab (Aimovig), which was approved and launched in May. Both fremanezumab and galcanezumab are monoclonal antibodies that bind to calcitonin gene-related peptides.

Teva’s Fremanezumab with a PDUFA Date of September 16, 2018, due to its Fast Track status. It’s seeking approval for migraine prophylaxis. Teva's PDUFA date was delayed from June to September due to its API provider, Celltrion, receiving an FDA warning letter for manufacturing issues. Celltrion received a second warning letter from the FDA after a July 2018 inspection revealed problems with employee training quality control protocols. If the FDA delays the PDUFA date, it would potentially move fremanezumab to be the third approved product, instead of the second. In early September, Teva still felt the drug would be approved by the mid-September date.

Lilly’s Galcanezumab with expected approval by the end of September 30, has Fast Track status for cluster headache. It’s indicated for migraine prophylaxis. Lilly plans to offer an auto-injector for galcanezumab like erenumab. Fremanezumab is a more viscous solution and will initially be offered as a prefilled syringe. So, while the drug may be approved as a quarterly subcutaneous injection, it may require a visit to a physician's office for administration, while monthly doses of galcanezumab and erenumab can be administered by the patient. Teva is working on an auto-injection system for fremanezumab, but it is not known when it will be available.

One other Anti-CGRP monoclonal antibody investigational drug exists:  Alder Biopharmaceuticals’ eptinezumab, which is administered every three months by IV infusion.  Alder plans to file a BLA in the second half of 2018.  Alder has the ongoing PROMISE-2 study involving chronic migraine patients. Erenumab is available as an auto-injector for monthly sub-Q injection.
​
 ICER released a final review of CGRP inhibitors on July 3, 2018. ICER found insufficient evidence to recommend erenumab or fremanezumab over oral preventative drugs or botulism toxin for prevention of migraine in untreated patients. ICER did find evidence of a benefit for use of erenumab or fremanezumab in patients that had previously failed preventative therapy for chronic migraine. Data was supportive but inconclusive for prevention of episodic migraine. ICER found insufficient evidence for use of galcabezumab in either indication. ICER estimated that CGRP inhibitors would improve quality of life years (QALY) for episodic and chronic migraine patients. The estimated cost per QALY were lower for chronic migraine compared to episodic migraine. ICER felt that insurers would be justified in setting limits or restrictions on CGRP inhibitors due to insufficient long-term safety data and high cost. With an announced WAC price of $6,900/year for erenumab, ICER estimates an annual cost of $5,000/year after discounts. ICER stated that a price of $3,700 to $5,300 per year to be cost effective. Erenumab or fremanezumab were found to be cost effective in QALY gained in patients that had failed at least one preventative therapy.

Stay current with developments for drugs to reduce monthly migraines and all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.

Prescribe Right Pharmaceutical Pipeline Update for September 4

9/4/2018

 
Update on investigational drugs with recent PDUFA dates:
  • An MAA has been filed for larotrectinib (Loxo Oncology, Bayer). PDUFA date November 26, 2018 with no Priority Designations.

​In other developments: 

In a Phase III trial, Pfizer’s tafamidis reduced all-cause mortality (30% vs 43%) compared to placebo. Tafamidis also reduced cardiovascular-related hospitalizations, decline in 6-minute walk test and decline in the Kansas City Cardiomyopathy Questionnaire–Overall Summary that measures health status. ADR were similar between tafamadis and placebo in the trial. Approved in the EU, it does not have PDUFA Date but does have Orphan Drug, Breakthrough Therapy, and Fast Track Priority Designations.

Esperion announced that in a Phase III trial, in patients with atherosclerosis already receiving a high dose of statin, bempedoic acid and ezetimibe reduced LDL another 35% compared to 3% with placebo, 24% with ezetimibe monotherapy and 20% with bempedoic acid monotherapy. In a posthoc analysis of patients intolerant to statins, LDL was reduced 43% compared to 1% with placebo. In a 2,230 patient, Phase III trial, 13 patients (0.9%) that received bempedoic acid died compared to 2 (0.3%) in the placebo group. Esperion explains that 5 of the deaths were related to lung cancer. Esperion plans to price bempedoic acid < $4,000/ year. No morbidity or mortality results have been announced in bempedoic acid trials. No PDUFA date and no Priority Designations.

A small study found that naltrexone reduces the antidepressant effects of intravenous ketamine, suggesting that opioid system activation is required for the antidepressant effect with ketamine. Since esketamine is a derivative of ketamine, this new data will affect how the drug is viewed for its place in therapy.

AstraZeneca announced that in the 12-month Phase III trial, in patients with systemic lupus erythematous, anifrolumab did not reduce disease activity as measured by the SLE Responder Index, compared to placebo. AstraZeneca has another Phase III trial, ongoing evaluation of anifrolumab as a treatment for systemic lupus erythematous.
​
Biogen announced that interim data from a Phase Ib trial suggest that after 3 years with a titrated dose and 4 years with a fixed dose, aducanumab (for Alzheimer’s disease) continued to show a reduction in amyloid plaque and a continued benefit on the rate of decline of the Clinical Dementia Rating Sum of Boxes (CDR-SB) and the Mini-Mental State Examination (MMSE).

Ultragenyx may file an NDA for triheptanoin in late 2018 or 2019 with possible review in 2019. The drug has an Orphan Drug Priority Designation.

Karyopharm announced that in the 122 patient, Phase IIb, STORM trial, treatment with selinexor resulted in a 25% overall response rate in patients with highly resistant multiple myeloma.

In a Phase IIb trial, progressive multiple sclerosis patients treated with MediciNova’s ibudilast had 47% less brain atrophy (about 2.5 ml) compared to placebo. 

In a Phase II trial, treatment with capmatinib plus gefitinib demonstrated an overall response rate of 47% in patients with advanced c-MET-dysregulated non-small cell lung cancer (NSCLC). Among 161 patients in two early trials the most common adverse events with capmatinib were nausea, peripheral edema, decreased appetite, and rash. 
In a draft report, ICER estimated an annual cost of $24,700 and $46,488 for patisiran to be cost effective and $15,300 and $25,400 for inotersen to be cost effective.


You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.
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