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Pipeline News and Updates
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FDA Approves Siponimod – ICER weighs in and April PDUFA Dates

3/28/2019

 
The FDA approved siponimod (Mayzent, Novartis) on 3/26/2019 to treat adults with relapsing multiple sclerosis. In a draft review ICER felt that compared to best supportive care siponimod reduced the risk of disability progression and decreased inflammation. ICER analysis found that siponimod lowered the risk of relapse by 46% and had an annualized relapse rate of 0.07/year compared to 0.16/year with placebo. Due to the lack of comparative trials with other disease modifying therapies and the difference in patient populations and outcome assessments with current trials, ICER analysts did not feel enough data existed to compare siponimod to those therapies. ICER estimated a quality-adjusted life year (QALY) of $826,000 for use of siponimod over best supportive care in the treatment of secondary progressive multiple sclerosis, assuming the same price as ocrelizumab. The cost per QALY dropped to $396,000 in patients with relapses within two years of the initiation of treatment. ICER reported an annual acquisition price for ocrelizumab of $64,308 after discounts. Novartis has announced an annual WAC of $88,000 for  siponimod. There have been no estimates or reports of potential discounts for siponimod, during the week the drug was approved. ICER will accept comments on the draft report until April 10 and then release a final report on May 2 with a discussion of the report set for May 23.

March was a busy review month for the FDA, but in April, there is only one PDUFA deadline. Risankizumab (Skyrizi, Boehringer Ingelheim and AbbVie) is an interleukin-23 inhibitor being developed for the treatment of Psoriasis and Crohn’s disease. The drug is an Orphan Drug and has a PDUFA date of 4/25/2019. Risankizumab will compete with other IL-23-targeting drugs (ustekinumab, tildrakizumab, guselkumab), IL-17 blockers (secukinumab) and TNF-blockers (adalimumab, etanercept). The drug is given as a subcutaneous injection every three months. The NDA is based on four Phase III trials. In two trials 75% of risankizumab patients achieved Psoriasis Area and Severity Index (PASI 90) compared to 42 to 48% with ustekinumab and 2 to 5% with placebo. In a third 72% of risankizumab patients achieved PASI 90 compared to 47% with adalimumab. The fourth trial was a withdrawal and re-treatment trial where 73% of risankizumab patients achieved PASI 90 compared to 2% with placebo. 

Two Approvals, One new QIDP, One rejection and a BLA withdrawal

3/26/2019

 
FDA Actions

The FDA approved solriamfetol (Sunosi, Jazz Pharmaceuticals) on 3/20/2019 to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea.

The FDA approved brexanolone (Zulresso, Sage Therapeutics)on 3/19/2019 for the treatment of postpartum depression. 

The FDA granted Qualified Infectious Disease Product (QIDP)to the oral and IV formulations of sulopenem in March 2019.  

The FDA rejected sotagliflozin on 3/22/2019.Lexicon and Sanofi are working with the FDA to determine future development of the drug. 

Spectrum withdrew the BLA for eflapegrastimto provide more information to the FDA on the chemistry, manufacturing and controls process.

Two
Trials Published

In a draft cost-effectiveness analysis, ICER estimated a quality-adjusted life year of $826,000 for use of siponimod in the treatment of secondary progressive multiple sclerosis, assuming the same cost for siponimod as ocrelizumab.

In a 48-hour, 389 patient, Phase III trial, 50% of Trevena’s oliceridine 0.1 mg patients, 62% of 0.35 mg patients and 65.8% of 0.5 mg patients reached the combination endpoint of a 30% or > improvement in pain (SPID-48), received no rescue drugs, completed the study period and did not reach dosing limits compared to with 15.2% that received placebo in patients with moderate-to-severe pain following bunionectomy. The 0.35 mg and 0.5 mg doses were non-inferior to morphine in reaching the primary endpoint. In a 389 patient, Phase III trial, GI ADR increased in a dose-dependent manner in oliceridine regimens; 40.8% with 0.1 mg, 59.5% with 0.35 mg and 70.9% with 0.5 mg compared to 24.1% with placebo and 72.4% with morphine.

Five Announced Investigational Drug Developments
  1. Demira announced that in a 16-week, 280 patient, Phase Ib trial, lebrikizumab improved the EASI score by 62.3% in patients receiving 125 mg every four weeks, 69.2% with 250 mg every four weeks and 72.1% for patients receiving 250 mg every two weeks compared to a 41.1% improvement with placebo in patients with moderate-to-severe atopic dermatitis. Dermira plans to initiate Phase III trials at the end of 2019.
  2. Urovant announced that in the 12-week, 1,518 patient, Phase III, EMPOWUR trial, treatment with vibegron reduced micturitions per 24 hours and urge urinary incontinence episodes per 24 hours compared to placebo in patients with overactive bladder. Vibegron was no different than tolterodine in the trial. Compared to placebo, vibegron reduced both primary endpoints as early as two weeks. Urovant enrolled 507 patients from the EMPOWUR trial in a 40-week RCT extension trial to evaluate the safety of longer-term treatment of vibegron. The FDA accepted the BLA for AR101 and set a PDUFA date for late January 2020.
  3. GenNeuro announced that in the ANGEL-MS trial, a 96-week, 154 patient, extension of the Phase IIb CHANGE-MS trial, where the highest dose of temelimab (18mg/kg) had 2.4% of patients develop worsening of 20% or more in the Timed 25-Foot Walk Test compared to 10.2% with placebo in patients with multiple sclerosis. There was no statistical difference in worsening in neurological disability between temelimab and placebo.
  4. Biogen and Eisai announcedthat an interim analysis by an independent committee of the Phase III aducanumab trials ENGAGE and EMERGE involving patients with mild cognitive impairment due to Alzheimer's disease and mild Alzheimer's disease dementia were unlikely to meet their primary endpoints resulting in a discontinuation of both studies. Biogen also discontinued a Phase II safety trial and long-term extension of a Phase Ib trial. Biogen is considering whether to initiate a Phase III secondary prevention trial.
  5. Conatus announced that 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.

Potential Blockbuster Approvals in 2019

3/21/2019

 
Four forecast as having $1 billion in sales by 2023
 
ClarivateAnalytics™ in its Cortellis™ Drugs to Watch 2019 report published a forecast of four drugs that may be approved in the U.S. in 2019, which they forecast as having $1 billion in sales by 2023. All four drugs have been designated Orphan Drugs. In addition, there are three drugs with breakthrough status and two have Fast Track status. The four drugs are being developed to treat a variety of diseases, but surprisingly none are oncology drugs.
  1. AbbVie’s updacitinib, a JAK1 inhibitor being developed for rheumatoid arthritis, atopic dermatitis, Crohn’s disease, and juvenile idiopathic arthritis. Updacitinib will compete with the already approved JAK inhibitors tofacitinib (Xeljanz, Pfizer) and baricitinib (Olumiant, Eli Lilly) in addition to tumor necrosis factor (TNF) blockers (etanercept, golimumab, infliximab) and non-TNF biologicals (tocilizumab, abatacept, rituximab). Updacitinib has a PDUFA date of 8/19/2019 and has been designated a Breakthrough Therapy and an Orphan Drug. 
  2. Onasemnogene abeparvovec (Novartis/AveXis’ Zolgensma) is a single injection gene therapy to replace survival motor neuron (SMN) protein, which is the genetic defect causing spinal muscular atrophy. Onasemnogene abeparvovec will compete with nusinersen (Spinraza, Biogen) an antisense oligonucleotide that increases functional SMN protein production by modifying translation from the SMN2 gene to SMN protein. Onasemnogene abeparvovec has a PDUFA date of 5/3/2019. In addition to Orphan Drug status, onasemnogene abeparvovec has also been designated a Breakthrough Therapy and has Fast Track status. In Europe the drug has Orphan Drug and PRIME status. 
  3. Risankizumab (Skyrizi, Boehringer Ingelheim and AbbVie) is an interleukin-23 inhibitor being developed for the treatment of Psoriasis and Crohn’s disease. The drug is an Orphan Drug and has a PDUFA date of 4/25/2019. Risankizumab will compete with other IL-23-targeting drugs (ustekinumab, tildrakizumab, guselkumab, IL-17 blockers (secukinumab) and TNF-blockers.
  4. Aimmune’s AR101 is an oral desensitization immunotherapy being developed for peanut allergy. AR101 has been granted Breakthrough Therapy and Fast Track status by the FDA. Because the FDA feels that AR101 is not a drug, but an allergenic extract, the product would not be reviewed under the PDUFA process which would provide a priority review in 6 months or a regular review in 10 months. Therefore, the FDA has said a 12-month review would apply, which would place the review in January 2020. Immune is discussing the prospect of AR101 being declared a drug and Clarivate has forecast a decision in November 2019 (10-month PDUFA review timeline). If AR101 is approved at the end of 2019 or early 2020, it will enter the market with no commercial competition. The closest challenger is the transdermal Visken Peanut patch from DBV Technologies. DBV initially submitted a BLA in 2018 but pulled the BLA to provide more data on manufacturing and quality control. ​
About Clarivate: Clarivate Analytics™ is the global leader in providing trusted insights and analytics to accelerate the pace of innovation. 
 
About Cortellis: Cortellis™, a suite of life science solutions from Clarivate Analytics™, curates the broadest and deepest sources of intelligence to enable precise, actionable answers to specific questions across the R&D lifecycle – from discovery and clinical development through regulatory submission and commercialization.

FDA Approves One, Delays Another, Grants New Orphan Drug

3/19/2019

 
Regulatory Actions

The FDA approved netarsudil and latanoprost ophthalmic solution (Rocklatan, Aerie Pharmaceuticals) on 3/13/2019 to decrease elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension.
​
 In March 2019, the FDA delayed the PDUFA date for Karyopharm Therapeutics’ selinexor by three months (from April 6, 2019 to July 6, 2019).

The FDA accepted the NDA for ubrogepant on 3/11/2019. Allergan expects a 4Q19 PDUFA date of 12/27/19
 
The FDA accepted the NDA for lemborexant on 3/11/2019. Allergan expects a 4Q19 PDUFA date of 12/27/19
 
The FDA granted Orphan Drug status to Armetheon’s tecarfarin for the prevention of systemic thromboembolism of cardiac origin in patients with End Stage Renal Disease (ESRD) and Atrial Fibrillation (AFib).

Celgene filed an MAA filed for ozanimod in March 2019.

New Published Research 

In a Phase III, open label, follow-up safety study, 49% completed the Alkermes samidorphan/buprenorphine study and had a mean MADRS score decrease from 22.9 at baseline to 9.8 with a remission rate of 52.5%. There were 11% of patients that discontinued due to an adverse event. The most common ADR were nausea, headache, constipation, dizziness, and somnolence. There was no evidence of an increased risk for suicidal ideation or behavior or withdrawal. There was a 1.2% incidence of euphoria-related events. No meaningful changes in laboratory values, metabolic parameters or bodyweight were found.


In a 12-week, 2,230 patient, Phase III trial, Esperion Therapeutics’ bempedoic acid lowered LDL 18% compared to a 2% increase with placebo in patients with atherosclerotic cardiovascular disease, heterozygous familial hypercholesterolemia, or both not adequately controlled on maximally tolerated statins. The frequency of ADR at 52 weeks was similar between bempedoic acid and placebo for all events (78.5% and 78.7%) or serious events (14.5% and 14.0%). Bempedoic acid patients experienced more gout (1.2% vs 0.3%). This was a non-significant increased incidence of heart failure hospitalizations and increased mortality, but the incidence was 0.5% or less.

New Announced Research 

TG Therapeutics announced interim data from 69 patients in the open label, Phase II, UNITY-NHL trial. The UNITY-NHL trial is evaluating umbralisib as a treatment for multiple kinds of Non-Hodgkin's lymphoma. A cohort of 69 patients with Marginal Zone Lymphoma had an overall response rate of 40-50%. TG Therapeutics expects to file an NDA for umbralisib in late 2019.

A 35 patient, Phase II resistant renal cell carcinoma trial was stopped early due to inadequate response with Amgen’s trebananib and an anti-VEGF agent.

Akebia announced interim data at 24 weeks from a 52-week, 304 Japanese patient, Phase III, open label trial, where vadadustat was non-inferior to darbepoetin alfa in mean Hb (11.66 g/dL vs 11.93 g/dL) in patients with non-dialysis dependent CKD. Akebia also announced interim data at 24 weeks from a 52-week, 323 Japanese patient, Phase III, trial, in which vadadustat was non-inferior to darbepoetin alfa in mean Hb (10.61 g/dL vs 10 g/.65dL) in patients with hemodialysis-dependent CKD. Akebia announced that in a 24-week, 42 Japanese patient, Phase III, open label trial, treatment with vadadustat resulted in a mean Hb of 11.35 g/dL in patients with peritoneal dialysis-dependent CKD.

Arena announced that in the 12-week, 156 patient, Phase II, OASIS trial, treatment with etrasimod improved the Mayo Clinic Score (a composite of rectal bleeding, stool frequency, and endoscopic findings) 1 point more than placebo in patients with moderate to severe ulcerative colitis. Etrasimod 2 mg, but not 1 mg achieved endoscopic improvement (16.3% vs. 43.2%), histological improvement (10.2% vs. 31.7%), and histological remission (6.1% vs. 19.5%) compared to placebo. Arena also announced that in a 34-week, 118 patient open label extension of the OASIS trial 84 patients received 2 mg of etrasimod during the extension study with 79% achieving a clinical response, 39% achieving clinical remission and 51% had endoscopic at 46 weeks. Among 22 patients who also received 2 mg of etrasimod during the OASIS trial, 82% experience clinical response, 50% were in clinical remission and 55% had endoscopic improvement. In the patients that achieved a clinical response or clinical remission in OASIS, 93% experienced sustained response and 75% experienced sustained remission at both 12 and 46 weeks.

Lilly announced that in a 12-week, 249 patient, Phase II trial, treatment with mirikizumab 200 mg resulted in greater remissions than placebo in patients with moderate-to-severe ulcerative colitis who had failed at least one conventional drug. Treatment with 50 mg or 600 mg was no different than placebo. Patients that achieved a clinical response at week 12 (93 patients) were randomly reassigned to mirikizumab 200 mg subcutaneously every 4 weeks or every 12 weeks. At week 52, 46.8% of the q4w patients and 37% of the q12w patients were in clinical remission, while 57.4% of q4w patients and 47.8% of the q12w patients had endoscopic scores of zero or one.

Bayer announced that in the 52-week, 330 patient, Phase III, CHICO trial, treatment with 60 days of nifurtimox resulted in a superior serological response compared with historical placebo control in pediatric patients with acute or chronic Chagas disease.

Investigational Update for the Week Ending March 8

3/12/2019

 
Three FDA Actions Last Week, Celgene’s Plans and Fourteen Research Updates

The FDA’s Vaccines and related Biological Products Advisory Committee voted to recommend approval for Sanofi’s Dengue vaccine for patients 9 to 17 years, but not in adult patients.

The FDA has granted fedratinib a priority review, with a PDUFA date of 9/3/2019. There are now twenty-five investigational drugs with PDUFA Dates between March 14, 2019 and December 25, 2019

The FDA removed the Breakthrough Therapy designation for oliceridine because current data did not support the designation. The FDA does feel the current safety data will support a refiled NDA after Trevena completes a Phase I safety study examining QT-interval prolongation.
​
Celgene plans to submit an NDA for ozanimod in March 2019 and a BLA for luspatercept in April.
  1. Vertex announced interim 4-week data from a Phase III trial, where VX-445 in combination with tezacaftor/ivacaftor demonstrated a mean improvement in ppFEV1 of 13.8% compared to a 0.2% loss with placebo in cystic fibrosis patients with one F508del mutation and one minimal function mutation. Vertex announced interim 4-week data from a 24-week Phase III trial, in which the addition of VX-445 to a combination of tezacaftor/ivacaftor demonstrated a mean improvement in ppFEV1 of 10.4% compared to a 0.4% improvement with placebo in cystic fibrosis patients with two F508del mutations already receiving tezacaftor and ivacaftor. Vertex plans to submit an NDA for either VX-445 or VX-659 in 3Q19 and a MAA in 4Q19.
  2. Alnylam announced that in a Phase III trial, treatment with givosiran reduced the annualized rate of composite porphyria attacks compared to placebo in patients with acute hepatic porphyria. In the trial, 21% of givosiran patients experienced a serious adverse event compared to 9% with placebo. Alnylam is planning to begin a rolling NDA for givosiran in mid-2019, after full results from Phase III trials are available.
  3. Menlo announced results from a Phase II trial, in which 33.3% of plaque psoriasis patients treated with serlopitant had at least a 4-point improvement in their Worst Itch Numeric Rating Scale score compared to 21.1% of patients that received placebo.
  4. Shield announced that in a Phase IIIb trial, oral ferric maltol increased hemoglobin within 9% of the response rate seen with IV ferric carboxymaltose in patients with Iron Deficiency Anemia with inactive Inflammatory Bowel Disease in whom other oral iron therapies had failed.
  5. Apellis has two ongoing Phase III trials evaluating APL-2 in the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Apellis halted enrollment in two trials in October 2018 due to the occurrence noninfectious inflammation. An investigation by Apellis determined the cause to be an impurity or contaminant from a change in the manufacturing process after Phase II trials. A correction has been made to the manufacturing process and Apellis dosed 10 patients in a Phase Ib geographic atrophy trial without incident. 
  6. Ascendis announced that in a Phase III, open label trial, TransCon Growth Hormone was non-inferior to daily growth hormone (11.2 cm/year vs 10.3 cm/year) in children with pediatric growth hormone deficiency.
  7. TG Therapeutics announced that in a phase II trial, treatment with ublituximab resulted in median B cell depletion of > 99%, a decrease in mean T2 lesion volume of 10%, and an annualized relapse rate of 0.07 with 93% of patients with relapsing multiple sclerosis being relapse free. TG Therapeutics has two Phase III trials comparing ublituximab to teriflunomide in the treatment of relapsing MS. Results are expected in mid-2020.
  8. In a Phase III trial, the triglyceride levels of patients treated with Kowa Pharmaceuticals’ pemafibrate were reduced by 45% compared to placebo in patients with type 2 diabetes comorbid with hypertriglyceridemia. In a 28-week open label extension of the trial, pemafibrate maintained triglyceride reductions at 52 weeks (42.3 to 48.2%).
  9. MyoKardia announced that interim data from 10 patients enrolled in a Phase II extension a trial, suggested that 8 patients treated with mavacamten had improved their NYHA Class and 7 were asymptomatic.
  10. Allergan announced that rapastinel did not improve depression compared to placebo in three Phase III trials involving 1,510 patients with major depression disorder (MDD) who had a partial response to their current antidepressant therapy. Allergan is evaluating rapastinel monotherapy compared to placebo in the treatment of major depression disorder in a Phase III study and in a Phase II study of patients with MDD at imminent risk of suicide. 
  11. Syndax announced that in a Phase Ib/II trial, entinostat in combination with atezolizumab did not improve progression free survival (PFS) in patients with triple negative breast cancer. Syndax announced that in a Phase Ib/II trial, entinostat in combination with avelumab did not improve PFS in patients with ovarian cancer. Syndax is evaluating entinostat in combination with exemestane in a Phase III trial of patients with advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer.
  12. Eisai announced that in a Phase III trial, lemborexant reduced sleep latency compared to extended-release zolpidem or placebo. In a sub-analysis of 453 patients over the age of 65 from the trial sleep latency was reduced compared to extended-release zolpidem or placebo.
  13. ViiV announced the results of two Phase III trials. In the 48-week, 616 patient, Phase III Atlas Trial, 92.5% of patients that received a monthly IM injection of cabotegravir and rilpivirine had an HIV-1 RNA < 50 copies/mL compared to 95.5% of patients that received a daily thee drug oral regimen of 2 NRTIs plus a third drug. In the 48-week, 566 patient, Phase III FLAIR trial, 93.6% of patients that received a monthly IM injection of cabotegravir and rilpivirine had an HIV-1 RNA < 50 copies/mL compared to 93.3% of patients that received daily abacavir/dolutegravir/lamivudine.
  14. In an 8-week, 152 IBS-C patient Phase III trial, the mean change in serum phosphate from pooled data from 3 doses of Ardelyx’s tenapanor over the four weeks after treatment was completed was a 0.02 mg/dl increase in the pooled tenapanor group compared to a mean increase of 0.85 mg/dl in the placebo group in dialysis patients with hyperphosphatemia.​

Drugs with March PDUFA Dates

3/8/2019

 
The FDA has several drugs with PDUFA Dates in March. One was approved earlier this week and six more have PDUFA Dates coming up in the coming days.

On March 5, 2019 the FDA approved esketamine nasal spray, in conjunction with an oral antidepressant, for the treatment of resistant depression. The labeling for esketamine nasal spray contains a Boxed Warning describing a risk for sedation and difficulty with attention, judgment and thinking (dissociation), abuse and misuse, and suicidal thoughts and behaviors. The drug is only available through a restricted distribution system, under a Risk Evaluation and Mitigation Strategy (REMS). Esketamine must be administered in a certified medical office where the health care provider can monitor the patient for a minimum of 2 hours and determine when the patient can leave. The patient also needs transportation to and from the visit for a dose because they are unable to drive until the day after a dose. Since the patient cannot take the spray home, the treatment sites will need to determine where patients will wait during the monitoring period and coordinate patient visit to avoid a slowdown in office workflow. WAC for esketamine nasal spray is $590 to $885 per treatment session. The drug is dosed twice a week for the initial 4 weeks, so the monthly cost will be $4,720 to $6,785. Esketamine nasal spray is dosed once weekly during the second month ($2,360 to $3,540). In the third month the patient may continue weekly dosing or have their dosing further reduced to every other week, so the WAC price for maintenance dosing is $1,180 to $3,540.

​Drug with upcoming PDUFA Dates in March

  • Aerie Pharmaceuticals’ combination ophthalmic netarsudil mesylate and latanoprost has a PDUFA date of March 14 for the treatment of glaucoma.
  • Sage Therapeutics’ has a March 19, 2019 PDUFA for its GABA modulator brexanolone being developed for the treatment of postpartum depression. Brexanolone has orphan drug and breakthrough therapy priority designations.
  • Jazz Pharmaceuticals has a March 20 PDUFA date for Solriamfetols it’s selective norepineprhine and ropamine Reuptake Inhibitor being developed for the treatment of excessive sleepiness in narcolepsy and obstructive sleep apnea. The FDA had delayed the PDUFA date for solriamfetol by three months. Solriamfetol has an orphan drug priority designation.
  • Sanofi has a March 22 PDUFA date for sotagliflozin, a SGLT-1 and SGLT-2 being developed for the treatment of Type 1 and Type 2 diabetes.
  • AMAG has a March 23 PDUFA date for its melanocortin 4 receptor agonist, bremelanotide for the treatment of hypoactive sexual desire disorder.
  • Novartis has a March 31, 2019 PDUFA date for a decision on siponimod, its sphingosine-1-phosphate receptor modulator being developed for the treatment of relapsing/remitting multiple sclerosis and secondary progressive multiple sclerosis.

Four FDA actions plus five oncology developmental updates from the last week in February

3/5/2019

 
Seven non-oncology development updates included below.

The FDA delayed a decision on NKTR-181 by three months to allow additional time to review preclinical data from two additional studies and set a new PDUFA date of 8/29/2019.

On 2/28/2019, the FDA Oncologic Drugs Advisory Committee recommend a delay in approval of selinexor-dexamethasone to treat triple class-refractory myeloma until after the results of the Phase III BOSTON trial are available in the first half of 2020.

The FDA accepted an NDA for diroximel fumarate on 2/25/2019 suggesting a PDUFA date of 12/25/2019.

The FDA designated AT-GAA as a Breakthrough Therapy in February 2019.

Twelve investigational drug developmental updates: 
  1. Actinium announced interim data from the 47 patients in the 150 patient, Phase III, SIERRA trial, where 92% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant prior to day 100 compared to standard of care chemotherapy where 79% of patients failed to achieve a complete response in patients with active, relapsed or refractory acute myeloid leukemia.
  2. Bayer has submitted an NDA for darolutamide for the treatment of nonmetastatic castration-resistant prostate cancer.
  3. In a 59 patient, Phase II, open label trial, treatment with apatinib resulted in a progression-free survival of 3 months and overall survival of 7.93 months in patients with stage IV sarcoma who had failed chemotherapy. 
  4. In a 138 patient, Phase II, open label trial, the addition of PEGPH20 to mFOLFIRINOX increased toxicity and decreased treatment duration compared to mFOLFIRINOX alone in patients with metastatic pancreatic cancer. 
  5. ImmunoGen announced that in a 366 patient, Phase III trial, mirvetuximab soravtansine did not improve progression-free survival compared to standard chemotherapy in patients with platinum-resistant ovarian cancer that expressed medium to high levels of folate receptor alpha and had received up to three prior lines of therapy.
  6. Santhera announced that in the 6-year, open label, SYROS trial, a long-term follow-up in 18 of 64 patients that completed the Phase III DELOS trial, where switching to or maintaining long-term treatment with idebenone reduced the rate of decline in forced vital capacity percent of predicted (FVC%p) by 50% in patients with Duchenne muscular dystrophy.
  7. In a 12-week, 1,208 patient, Phase III, PERFECT 1 trial, more patients treated with trifarotene cream achieved an Investigator Global Assessment of IGA, clear/almost clear and ≥2 grade improvement than placebo (29.4% vs 19.5%), a reduction in facial lesion counts with inflammatory lesions (-19 vs -15.4) and non-inflammatory lesions ( -25 vs -17.9) in patients 9 years or older with moderate facial acne and moderate truncal acne. In a 12-week, 1,212 patient, Phase III, PERFECT 2 trial, more patients treated with trifarotene cream achieved an Investigator Global Assessment of IGA, clear/almost clear and ≥2 grade improvement than placebo (42.3% vs 25.7%), a reduction in facial lesion counts with inflammatory lesions (-24.2 vs -18.7) and non-inflammatory lesions ( -30.1 vs -21.6) in patients 9 years or older with moderate facial acne and moderate truncal acne. Mayne Pharma is evaluating trifarotene in the treatment of Lamellar Ichthyosis in a Phase II trial. Galderma has submitted an NDA for trifarotene for the treatment of facial and truncal acne.
  8. Redhill plans to file an NDA for RHB-105 in the first half of 2019 with a potential launch by the end of the year if the drug is approved. 
  9. Horizon announced that in the 24-week, 83 patient, Phase III, Optic trial, 82.9% of patients treated with teprotumumab had a 2 mm or more reduction in proptosis compared to 9.5% with placebo in patients with active thyroid eye disease. Horizon Pharma plans to file a BLA for teprotumumab in mid-2009. 
  10. CHMP recommended approval of approval of sotagliflozin and risankizumab and conditional marketing authorization for volanesorsen.
  11. In a 12-week, 43 patient, Phase II, open label trial, treatment with NGM282 reduced the NAFLD activity score by 1.9 points with 1 mg and 2.3 points with 3 mg in patients with biopsy‐confirmed nonalcoholic steatohepatitis. The fibrosis score decreased by 0.1 point with 1 mg and 0.5 points with 3 mg.
  12. UCB announced that in a 60-week, long-term follow-up to the Phase IIb BE ABLE trial, 80 to 100% of bimekizumab responders maintained PASI90.
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