It’s been another interesting week in biotech sector with a couple of announcements related to clinical trial results.
For Clearside BioMedical Inc. (CLSD), which announced positive top line results from its pivotal PEACHTREE phase III trial on Mar.5, 2018, it was clearly an up week, with the stock having gained 66%.
Another stock that caught investors’ eyes this past week was Immuron Limited (IMRN). The Company reported positive results from its phase II NASH clinical trial on Mar.8, 2018, sending the stock soaring as much as 97% that day.
However, not all was positive last week regarding clinical trial results announcement.
Vascular Biogenics Ltd (VBL) cratered 66% on Mar.8, 2018, following the failure of its phase III brain tumor trial, and Dermira Inc. (DERM) plunged nearly 61% on Mar.5, 2018 after two of its late-stage clinical trials of acne drug candidate Olumacostat glasaretil failed to meet the co-primary endpoints.
Let’s take a look at the pharma stocks and upcoming events to keep an ear out for in the coming week.
1. Regeneron Pharmaceuticals Inc. (REGN)
The much awaited results of ODYSSEY Outcomes trial, conducted by Sanofi (SNY) and Regeneron Pharmaceuticals Inc. (REGN) are out.
The ODYSSEY OUTCOMES study was designed to determine whether the addition of cholesterol-lowering drug Praluent to intensive statin therapy reduced major adverse cardiac events among patients who had previously experienced an acute coronary syndrome, such as a heart attack or unstable angina.
Praluent, developed by Sanofi (SNY) and Regeneron Pharmaceuticals Inc. (REGN), was approved by the FDA on July 24, 2015, for the treatment of patients with heterozygous familial hypercholesterolemia or atherosclerotic heart disease who require additional lowering of LDL-cholesterol.
The ODYSSEY OUTCOMES trial involved 18,924 patients who had an acute coronary syndrome (ACS) within the previous 12 months, residual LDL cholesterol levels of 70 mg/dL or higher, non-high-density lipoprotein cholesterol of 100 mg/dL or higher, or apolipoprotein B of 80 mg/dL or more
after two to 16 weeks of intensive or maximally tolerated statin therapy. The median duration of follow-up in this trial was 2.8 years.
According to the study results, reported on mar.10, 2018, Praluent reduced the overall risk of major adverse cardiac events (MACE) by 15%, a significant difference compared to placebo. The MACE, which is the primary efficacy endpoint of the study, included hospitalization for unstable angina, heart attack, stroke or cardiovascular death.
Although there was no significant difference between Praluent group and placebo group for coronary heart disease death and cardiovascular death, the rate of all-cause death was significantly lower by 15 percent with Praluent compared to placebo. The LDL-C levels after a median follow-up of 2.8 years were 53.3 mg/dL in the Praluent arm compared with 101.4 mg/dL in the placebo group.
Praluent had a more pronounced effect in patients with baseline LDL-C levels at or above 100 mg/dL. In this patient population, Praluent reduced MACE by 24%, coronary heart disease death was reduced by 28%, cardiovascular death by 31% and all-cause death by 29% compared with placebo.
The U.S. Wholesale Acquisition Cost (WAC) price of Praluent, excluding discounts, is $14,600 per year. The global net sales of Praluent were $194 million in the full year 2017, up from $116 million in 2016.
Based on the ODYSSEY OUTCOMES study results, the Institute for Clinical and Economic Review has calculated the value-based price benchmarks for Praluent to be $2,300-$3,400 per year if used to treat all patients who meet trial eligibility criteria, and $4,500-$8,000 per year if used to treat higher-risk patients with LDL cholesterol at 100 mg/dL or more despite intensive statin therapy. Value-based pricing is the cost of the drug that would align with its benefits to patients.
Sanofi and Regeneron will be meeting with U.S. payers to discuss potential net pricing adjustments for those that agree to provide straightforward access for high-risk patients.
REGN closed Friday’s (Mar.9, 2018) trading at $341.70, up 0.27%.
2. Savara Inc. (SVRA)
Savara is a clinical-stage company developing drugs for the treatment of rare respiratory diseases.
The Company has 2 drug candidates in late-stage development and 1 under phase II development.
Molgradex for Pulmonary Alveolar Proteinosis (PAP) and AeroVanc for MRSA in Cystic Fibrosis (CF) are the Company’s late stage assets. Aironite for Diastolic Heart Failure (HFpEF) is a phase II drug candidate.
The top line results from the phase II study of Aironite in Diastolic Heart Failure, dubbed INDIE, are expected to be presented at the American College of Cardiology 67th Annual Scientific Sessions & Expo on March 11th, 2018 at 11:00am ET.
SVRA closed Friday’s trading at $10.98, down 2.49%.
3. Aeglea Biotherapeutics Inc. ( AGLE )
Aeglea is a clinical-stage biotechnology company focused on developing novel human enzyme therapeutics for patients with rare genetic diseases like Arginase 1 Deficiency, and cancer.
Arginase deficiency, a rare inherited disorder, is characterized by gradual accumulation of ammonia in the blood.
The Company’s lead product candidate is AEB1102, which is under a phase 1/2 clinical trial in patients with Arginase 1 Deficiency.
The trial is designed to enroll approximately 10 patients, adult and pediatric, with Arginase 1 Deficiency in the United States, Canada, and Europe. The trial investigates both single ascending doses (Part 1) and repeated dosing (Part 2).
Early results from the phase 1/2 Adult Repeat Dose part of the trial are expected to be reported at the 2018 Annual Meeting of The Society for Inherited Metabolic Disorders on March 12, 2018, 7:30 p.m. to 10:00 p.m.
4. TESARO Inc. (TSRO)
TESARO is an oncology-focused biopharmaceutical company. The lead drug candidate is Niraparib, an orally active and potent PARP inhibitor being developed for the treatment of ovarian and breast cancer.
The Company’s CEO Lonnie Moulder will be participating in an analyst-led moderated Q&A session in the Barclays Global Healthcare Conference on Wednesday, March 14, 2018.
A clinical trial catalyst related to Niraparib is due in March.
Last month, while announcing its operating results, the Company had revealed that top-line results from a phase II study of Niraparib in patients with Ovarian Cancer who have received three or four previous chemotherapy regimens, dubbed QUADRA, will be available in the first quarter. (i.e., period ending March 2018).
TSRO closed Friday’s trading at $68.12, down 1.90%.
