Aerie Pharma Receives Positive Feedback from FDA; Cleveland BioLabs Announces Submission of Pre-EUA Dossier for Entolimod to FDA Print
By Marilyn Mullen   
Monday, 15 June 2015 19:11
Below is a look at some of the headlines for companies that made news in the healthcare sector on June 15, 2015.
Aerie Pharmaceuticals, Inc. (NASDAQ: AERI), reported the U.S. FDA has agreed in written and verbal communications that Aerie may change the primary endpoint range of its second Phase 3 registration trial of RhopressaTM, named Rocket 2. With this agreement, the Company is changing the primary endpoint range to include patients with baseline intraocular pressures (IOPs) ranging from above 20 mmHg (millimeters of mercury) to below 25 mmHg. The former range for the primary endpoint of above 20 mmHg to below 27 mmHg will now represent a secondary endpoint range for Rocket 2.
   
Highlights -- The Rocket 2 primary endpoint range is now changed to the same range where the Phase 3 registration trial results of Rocket 1 demonstrated success, ranging from above 20 mmHg to below 25 mmHg. In the Rocket 1 trial, in this range, RhopressaTM demonstrated non-inferiority to timolol, and numerical superiority over timolol at the majority of time points. According to the Baltimore Eye Survey, nearly 80 percent of newly diagnosed glaucoma patients have unmedicated baseline IOPs below 26 mmHg.
  
The FDA also agreed that the Company may use a hierarchically-based statistical approach in determining whether this three-arm trial is adequately powered at the revised primary endpoint range. Using this methodology, the Company believes that the new primary endpoint range is adequately powered, and there is no need to recruit additional patients into Rocket 2. Three-month efficacy results for Rocket 2 are expected by the end of the third quarter of 2015.
    
An additional RhopressaTM Phase 3 registration trial, named Rocket 4, is expected to commence in the third quarter of 2015, along with the first RoclatanTM Phase 3 registration trial, named Mercury 1.
   
"We are extremely pleased with the outcome of our communications with the FDA. If Rocket 2 results resemble those of Rocket 1, we believe we may have a much greater opportunity for success in meeting the clinical endpoint of non-inferiority to timolol. We are also very appreciative of the thoughtful guidance provided by the FDA, and believe their feedback will prove very useful as our programs progress. Looking ahead, we expect to commence our next Phase 3 registration trial for RhopressaTM, named Rocket 4, in the third quarter of 2015. Rocket 4 is expected to be established with a primary endpoint range of above 20 mmHg to below 25 mmHg," said Vicente Anido, Jr., Ph.D., Chairman and Chief Executive Officer.
   
Dr. Anido continued, "We are also preparing to commence our first Phase 3 registration trial for RoclatanTM, named Mercury 1, in the third quarter of 2015. We believe the apparent synergies we observed in Rocket 1 for RhopressaTM trial patients previously on prostaglandin analogues such as latanoprost may explain why the RoclatanTM Phase 2b results were so impressive. If successful in the Phase 3 registration trials, we believe this once-daily quadruple-action product candidate has the potential to be the most efficacious eye drop in the market for patients with glaucoma and ocular hypertension."
Triple-Action Rhopressa™  --  Rhopressa™ is a novel triple-action eye drop that we believe, if approved, would become the only once-daily product available that specifically targets the trabecular meshwork (TM), the eye's primary fluid drain and the diseased tissue responsible for elevated IOP in glaucoma. Preclinical results have demonstrated that RhopressaTM also lowers episcleral venous pressure (EVP), which contributes approximately half of IOP in healthy subjects. Further, RhopressaTM provides an additional mechanism that reduces fluid production in the eye and therefore lowers IOP. Biochemically, RhopressaTM is known to inhibit both Rho Kinase (ROCK) and norepinephrine transporter (NET).
   
There were originally three Phase 3 registration trials for RhopressaTM. "Rocket 1" was a 90-day efficacy trial, the results of which were initially reported on April 23, 2015, "Rocket 2" is a 12-month safety trial with a 90-day interim efficacy readout, and "Rocket 3" is a safety-only study being conducted in Canada. In Rocket 1, for the primary endpoint range of above 20 mmHg to below 27 mmHg, RhopressaTM did not demonstrate non-inferiority to timolol. However, RhopressaTM did demonstrate non-inferiority to timolol at all ranges below 26 mmHg. As a result, the Company plans to commence in the third quarter of 2015 an additional Phase 3 registration trial, named "Rocket 4". Based on the current clinical trial status, the Company may submit a New Drug Application filing in the second half of 2016.
   
Quadruple-Action Roclatan™  --  Roclatan™ is a once-daily eye drop that combines our triple-action Rhopressa™ with latanoprost, a prostaglandin analogue that is the most widely prescribed glaucoma drug. If approved, we believe that Roclatan™ would be the first glaucoma product to lower IOP through all known mechanisms: (i) increasing fluid outflow through the TM, the eye's primary drain, (ii) increasing fluid outflow through the uveoscleral pathway, the eye's secondary drain, (iii) reducing fluid production in the eye, and (iv) reducing EVP.
  
A successful 28-day Phase 2b clinical trial for Roclatan™ was completed in June 2014. RoclatanTM achieved its primary efficacy endpoint on day 29 and demonstrated statistical superiority over the product's individual components at all time points. We believe that Roclatan™, if approved, would be the only glaucoma product that covers the full spectrum of known IOP-lowering mechanisms, giving it the potential to provide a greater IOP-lowering effect than any currently approved glaucoma product. The first Phase 3 registration trial for RoclatanTM, "Mercury 1," is expected to commence in the third quarter of 2015. The Company expects to commence two additional Mercury trials in 2016.
  
  
  
  
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Cleveland BioLabs, Inc.  (NASDAQ: CBLI) announced the company has submitted an application for pre-Emergency Use Authorization (pre-EUA) to the U.S. Food and Drug Administration (FDA) in support of use of entolimod as a medical radiation countermeasure. Entolimod would be indicated for reducing the risk of death following exposure to potentially lethal irradiation occurring as the result of a radiation disaster.
  
Pre-EUA is the regulatory path through which the FDA determines that certain unapproved medical products may be used in an emergency when there are no adequate, approved, and available alternatives. Products with pre-EUA status can be purchased by the US government for stockpiling in the event of a disaster. Achieving pre-EUA status in the United States can also support partnerships and
foreign government interest in this program.
  
In July 2014, the Company met with the FDA and confirmed that the entolimod efficacy and

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safety data and
animal-to-human dose conversion are sufficient to proceed with a pre-EUA submission for entolimod. CBLI has now prepared an application under the FDA Animal Rule that demonstrates drug efficacy in animals and safety in humans. A pivotal efficacy study in 179 nonhuman primates showed that a single intramuscular injection of entolimod given 25 hours after high-dose, whole-body irradiation increased survival from 27.5% in control animals to 75% in entolimod-treated animals. Clinical studies of entolimod in 176 human subjects have characterized the safety profile of entolimod and documented dose-dependent effects on the same efficacy biomarkers that were correlated with survival benefit in animals.
  
Yakov Kogan, PhD, MBA, Chief Executive Officer, stated, "We are very pleased to have achieved this significant milestone. We believe that entolimod efficacy and operational feasibility are best in class and that our pre-EUA submission is compelling and aligned with our previous FDA discussions. Our top priority will be to work with the FDA to facilitate their review of our application. There is no Prescription Drug User Fee Act (PDUFA) goal associated with review of a pre-EUA. However, we estimate a 6- to 9-month period for evaluation of our application, depending on the extent of the FDA's comments and questions. We have submitted the dossier electronically with the intent of facilitating the review process. We believe that a positive review of our submission may serve as the basis for the future commercialization of entolimod."
  
In a separate, parallel initiative, CBLI continues negotiations regarding two proposals with the Department of Defense office of Congressionally Directed Medical Research Programs. Awards under these proposals would fund additional studies supportive of future full licensure of entolimod under a Biologics License Application.
  
  
  
  
Also Monday:  
  
  
  
Adamas Pharmaceuticals, Inc. (Nasdaq:ADMS) today announced additional findings from its Phase 2/3 study (EASED) evaluating ADS-5102 for the treatment of levodopa-induced dyskinesia (LID), a movement disorder associated with the treatment of Parkinson's disease. 
  
Agile Therapeutics, Inc. (Nasdaq:AGRX) a women's health specialty pharmaceutical company focused on the development and commercialization of new prescription contraceptive products, announced today that Al Altomari, President and Chief Executive Officer will present at the JMP Securities Life Sciences Conference 2015 scheduled on Tuesday, June 23th, 2015 at 9:00 AM ET at the St. Regis Hotel in New York City.
  
Amarantus BioScience Holdings, Inc. (OTCQB:AMBSD), a biotechnology company focused on developing therapeutic and diagnostic products for neurological disorders and orphan indications, announced that data from the Phase 1/2a clinical study of eltoprazine for the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID) has been accepted for presentation at the 19th International Congress of Parkinson's Disease and Movement Disorders being held June 14-18, 2015, in San Diego, CA.
  
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that it will host an Analyst and Investor Day in New York City on June 22, 2015.
  
Apricus Biosciences, Inc. (Nasdaq:APRI), a biopharmaceutical company advancing innovative medicines in urology and rheumatology, today announced that it has enrolled the last patient in its RayVa™ Phase 2a proof-of-concept study.  
   
Argos Therapeutics Inc. (Nasdaq:ARGS), an immuno-oncology company focused on the development and commercialization of fully personalized immunotherapies for the treatment of cancer based on the Arcelis® technology platform, today announced that John D. Menditto has joined the Company as vice president of corporate communications and investor relations.
  
BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today the start of a Phase 3 pivotal study in the USA and Canada for Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy (OPMD).
  
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that EY awarded BioMarin CEO Jean-Jacques Bienaimé the EY Entrepreneur Of The Year® 2015 Award in the Health and Life Sciences category in Northern California. 
  
Burcon NutraScience Corporation (TSX:BU) (Nasdaq:BUR), a leader in functional, renewable plant-based proteins, will hold a conference call on Tuesday, June 23, 2015 at 5:00 p.m. Eastern time to discuss results for its fiscal fourth quarter and year ended March 31, 2015.
  
Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases and CureDuchenne, a national nonprofit organization dedicated to finding a cure for Duchenne muscular dystrophy, invite participants to join a webinar on Friday, June 19, 2015 at 3:00 p.m. ET/noon PT.
  
Everydayhero™, a Blackbaud, Inc. (Nasdaq:BLKB) company and innovative online giving platform, will empower MapMyFitness users to make their workouts count for the charities or causes closest to their hearts by participating in the #iworkoutbeCAUSE 30-Day Charity Challenge beginning June 16.
  
Galena Biopharma, Inc. (Nasdaq:GALE), a biopharmaceutical company developing and commercializing innovative, targeted oncology therapeutics that address major medical needs across the full spectrum of cancer care, today announced that data from the Company's Phase 2 clinical trial of GALE-401 was presented at the European Hematology Association 20th Congress in Vienna, Austria.
  
GrowBLOX Sciences, Inc. (OTCQB:GBLX), a biopharmaceutical company with state-of-the-art technologies in plant biology and cultivation designed to produce consistent medicinal cannabis, announced here today that it has received a $1.75 million funding commitment to enable GrowBLOX to complete construction and set-up operations at their MME Cultivation Facility in Las Vegas, Nevada.
  
Innocoll AG (Nasdaq:INNL), a global, commercial-stage, specialty pharmaceutical company that develops, manufactures and supplies a range of pharmaceutical products and medical devices using its proprietary collagen-based technologies, today announced that the first patient was dosed in the COACT-2 (COgenzia Adjuvant for Complete Therapy) Phase 3 study for the treatment of diabetic foot infections (DFIs) using Cogenzia, Innocoll's topically applied, bioresorbable collagen sponge. 
 
Intra-Cellular Therapies, Inc. (Nasdaq:ITCI), a biopharmaceutical company focused on the development of therapeutics for central nervous system (CNS) disorders, today announced completion of enrollment for the first Phase 3 clinical trial (ITI-007-301) of the Company's lead product candidate ITI-007 for the treatment of schizophrenia. 
  
Lipocine Inc. (Nasdaq:LPCN), a specialty pharmaceutical company, today announced the successful completion of its labeling "food effect study" for LPCN 1021, its Oral Testosterone product for hypogonadal men with low testosterone. 
  
Matinas BioPharma Holdings, Inc. (OTCQB:MTNB), a clinical-stage biopharmaceutical company, announced the presentation of data from a head-to-head study that showed MAT9001, a prescription-only omega-3 fatty acid-based composition under development for the treatment of hypertriglyceridemia, demonstrated superiority versus Vascepa® (icosapent ethyl) in reducing lipids, triglycerides, apolipoproteins and PCSK9 levels.
  
MTBC (Nasdaq:MTBC), a leading provider of web-based revenue cycle management services and healthcare IT solutions, today announced that its shareholders held their 2015 annual meeting on June 10, 2015, and reelected John Daly to serve another term on its board of directors.
  
Natural Health Trends Corp. (Nasdaq:NHTC), a leading direct selling company that markets premium quality personal care, wellness and "quality of life" products under the NHT Global brand, today announced it is set to join the Russell Global Index, the Russell 3000® Index and the Russell Microcap® Index when Russell Investments reconstitutes its comprehensive set of U.S. and global equity indexes after the close of market on Friday, June 26, 2015, according to a preliminary list of additions posted June 12, 2015 on Russell Indexes Reconstitution.
  
Nature's Sunshine Products (Nasdaq:NATR), a leading natural health and wellness company engaged in the manufacture and direct selling of nutritional and personal care products, announced that Chairman and CEO, Gregory L. Probert, has joined the Board of Directors of the Direct Selling Education Foundation (DSEF), a non-profit that advances understanding of direct selling and promotes the industry's commitment to ethics, consumer protection and self-regulation.
  
Onconova Therapeutics, Inc. (Nasdaq:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced the presentation of clinical data on rigosertib in HR-MDS at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria, June 11 – 14, 2015.
  
Portola Pharmaceuticals (Nasdaq:PTLA) today announced the appointment of Tao Fu as executive vice president, chief commercial and business officer, effective immediately. Mr. Fu joins Portola from Bristol-Myers Squibb (BMS), where he was vice president and head of mergers and acquisitions and alliance management. 
  
PRA Health Sciences (Nasdaq:PRAH), a leading global contract research organization, today announced its new Predictivv™ platform, the beginning of an entirely new approach on the role technology will play in the future of clinical development. 
  
TriVascular Technologies, Inc. (Nasdaq:TRIV) announced today that the U.S. Food and Drug Administration (FDA) has approved the Ovation iX Iliac Stent Graft for the Ovation Prime® Abdominal Stent Graft System, together with manufacturing enhancements that will facilitate a broader commercial launch of the product. 
  
Everydayhero™, a Blackbaud, Inc. (Nasdaq:BLKB) company and innovative online giving platform, will empower MapMyFitness users to make their workouts count for the charities or causes closest to their hearts by participating in the #iworkoutbeCAUSE 30-Day Charity Challenge beginning June 16.



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