Your Daily Pharma Scoop: Voyager Advances, Roche Succeeds, Eisai Positive


Stocks in News: VYGR, RHHBY

Voyager advancing gene therapy for Parkinson’s, Phase 2 data may be sufficient for U.S. marketing application

Discussion: Voyager Therapeutics (VYGR) announced its plans to submit a BLA seeking approval of its gene therapy VY-AADC for Parkinson’s disease (‘PD). The plan is backed by FDA feedback of its Phase 2 data. The company sources stated that they plan to include Phase 3 results in the application only if the mid-stage study stumbles at some point. Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease that affects ~1M people in the U.S. and ~7-10 M people worldwide.

Further, preliminary trial data from a Phase 1 posterior trajectory trial were consistent with the improvements observed in its Phase 1b trial. The earlier demonstrated benefits like improved coverage of the putamen, reduced surgical times and improved motor functions at sixth month. Because of the above results the preferred surgical route of administration will now be the posterior approach. Pivotal studies continued the same dose concentration of the earlier Phase 1b study.

Apart from the trial stage VY-AADC, the company has a number of candidates in mature preclinical stages. These are indicated on various severe neurological diseases. The adeno-associated virus (‘AAV) gene therapy approach that controls the production of a specific protein is the main technology platform that lies at the core of the company’s development pipeline.

Three of the other advanced candidates of the company pipeline are VY-SOD101 on Monogenic Amyotrophic lateral sclerosis (‘ALS), VY-HTT01 on Huntington’s disease, VY-FXN01 on Friedrich’s ataxia etc. It has two more candidates in early preclinical stage. The incidence of ALS is about 2/0.1M person-years, and the prevalence is about 5/0.1M persons. In spite of the low incidence ALS should not be considered to be infrequent. By the age of 85, the lifetime risk rises to 1 in 300.

Roche’s antiviral balovavir marboxil successful in late-stage study

Discussion: Roche (OTCQX:RHHBY) announced meeting of primary endpoint of a Phase 3 trial of its candidate baloxavir marboxil. The trial was indicated on people at high risk of complications from the flu. Key secondary endpoints were also met by baloxavir marboxil and it further demonstrated its superiority over placebo and Tamiflu (oseltamivir phosphate). Secondary endpoints included time that the virus continued to be released and reducing viral levels in the body. Favourable safety profile was observed too.

The drug was discovered and developed by Shionogi & Co. The drug is marketed as Xofluza in Japan. Recent estimates think that the market for seasonal influenza vaccines will grow by almost $1B from $3.1B in 2015 to $4.3B by 2025 across the 7 MM. PDUFA is scheduled on December 24.

In other News

Eisai’s Belviq demonstrates favorable safety profile in large-scale CV outcomes study

Eisai (OTCPK:ESALY) reported positive data from a 12,000-subject cardiovascular (‘CV) outcomes study evaluating safety and potential CV benefits of weight loss med BELVIQ (lorcaserin HCl). FDA requirement mandated this post-marketing study.

Second late-stage Alzheimer’s study of AC Immune’s crenezumab fully enrolled

News of complete enrolment of the Phase 3 clinical trial of the AC Immune’s SA (NASDAQ:ACIU) evaluating its candidate crenezumab in patients with prodromal or early Alzheimer’s disease is announced. Genentech is conducting the study as a licensee.

Therapix Bio up 13% premarket on preclinical data on lead drug THX-160

Therapix Biosciences (NASDAQ:TRPX) announced encouraging preclinical data for its pain candidate THX-160. The drug was well-tolerated with no major adverse effects. It also showed comparable analgesic efficacy to high-dose morphine.

Arcus Bio’s AB928 shows favorable safety profile in early-stage study

Phase 1 unblinded data of Arcus Biosciences’ (NYSE:RCUS) dual adenosine receptor antagonist AB928 showed that the candidate was safe and well-tolerated at all evaluated doses. All adverse events were resolved by the conclusion of the study period.

Can-Fite granted Australian and Chinese patents for erectile dysfunction drug

Can-Fite BioPharm (NYSEMKT:CANF) receives patents from Australian and Chinese patent offices for the utilization of A3 adenosine receptor ligands in the treatment of sexual dysfunction.

Seattle Genetics down 1% on suspension of brentuximab vedotin study

Seattle Genetics (NASDAQ:SGEN) suspended an NCI-sponsored Phase ½ clinical trial of antibody-drug conjugate brentuximab vedotin + chemo in patients with stage II – IV human immunodeficiency virus (HIV)-associated Hodgkin lymphoma.

Cellectar’s CLR 131 shows encouraging action in mid-stage blood cancer study

Cellectar Biosciences (NASDAQ:CLRB) announced positive results from its Phase 2 clinical trial of CLR 131. 94% reduction in tumor burden and complete resolution in four of five targeted tumor masses was observed in the patient with advanced Waldenstrom macroglobulinemia.

Asterias up 9% premarket on updated data on AST-OPC1

Asterias Biotherapeutics (NYSEMKT:AST) released six-month data of its ongoing Phase 1/2a clinical trial of its candidate AST-OPC1 in patients with severe spinal cord injury. Out of 25 patients no major adverse events were related yet to the therapy. Some sections of the result showed definite statistically significant improvement.

Baxter receives FDA Warning Letter related to injectables manufacturing site in India

Baxter International (NYSE:BAX) informs of a warning letter it the FDA regarding certain observations noted during a July 2017 on-site inspection of its recently acquired Claris injectables manufacturing site. The company recently acquired this manufacturing site in Ahmedabad, India.

Axovant Sciences up 1% on U.S. patent covering nelotanserin for sleep behavior disorder

A new U.S. patent has been issued to Axovant Sciences (NASDAQ:AXON) covering nelotanserin for sleep behavior disorder.

Pulmatrix’s Pulmazole successful in early-stage study; shares up 4%

Pulmatrix (NASDAQ:PULM) announced positive data from Part 3 of its Phase 1/1b study of Pulmazole, for the treatment of allergic bronchopulmonary aspergillosis in asthma patients. Pulmazole is an inhaled iSPERSE formulation of antifungal med itraconazole. The study met all objectives.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Editor’s Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.



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