Novavax To Launch Clinical Trial Of Ebola Vaccine; Sarepta Announces Update on Eteplirsen Print E-mail
By Marilyn Mullen   
Monday, 27 October 2014 15:55

Below is a look at some of the headlines for companies that made news in the healthcare sector on October 27, 2014.
Novavax, Inc. (NASDAQ: NVAX) will launch a Phase 1 clinical trial of an Ebola vaccine candidate in December, the company said at a conference Sunday in Philadelphia.

Novavax gained 5 percent in Monday's session, trading recently at $5.36 per share.

Vice President of Vaccine Development Gale Smith said in a news release Monday that in pre-clinical models, its glycoprotein recombinant nanoparticle vaccine candidate produced antibody levels "well within ranges" thought to protect against Ebola in rodents and monkeys.

Separately at the conference, Novavax said its vaccine candidate for influenza among newborns, pediatrics and elderly, could also benefit pregnant women.

A Phase 2 clinical trial in 330 women of childbearing age showed the vaccine reduced infections by 50 percent, the company said.

 

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Sarepta Therapeutics, Inc. (NASDAQ: SRPT)
, a developer of innovative RNA-based therapeutics, today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding its planned New Drug Application (NDA) submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

In meeting minutes received last week from a Type B Pre-NDA meeting that took place in September 2014, the FDA provided updated guidance regarding the specific data to be included as part of, or at the time of, Sarepta’s NDA submission. The guidance states that additional data are now required as part of the NDA submission, including the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. Additionally, the guidance requests more specific data including a minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group. The FDA indicated that further discussion with Sarepta “will be necessary to determine what would constitute a complete NDA.” Based on these requests, Sarepta plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA.

"We are committed to satisfying the FDA’s updated requests for these specific data to be included as part of an NDA submission and will continue to work with the Agency toward the goal of a complete and acceptable NDA filing," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We believe all of the data requests and additional FDA discussions that have currently been outlined can be completed in time for an NDA submission by mid-year 2015. Obtaining an FDA approval of eteplirsen for the DMD patients who may benefit from the drug continues to be our highest priority.”

Excerpts from the Pre-NDA Meeting Minutes related to information that the FDA is requesting as part of an NDA submission included:

"The sponsor should include 3-month data from at least 12 to 24 newly exposed patients at the time the NDA is submitted."

"Available data from the other patients enrolled in the new eteplirsen studies (studies 301, 203, 204) should also be included at the time the NDA is submitted, even if exposure is less than 3 months in duration."

"Additional data from later time points and from newly enrolled patients should be submitted in the 120-Day Safety Update."

"FDA strongly advises the sponsor to obtain and submit patient-level natural history data.FDA is prepared to appeal to the academic groups holding the data to allow the sponsor a means to acquire the data."

"The study 201/202 clinical site inspection conducted in May, 2014, after the issuance of the April 15, 2014, guidance letter, uncovered marked disparities in the immunohistochemistry methodology and concerns about the reproducibility of the data.The lack of confirmation of robust dystrophin measurement during the site visit necessitates including the independent assessment of dystrophin-positive fibers and 168-week efficacy data from study 201/202 in the NDA."

“FDA strongly urged the sponsor to submit the MRI data with appropriate natural history controls.”

The FDA also stated that “[a]dditional discussion between the sponsor and the FDA will be necessary to determine what would constitute a complete NDA.”



Also Monday:




Actinium Pharmaceuticals, Inc. (NYSE MKT: ATNM),
a biopharmaceutical company developing innovative targeted payload immunotherapeutics for the treatment of advanced cancers, and Albert Einstein College of Medicine of Yeshiva University, a research-intensive medical school in Bronx, NY, started development of an antibody construct labeled with actinium 225 using a novel technology that potentially allows for the expansion of use of the Company’s proprietary platform and enables further manufacturing improvements of alpha therapy technology based drug candidates.

Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR)
, a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases, announced that the Company will host a conference call and live audio webcast at 5:00 p.m. EDT on Thursday, October 30, 2014, to provide a business update and to discuss its third-quarter 2014 financial and operating results.

Anavex Life Sciences Corp. (OTCQB:AVXL)
, a clinical-stage biopharmaceutical company developing novel drug candidates to treat Alzheimer's disease, other diseases of the central nervous system (CNS) and various types of cancer, has signed a $500,000 Securities Purchase Agreement with Lincoln Park Capital Fund, LLC ("Lincoln Park"), a long time institutional investor in the Company.

Arno Therapeutics, Inc. (OTCQB:ARNI)
, a clinical stage biopharmaceutical company focused on the development of oncology therapeutics, today announced that it has advanced its Phase I clinical trial evaluating its lead compound onapristone in women with progesterone receptor expressing tumors to the expansion stage and has now begun enrolling patients at the recommended Phase II dose of 50mg extended release tablets twice daily.

Bacterin International Holdings, Inc. (NYSE MKT:BONE)
, a leader in the development of bone graft material and coatings for medical applications, has been awarded a grant from the Montana Board of Research and Commercialization Technology (MBRCT) in the amount of $192,484 for the development and commercialization of a novel regenerative medicine technology.

CareDx, Inc. (Nasdaq:CDNA),
a molecular diagnostics company focused on the development and commercialization of clinically differentiated, high value, non-invasive surveillance solutions for transplant recipients, today announced that it will release its financial results for the third quarter of 2014 after the close of trading on November 10, 2014.

Cara Therapeutics, Inc. (Nasdaq:CARA)
, a biopharmaceutical company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced the unexpected death of Dr. Robert Medve, the company's Chief Medical Officer.

Pluristem Therapeutics Inc. (Nasdaq:PSTI) (TASE:PLTR), a leading developer of placenta-derived cell therapy products, announced today that researchers at Case Western Reserve University will conduct a preclinical study of the Company's PLacental eXpanded (PLX)-RAD cells.

Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that safety and efficacy results from its Firdapse™ Phase 3 trial will be presented as part of an Industry Forum at the 61st Annual Meeting of the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) being held at the Savannah, Georgia International Trade and Convention Center, Oct. 29-Nov. 1.

Cellular Dynamics International (Nasdaq:ICEL)
, today announced that the National Eye Institute (NEI), a division of the National Institutes of Health (NIH), awarded the company a $1.2 million contract to manufacture clinically compatible induced pluripotent stem cells (iPSCs) and iPSC-derived human retinal pigment epithelial (RPE) cells.

Celator Pharmaceuticals, Inc. (Nasdaq:CPXX)
, a biopharmaceutical company that is transforming the science of combination therapy and developing products to improve patient outcomes in cancer, today announced that the target enrollment of 300 patients has been achieved in the company's multicenter, randomized, open-label Phase 3 study of CPX-351 (cytarabine:daunorubicin) Liposome for Injection versus the current standard of care, conventional cytarabine and daunorubicin therapy (7+3) in patients with untreated high-risk (secondary) acute myeloid leukemia (AML).

Chembio Diagnostics, Inc. (Nasdaq:CEMI)
, a leader in point-of-care (POC) diagnostic tests for infectious diseases, today announced that it has entered into an exclusive agreement with Integrated BioTherapeutics, Inc. (IBT), a biotechnology company focused on the discovery of novel vaccines and therapeutics for emerging infectious diseases.

Galena Biopharma, Inc. (Nasdaq:GALE)
, a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major unmet medical needs to advance cancer care, today announced that the Company will report its third quarter financial results on Monday, November 3, 2014 after the close of the financial markets.

Intellipharmaceutics International Inc. (Nasdaq:IPCI) (TSX:I),
a pharmaceutical company specializing in the research, development and manufacture of novel and generic controlled-release and targeted-release oral solid dosage drugs, today announced that the United States Food and Drug Administration ("FDA") has provided the Company with written notification that its Toronto, Canada manufacturing facility has received an "acceptable" classification.

Intra-Cellular Therapies, Inc. (Nasdaq:ITCI)
, a biopharmaceutical company focused on the development of therapeutics for central nervous system (CNS) disorders, today announced that it will host a conference call and a live webcast discussion at 8:30 a.m. Eastern Time on Monday, November 3, 2014, to discuss financial results for the third quarter ended September 30, 2014, and provide a business update.

Karyopharm Therapeutics Inc. (Nasdaq:KPTI)
, a clinical-stage pharmaceutical company, announced today that it has expanded its operational footprint with a wholly-owned European subsidiary and appointed Ran Frenkel, an international executive with significant experience managing European clinical operations and regulatory affairs activities within the biopharmaceutical industry, as Executive Vice President, Worldwide Clinical Development.

Marrone Bio Innovations, Inc. (Nasdaq:MBII), a leading provider of bio-based pest management and plant health products for the agriculture, turf and ornamental and water treatment markets, today announced that Brian Ahrens has joined the company as its Vice President of Sales.

NeoStem, Inc. (Nasdaq:NBS)
, a leader in the emerging cellular therapy industry, announced today that Dr. Robert Preti, the Company's Chief Scientific Officer and President of its subsidiary, Progenitor Cell Therapy ("PCT"), was honored with the Excellence in Leadership award at the 2014 BioProcess International ("BPI") Awards.

NewLink Genetics Corporation (Nasdaq:NLNK)
will release its third quarter 2014 financial results on Thursday, November 6, 2014.

Pain Therapeutics, Inc. (Nasdaq:PTIE)
today reported it believes it will regain full development and commercial rights to REMOXY from Pfizer, Inc. On Friday, October 24th, Pfizer hosted a conference call with us during aftermarket hours to inform us of their intention to terminate development of REMOXY. Actual termination will become effective six months from today, pursuant to the terms of a Collaboration Agreement.

Progenics Pharmaceuticals, Inc., (Nasdaq:PGNX)
, an oncology company focused on developing innovative ways to target and treat prostate cancer, announced today that it will host a conference call and webcast to review third quarter 2014 financial results on Friday, November 7, at 8:30 a.m. ET.

The report by the New York City Office of the Chief Medical Examiner on the death of Joan Rivers points to a "predictable complication of medical therapy." Vision-Sciences (Nasdaq:VSCI) believes one solution to these complications is a sedation-free exam for the patient.




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