Auspex Receives FDA Orphan Drug Designation Of SD-809; Actinium Pharmaceuticals Actimab-A Phase I/II Trial Interim Data Print E-mail
By William Kent   
Thursday, 06 November 2014 20:13
Below is a look at some of the headlines for companies that made news in the healthcare sector on November 6, 2014.
Auspex Pharmaceuticals, Inc. (NASDAQ:ASPX), a biopharmaceutical company dedicated to developing innovative medicines for people with movement disorders and other rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation of Auspex's investigational compound SD-809 for treatment of Huntington's disease. Auspex is currently evaluating the efficacy and safety of SD-809 for treating chorea associated with this disorder in a Phase 3 registration clinical trial and is on track to announce topline data from this study in December 2014.

"The orphan drug designation of SD-809 marks a major regulatory milestone for Auspex and a significant step forward in our goal to bring this novel treatment approach to patients as quickly as possible," said Pratik Shah, president and CEO of Auspex. "Huntington's disease is a rare and devastating genetic disorder with very limited treatment options. The orphan drug designation recognizes the significant need that exists among individuals living with this disease."

The FDA's Orphan Drug program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides sponsors with development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.


Actinium Pharmaceuticals, Inc. (NYSE MKT: ATNM), a biopharmaceutical company developing innovative targeted payload immunotherapeutics for the treatment of advanced cancers, today announced positive interim data from the ongoing Phase I/II trial of Actimab-A in older patients with newly diagnosed Acute Myeloid Leukemia ("AML"). Most notably, median overall survival ("OS") of the seven secondary AML patients (with prior myelodysplastic syndrome, or MDS) in the study was 9.1 months, which is a prolongation of life compared to historical norms of typically 2 to 5 months.1 Older AML patients are already higher risk, with secondary AML patients considered to have the more severe and less treatable form of AML, and the shortest expected survival. The clinical abstract will be published and available online in Blood, the official Journal of the American Society of Hematology.

"Alpha emitting isotopes may result in more efficient leukemia cell killing without the toxicity of intensive chemotherapy," said Joseph Jurcic, M.D., Professor of Medicine and Director of the Hematologic Malignancies Section of the Hematology/Oncology Division at Columbia University Medical Center, and lead study investigator. "In this study, Actimab-A was safely and effectively combined with low-dose chemotherapy in older AML patients. Even at this early stage in development, the tolerability of the regimen and promising survival data in this poor-risk population are highly encouraging and support our center's commitment to this program. Because many of these patients cannot tolerate intensive chemotherapy, potentially less toxic treatments such as this are desperately needed."

"We believe the data presented provide further evidence that Actimab-A has substantial clinical activity, including a survival benefit, in the hardest to treat AML patients," said Dragan Cicic MD, Chief Medical Officer of Actinium. "The potential efficacy in killing other treatment resistant leukemia cells combined with the limited side effects identified in the study to date could offer a new hope to patients whose age, comorbidities and nature of disease currently leaves them with very limited treatment options. We continue to work with a world-class team of clinical investigators to advance this program and technology."

The interim analysis from this company-sponsored trial is consistent with results from the prior three trials in Actinium's HuM195-Alpha Program. The abstract, Phase I Trial of Targeted Alpha-Particle Therapy Using Actinium-225 (225Ac)-Lintuzumab (Anti-CD33) in Combination with Low-Dose Cytarabine (LDAC) for Older Patients with Untreated Acute Myeloid Leukemia (AML), will be published and available online in Blood, the official Journal of the American Society of Hematology.

In this interim analysis, a total of 9 patients were evaluated thus far with a median age of 76 (range 73-81). All had intermediate or poor risk cytogenetics, and 7 of 9 patients had secondary AML as a result of prior MDS. These 7 secondary AML patients had a median OS of 9.1 months from study entry (range 2.3-24 months). Of these, 2 patients lived longer than 12 months and the longest surviving patient lived greater than 24 months. Overall, for all 9 patients median OS was 5.4 months (range 2.2-24 months).

Two dosing levels have been evaluated to date (0.5 or 1.0 μCi/kg/fraction), and the study is ongoing at higher doses until the maximum tolerated dose ("MTD") is reached. Despite not having yet reached MTD, the Company has observed significant bone marrow blast reductions, another important marker of efficacy. Of the 7 evaluable patients in the overall study, 5 patients (71%) had bone marrow blast reductions with a mean of 61% reduction.

The sites participating in this multi-center trial are Memorial Sloan Kettering Cancer Center, MD Anderson Cancer Center, Johns Hopkins Medicine, Columbia University Medical Center, University of Pennsylvania Health System, Fred Hutchinson Cancer Research Center, and the Texas Oncology-Baylor Charles A. Sammons Cancer Center. The Company expects to announce further information related to Actimab-A development subsequent to its Clinical Advisory Board meeting, during the ASH 2014 time frame (December 6-9, 2014).

Elderly, high risk patients ordinarily have a life expectancy of 5 or fewer months if treated with standard chemotherapy, though only about a third of them do receive treatment because of toxicity. The other two-thirds receive best supportive care, with 2 months survival, according to Oran and Weisdorf (Haematologica 2012; 1916-24). The majority (5 of 7) of the secondary AML patients receiving Actimab-A had been previously treated with hypomethylating agents, a criterion which would have excluded such patients from some other clinical trials. Previous treatment with hypomethylating agents, and subsequent failure, further demonstrates the disease severity of patients in the Actimab-A trial.

The safety profile of Actimab-A was satisfactory and acceptable for this patient population in this interim analysis. The only drug-related serious adverse events seen were related to myelosuppression, which is expected in the treatment of leukemia.

The antibody portion of Actimab-A, HuM195 (also known as lintuzumab) when labeled with alpha particles has been evaluated in three prior studies, including two studies of Bismab-A, which was an earlier, first generation construct, and an investigator sponsored trial with Actimab-A. Today's interim results mark the first look at what the Company believes are clinically meaningful data in the ongoing, Company sponsored Phase I/II trial of Actimab-A.

Secondary AML is a common form of AML in the U.S., and is defined as AML that develops following exposure to cytotoxic agents or as a subsequent event in another hematologic disorder, usually MDS. According to the American Cancer Society there is an annual incidence of 12,000 new cases of MDS in the U.S. Between 30% and 50% of these new cases go on to develop AML, or approximately 3,600-6,000 secondary AML patients in the U.S.

Also Thursday:

ACETO Corporation (Nasdaq:ACET)
, a global leader in the marketing, sale and distribution of products for Human Health, Pharmaceutical Ingredients and Performance Chemicals, announced today financial results for the first quarter of fiscal 2015 ended September 30, 2014.

Air Methods Corporation (Nasdaq:AIRM)
reported results for the quarter ended September 30, 2014.

Coherus BioSciences, Inc. (Nasdaq:CHRS)
announced today the pricing of its initial public offering of 6,296,300 shares of its common stock at a public offering price of $13.50 per share, before underwriting discounts, commissions and estimated expenses.

Cutera, Inc. (Nasdaq:CUTR)
, a leading provider of laser, light and other energy-based aesthetic systems for practitioners worldwide, announced that its picosecond laser platform, enlighten, received U.S. Food and Drug Administration 510(k) clearance for tattoo removal.

Diversicare Healthcare Services, Inc. (Nasdaq:DVCR)
, a premier provider of long-term care services, today announced its results for the third quarter ended September 30, 2014.

Emergent BioSolutions Inc. (NYSE:EBS)
reported financial results for the quarter and nine months ended September 30, 2014.

Endologix, Inc. (Nasdaq:ELGX)
, developer and marketer of innovative treatments for aortic disorders, announced today that Company management is scheduled to participate at three investor conferences in November.

Flexion Therapeutics, Inc. (Nasdaq:FLXN)
announced today that the company will host a conference call and webcast to report its third-quarter 2014 financial results on Thursday, November 13, 2014, at 4:30 p.m. Eastern Time.

Hyperion Therapeutics, Inc. (Nasdaq:HPTX)
today reported net revenue of $26.2 million during the third quarter of 2014 from the sales of its two products for the treatment of urea cycle disorders, RAVICTI® (glycerol phenylbutyrate) Oral Liquid and BUPHENYL® (sodium phenylbutyrate) Tablets and Powder.

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT)
, announced today that results from the FLINT trial evaluating obeticholic acid (OCA) for the treatment of nonalcoholic steatohepatitis (NASH) were published today in The Lancet.

LifeVantage Corporation (Nasdaq:LFVN)
today reported financial results for its first quarter ended September 30, 2014.

Medical Marijuana, Inc. (OTC Pink:MJNA)
is pleased to inform shareholders and the general public that HempMeds™ — a corporate portfolio company, exclusive master distributor and contracted marketing company — is the premier sponsor of the 3rd annual Marijuana Business Conference.

Minerva Neurosciences, Inc. (Nasdaq:NERV)
, a leader in the development of innovative therapies to treat neuropsychiatric diseases and disorders, today reported business highlights and financial results for the third quarter ended September 30, 2014.

MRI Interventions, Inc. (OTCQB: MRIC)
announced today that it will release its third quarter 2014 financial results on Thursday, November 13, 2014, after the close of the U.S. financial markets.

Novadaq® Technologies Inc. (Nasdaq:NVDQ) (TSX:NDQ)
, the leading developer and provider of clinically relevant fluorescence imaging solutions for use in surgical and diagnostic procedures, today announced financial results for its third quarter ended September 30, 2014.

Novavax, Inc. (Nasdaq:NVAX)
, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of recombinant nanoparticle vaccines and adjuvants, announced today that representatives of Novavax, along with a number of its research collaborators, will be making multiple oral presentations and poster presentations related to Novavax' RSV F-protein nanoparticle vaccine candidate (RSV F Vaccine) at the 9th International Respiratory Syncytial Virus Symposium to be held November 9 – 13, 2014 in Stellenbosch, South Africa.

Oncothyreon Inc. (Nasdaq:ONTY)
today reported financial results for the third quarter ended September 30, 2014.

Receptos, Inc. (Nasdaq:RCPT)
, a biopharmaceutical company developing therapeutic candidates for the treatment of immune and metabolic diseases, today announced that Faheem Hasnain, its President and Chief Executive Officer, will be presenting at the Credit Suisse Healthcare Conference at The Biltmore Hotel in Phoenix, Arizona.

Response Genetics, Inc. (Nasdaq:RGDX), a company focused on the development and sale of molecular diagnostic tests that help determine a patient's response to cancer therapy, will announce its third quarter 2014 financial results and give an operational update in a press release to be issued before the market opens on Thursday, November 13, 2014.

Repros Therapeutics Inc.® (Nasdaq:RPRX)
today reported the outcome from its meeting with the FDA to discuss the Company's expected NDA submission.

Rockwell Medical, Inc. (Nasdaq:RMTI)
, a fully-integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron replacement, secondary hyperparathyroidism today announced that the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food & Drug Administration (FDA) recommended that the Phase 3 Triferic efficacy and safety results support a positive benefit/risk to treat iron loss to maintain hemoglobin in patients with hemodialysis-dependent stage 5 chronic kidney disease (CKD). The ODAC voted in favor of Triferic by a vote of 8 to 3.

Tandem Diabetes Care®, Inc. (Nasdaq:TNDM)
, a medical device company and manufacturer of the t:slim® Insulin Pump, today announced that Kim Blickenstaff, president and CEO, will present a company update at the Stifel Healthcare Conference in New York, NY.

Tornier N.V. (Nasdaq:TRNX)
, a global medical device company focused on providing surgical solutions to orthopaedic extremity specialists, reported today its financial results for the third quarter ended September 28, 2014.

Vascular Solutions, Inc. (Nasdaq:VASC)
today announced that the Company is scheduled to present at the Canaccord Genuity Medical Technology and Diagnostics Forum in New York City on Thursday, November 20th at 2:30pm (EST).

Versartis, Inc. (Nasdaq:VSAR),
an endocrine-focused biopharmaceutical company that is developing a novel, long-acting form of recombinant human growth hormone (rhGH), today announced financial results for the three- and nine- months ended September 30, 2014.

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