Jazz Pharma and Concert Pharma Provide JZP-386 Update; ARIAD Reports Follow-up Data from Phase 1, PACE Trials of Ponatinib Print
By William Kent   
Tuesday, 09 December 2014 20:13
Below is a look at some of the headlines for companies that made news in the healthcare sector on December 9, 2014.
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) announced Phase 1 clinical data generated to date supports completing the Phase 1 evaluation of JZP-386 at the originally planned highest dose, which was not administered in the first Phase 1 trial due to a technical dosing issue. The existing Phase 1 clinical data was generated in a first-in-human trial evaluating the safety, pharmacokinetics, and pharmacodynamics of JZP-386; enrollment was completed in the third quarter. A second Phase 1 trial evaluating JZP-386 at the originally planned highest dose is expected to be initiated in the first quarter of 2015, with data expected in the second quarter of 2015.

JZP-386 is a deuterium-containing analog of sodium oxybate. The Phase 1 program is comparing JZP-386 to sodium oxybate versus placebo in healthy volunteers. The companies expect that the results from the upcoming Phase 1 trial will inform the next steps in the development program for JZP-386.

Sodium oxybate is the active ingredient in Xyrem®, a prescription medicine marketed in the United States by Jazz Pharmaceuticals to treat cataplexy and excessive daytime sleepiness in patients with narcolepsy, a serious neurological disorder that affects approximately 1 in 2000 people in the United States.


 
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ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA)
announced safety and efficacy follow-up data on Iclusig® (ponatinib), its approved BCR-ABL inhibitor, in patients with a baseline T315I mutation from its Phase 1 and Phase 2 PACE trials in heavily pretreated patients with resistant or intolerant chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

With a median follow-up of three years for CP-CML patients and nine months overall, Iclusig continues to exhibit responses in patients with the T315I mutation, for whom there is no other approved tyrosine kinase inhibitor (TKI) therapy. Among patients with the T315I mutation, major cytogenetic response (MCyR) was achieved by 92 percent (11/12) of chronic phase CML patients (CP-CML) in the Phase 1 trial and 72 percent (46/64) of CP-CML patients in the PACE trial; taken together, the combined MCyR rate for all T315I CP-CML patients was 75 percent. Long-term safety data show that careful benefit-risk evaluations should guide decisions to use and maintain ponatinib therapy, particularly in patients who may be at increased risk for arterial thrombotic events.

These data were featured on Monday December 8, at the 56th Annual Meeting of the American Society of Hematology (ASH) taking place in San Francisco.

“For CML patients with the T315I mutation, there were few treatment options available prior to ponatinib, and prognosis for these patients was poor. Now, with nearly six years of treatment for some patients in these trials, we have longer term data demonstrating that ponatinib continues to evoke responses in these CP-CML patients,” stated Michael J. Mauro, M.D., Leader, Myeloproliferative Neoplasms Programs, at the Memorial Sloan Kettering Cancer Center. “With these follow-up data, overall survival of 78 percent is now estimated at three years for chronic-phase patients with the T315I mutation in these trials.”

Phase 1 and PACE Data in Patients with T315I - The analysis describes the pooled efficacy and safety of ponatinib in patients with a T315I mutation detected at baseline from the ongoing Phase 1 dose-escalation study and Phase 2 PACE trials. The analysis includes 147 patients with a T315I mutation from the Phase 1 (n=19) and PACE (n=128) trials. Half of the 76 CP-CML patients with T315I remain on treatment and 44 (30%) total patients (all disease phases) with T315I remain on treatment in the respective trials. Data presented on these patients are as of September 26, 2014 for the Phase 1 trial and October 6, 2014 for the PACE trial.

  • Median follow-up for all T315I patients in this analysis is 8.9 months. The median follow-up for CP-CML patients with T315I (n=76) is three years (maximum follow-up, 70 months).
  • In these heavily pre-treated T315I patients, 45 percent of the total patients had received treatment with two prior TKIs, and 40 percent had received three or more prior TKIs. In patients with CP-CML, 46 percent had received two prior TKIs, and 39 percent had three or more prior TKIs.
  • For CP patients, responses continue to be observed in T315I patients treated with ponatinib at 3 years. In the combined analysis of these patients from both trials:
  • 75 percent of all CP-CML patients achieved MCyR, 72 percent achieved a complete cytogenetic response (CCyR), and 61 percent achieved a major molecular response (MMR).
  • By Kaplan-Meier estimate, 83 percent of these CP patients were estimated to maintain MCyR at 3 years, and 81 percent were estimated to maintain CCyR at 3 years. The median duration of response has not yet been reached.
  • 58 percent of advanced phase (AP) CML patients with T315I achieved major hematologic response (MaHR), the end-point for those patients in the trials. Twenty-seven percent of blast phase (BP) CML patients, and 38 percent of Ph+ ALL patients achieved MaHR.
  • The probability for overall survival in CP patients with T315I at 36 months is 78 percent and 63 percent for patients with AP-CML.
  • The most common treatment-emergent adverse events in T315I patients (all disease phases) were rash (42%), abdominal pain (39%), headache (39%), and nausea (36%). The most common serious treatment-emergent adverse events were neoplasm progression (10%), pneumonia (7%), and acute myocardial infarction (5%).
  • Thirty-two percent (n=24) of CP-CML patients with T315I experienced arterial thrombotic events, and 7 percent experienced a venous thrombotic event (VTE). Exposure-adjusted incidences of arterial and venous thrombotic events in patients with the T315I mutation were similar to those observed in the overall patient population.

“Ponatinib is the only TKI approved for patients with the T315I mutation of BCR-ABL, which is a common mutation that is associated with resistance in Philadelphia-positive leukemias,” said Frank G. Haluska, M.D., Ph.D., chief medical officer and senior vice president, clinical R&D at ARIAD Pharmaceuticals. “The combined data from the two ongoing trials of ponatinib show continued, clinically meaningful responses in this difficult-to-treat patient population.”

 

 
Also Tuesday:



Agios Pharmaceuticals, Inc. (Nasdaq:AGIO)
, a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, today announced the addition of Kaye Foster-Cheek as an independent director to the company's board of directors, effective immediately.

Analogic Corporation (Nasdaq:ALOG)
, enabling the world's medical imaging and aviation security technology, today announced results for its first quarter ended October 31, 2014.

AngioDynamics (Nasdaq:ANGO)
, a leading provider of innovative, minimally invasive medical devices for vascular access, surgery, peripheral vascular disease and oncology, today announced it signed an agreement with HealthTrust covering PICC (peripherally inserted central catheter) products.

Arno Therapeutics, Inc. (OTCQB:ARNI)
, a clinical stage biopharmaceutical company focused primarily on the development of oncology therapeutics, today announced that data from a study supporting diagnostic development for its lead compound onapristone will be presented at a poster session at the 2014 San Antonio Breast Cancer Symposium (SABCS), being held December 9-13, 2014 in San Antonio, Texas.

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN)
will host an Analyst and Investor Day from 8am (ET) to 1pm (ET) on Wednesday, December 10, 2014 at The Plaza Hotel.

Calithera Biosciences, Inc. (Nasdaq:CALA)
announced today the signing of an exclusive global license agreement with Mars Symbioscience ("Symbioscience"), a division of Mars, Incorporated, granting Calithera rights to research, develop and commercialize Symbioscience's portfolio of arginase inhibitors, discovered as part of Mars' cocoa flavanol research program, for use in human healthcare.

Cancer Genetics, Inc. (Nasdaq:CGIX)
, an emerging leader in DNA-based cancer diagnostics, announced today that it has received preliminary approval for a $570,000 tax credit from the New Jersey Technology Business Tax Certificate Transfer Program for the year 2014.

CONMED Corporation (Nasdaq:CNMD)
today announced a number of management transitions. After 25 years of service, Robert D. Shallish, Jr., Executive Vice President, Finance and Chief Financial Officer, recently informed the Company of his plan to retire on March 31, 2015.

Ekso Bionics Holdings, Inc. (OTCQB:EKSO)
, announced the appointment of Stanley Stern as an independent director of the firm, effective immediately. Mr. Stern's appointment expands the Board to six directors.

Eternity Healthcare Inc. (OTC:ETAH)
, a medical device and diagnostic company with advanced technologies in Needle-Free Injection system, announced today that the Food and Drug Administration (FDA) has given 510(K) clearance to West Pharmaceutical Services, Inc. to start producing Needle-stick Guards to help the Medical Professionals administer vaccines in a safer way.

FutureWorld Corp. (OTCQB; FWDG)
, a leading provider of advanced technologies and solutions to the global cannabis industry, announces today that FutureLand Properties, LLC, a subsidiary of FutureWorld Corp., has entered into a 5 Year land and greenhouse lease agreement with Colorado Flower Company, LTD, a Colorado LLC, for the growing of medical and recreational cannabis.

GrowBLOX Sciences, Inc. (OTCQB:GBLX)
, a company that focuses on plant biology and cultivation technology in order to optimize safe and consistent medical cannabis, today announced the appointment of Cathryn Kennedy as its Chief Financial Officer, effective immediately.

HealthEquity, Inc. (Nasdaq:HQY)
, one of the largest HSA non-bank custodians, today announced financial results for its third quarter ended October 31, 2014.

Hemispherx Biopharma, Inc. (NYSE MKT:HEB)
, announced today that it has received a new research report (dated December 5, 2014) from researchers at Howard University, Washington DC.

The Joint Corp. (Nasdaq:JYNT)
, a national healthcare franchisor of chiropractic clinics, today announced that it will release its third quarter 2014 financial results on Monday, December 22, 2014, at 9:00 a.m. EST (7:00 a.m. MST), with a conference call to follow at 11:00 a.m. EST (9:00 a.m. MST).

MediciNova, Inc., (Nasdaq:MNOV)
a biopharmaceutical company and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for a pending patent application which covers  MN-001 (tipelukast) and MN-002 (a major metabolite of MN-001) for the treatment of certain liver disorders.

Myriad Genetics, Inc. (Nasdaq:MYGN)
today announced results from a new study that demonstrated the ability of the myRisk™ Hereditary Cancer test to detect 105 percent more mutations in cancer causing genes than conventional BRCA testing alone.

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 and LifeNet Health®, a world leader in allograft bio-implants and cellular therapies, today jointly announced the signing of a multi-year agreement pursuant to which, NOVADAQ was appointed exclusive worldwide distributor of LifeNet Health's DermACELL® tissue products for wound and breast reconstruction surgery. 

RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL),
an Israeli biopharmaceutical company focused on late clinical-stage drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced that it has submitted a Marketing Authorization Application to the UK Medicines and Healthcare Products Regulatory Agency and several other member states seeking European marketing approval of RHB-102 for cancer patients suffering from chemotherapy and radiotherapy-induced nausea and vomiting. RHB-102, newly branded as BEKINDA™, is a proprietary, oral, extended-release, once-daily pill formulation of the antiemetic drug ondansetron. BEKINDA™ is targeting a considerable segment of the 5-HT3 antiemetic market, with estimated worldwide sales of approximately $940 million in 20131.
 
Sabra Health Care REIT, Inc. (Nasdaq:SBRA)
announced today that Fitch Ratings has initiated credit ratings on the Company with a "BB+" Issuer Default Rating ("IDR") and the Rating Outlook at Stable.

Sanomedics International Holdings, Inc. (OTCQB:SIMH)
, a medical technology holding company that focuses on providing game changing products, services and ideas, announced today that the Company's Thermomedics, Inc. division, makers of the world's leading TouchFree™ Clinical Thermometer, Caregiver®, has signed a comprehensive distribution agreement with Henry Schein, Inc. (Nasdaq:HSIC).

TG Therapeutics, Inc. (Nasdaq:TGTX)
, an innovative, clinical-stage biopharmaceutical company today announced clinical results from its Phase 1/2 clinical study of TG-1101 (ublituximab), the Company's novel glycoengineered anti-CD20 monoclonal antibody, in combination with TGR-1202, the Company's novel once per day PI3K delta inhibitor.

VeriTeQ Corporation (OTC Markets:VTEQ)
a provider of implantable medical device identification and radiation dose measurement technologies, announced today that more than 5,000 women have now received Motiva Implant Matrix® breast implants, manufactured by Establishment Labs S.A. ("EL"), with VeriTeQ's Q Inside Safety Technology™.

ViaDerma, Inc. (OTC:VDRM), a specialty pharmaceutical company devoted to bringing new products to market, recently announced TetraStemTM, a topical liquid tetracycline-based antibiotic that uses a patent-pending innovative transdermal delivery system that can convert oral medication active ingredients into topical drugs.

XOMA Corporation (Nasdaq:XOMA)
, announced today the pricing of 8,097,165 shares of its common stock and accompanying warrants to purchase one share of common stock for each share purchased at a price to the public of $4.94. The warrants are exercisable at an exercise price of $7.90 per share beginning on the date of issuance and will expire on the second anniversary of the date of issuance.



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