News Column

OPKO Announces Positive Interim Six-Month Lagova™ (hGH-CTP) Phase 2 Data; Pluristem Selects Rotator Cuff Repair as New Orthopedic Indication PLX Cells

June 25, 2014



Below is a look at some of the headlines for companies that made news in the healthcare sector on June 25, 2014.

{nfg}OPKO Health, Inc. (NYSE: OPK)
, a multinational biopharmaceutical and diagnostics company, announced 6 month results of a Phase 2 dose-finding study evaluating the safety and efficacy of its novel long-acting human growth hormone product (Lagova) to treat pediatric growth hormone deficiency disorder (GHD).



All three Lagova once-weekly doses demonstrate strong catch-up growth during the six months treatment. The annualized growth rates are above 12 cm in all three doses. The results are supported by excellent dose dependent pharmacokinetics (PK) and pharmacodynamics (PD) profiles. Lagova shows a promising safety profile with no serious adverse events reported. Glucose and lipid metabolism markers are within the normal ranges. No lipoatrophy was observed in any patients dosed, and no clinically significant local tolerability issues were identified.



“The interim results further affirm that a once-weekly administration of Lagova can replace daily injections of marketed hGH in pediatric GHD patients. The results enable dose selection for the company’s upcoming Phase 3 pediatric trial,” said Dr. Ron Rosenfeld, clinical advisor on the study and professor of Pediatrics (emeritus), Stanford University and professor of Pediatrics at Oregon Health and Science University (emeritus). “Because Lagova consists of native human growth hormone attached to a C-Terminal Peptide of endogenous hormone, one would anticipate low immunogenicity,” Rosenfeld noted.



“Based on these encouraging safety and efficacy results, OPKO plans to move aggressively into a single confirmatory pivotal Phase 3 study for pediatric GHD patients. We hope to make Lagova available to pediatric GHD patients as soon as possible,” said CEO, Phillip Frost, M.D. “Lagova is one of a family of important products being developed at OPKO Biologics designed to improve compliance and offer ease of administration to patients.”



Study Design -- The randomized, comparator-controlled Phase 2 study was conducted in up to 56 pre-pubertal, naÏve GHD children receiving one of three Lagova doses as once-weekly regimen (0.25, 0.48, 0.66mg/Kg/week; equivalent of 0.18, 0.35, 0.48 mg/Kg/week of hGH) or daily hGH (34µg/Kg/day) subcutaneously. In order to introduce naÏve patients to the allocated Lagova dose in a gradual manner, a stepwise dose increase approach was implemented. Once patients reached the targeted doses, Lagova, GH, IGF-1 and IGF-BP3 concentrations were measured and PK-PD analysis was conducted utilizing a population based approach.



Study Results -- An interim analysis of 6 months data demonstrated that all doses of Lagova used in the study provided strong catch-up growth response better than historical controls of daily growth hormone therapy.



The baseline characteristics of all patients were comparable among all groups. Interim analysis of the PK profile following administration of Lagova demonstrates a significantly extended half-life as reflected by the T1/2 and AUC respectively. A dose dependent PD (IGF-1) response was observed between Lagova cohorts, reaching steady state with no accumulation or excessive levels. All cohorts demonstrated promising “catch-up” growth, in line with reported age and GHD severity-matched data. The annualized height velocities are more than 12 cm, which correlates with the PK/PD profile in those patients.







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Pluristem Therapeutics Inc., (NASDAQ:PSTI)
a leading developer of placenta-based cell therapies, today announced it has selected rotator cuff repair as the second indication in its orthopedic clinical program. The Company's PLacental eXpanded (PLX) cells offer potential as the first off-the-shelf cell therapy product for muscle and tendon regeneration that can be used to enhance repair after rotator cuff surgery. There are currently no FDA-approved therapies using engineered cells for the treatment of rotator cuff injuries.



According to the American Academy of Orthopaedic Surgeons (AAOS), nearly 2 million patients in the U.S. seek help for their rotator cuff injuries. The incidence of these injuries increases with age, so as the U.S. population ages the prevalence and potential cost burden is likely to grow.



"Following the favorable results of our Phase I/II trial in our first orthopedic indication, the treatment of injured gluteal muscle after total hip replacement, we, together with Key Opinion Leaders in orthopedic surgery, selected rotator cuff repair as an important indication in need of a novel cell therapy solution. We are progressing with our study design, and are considering the addition of a third orthopedic indication," stated Pluristem CEO Zami Aberman.



"There is a clear and unmet need for a new kind of therapy that can improve the outcome of orthopedic surgery for rotator cuff tears. Our PLX cells can be administered as a simple office procedure. The team of Key Opinion Leaders advising us is particularly excited by the potential of a non-invasive, effective cell therapy for orthopedic indications," added Aberman.







Also Wednesday:





Advaxis, Inc. (Nasdaq:ADXS),
a clinical-stage biotechnology company developing cancer immunotherapies, today announced that the Company will be added to the Russell Microcap Index when Russell Investments reconstitutes its comprehensive set of U.S. and global equity indices on June 27.



Dehaier Medical Systems Ltd. (Nasdaq:DHRM)
, an emerging leader in the development, assembly, marketing and sale of medical devices and sleep respiratory products in China, today announced that it attended the 8th Biennial Chinese Sleep Research Society ("CSRS") Conference and 20th Anniversary Celebration of CSRS, held in Beijing, from June 13-15, 2014, to showcase its Morpheus OX System and work station.



Hemispherx Biopharma, Inc. (NYSE MKT:HEB)
, will present a corporate update on their Advanced Therapies for the Human Immune System at 9:45 a.m. Pacific Time on June 26, during the 2014 BIO International Convention at the San Diego Convention Center.



Hyperion Therapeutics, Inc. (Nasdaq:HPTX)
today announced that the European Medicines Agency (EMA) has completed technical and content validation and accepted the filing of the Marketing Authorisation Application (MAA) for RAVICTI (glycerol phenylbutyrate) Oral Liquid for the treatment of urea cycle disorders (UCDs).



Integrated Environmental Technologies, Ltd. (OTCBB:IEVM)
today announced that Carson Tahoe Health System has published the results of Carson Tahoe's independent study testing the effectiveness of Excelyte™ in reducing the amount of bacteria present in a hospital setting.



Intuitive Surgical, Inc. (Nasdaq:ISRG)
, the global leader in robotic-assisted minimally invasive surgery, today announced it has received a CE Mark for its da Vinci Xi Surgical System.



Merrimack Pharmaceuticals, Inc. (Nasdaq:MACK)
today announced detailed results from NAPOLI-1, a large, randomized, three-arm Phase 3 study of MM-398, a nanoliposomal encapsulation of irinotecan, in patients with metastatic pancreatic cancer previously treated with gemcitabine-based therapy.



Premier, Inc. (Nasdaq:PINC)
today announced that its subsidiaries have entered into a new, five-year unsecured revolving facility that provides up to $750.0 million of borrowing ability.



Prosensa Holding N.V. (NASDAQ: RNA)
, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the successful completion of enrollment in its prospective Natural History Study designed to increase the understanding of Duchenne muscular dystrophy (DMD).



Prothena Corporation plc (Nasdaq:PRTA)
, a clinical stage biotechnology company focused on the discovery, development and commercialization of novel antibodies for the potential treatment of diseases that involve protein misfolding or cell adhesion, announced today that it has priced an underwritten public offering of 4,750,000 of its ordinary shares at a price to the public of $22.50 per ordinary share, before underwriting discounts and commissions.



Spine Pain Management, Inc. (OTCQB:SPIN)
, a technology-driven, financial service, device and healthcare solution company servicing the multi-billion dollar spine injury sector, today announced the formal addition of Back Pain Institute of West Texas (BPIWT) as a new Diagnostic Affiliate.



SunOpta Inc. (Nasdaq:STKL) (TSX:SOY)
, a leading global company focused on natural and organic foods, today announced the release of its 2013 Corporate Social Responsibility Report.



Verde Media Group Inc. (OTC:VMGI)
through its Verde Media Biotechnology Division ("VMBD") announces the set-up plans for the launch of the VMBD-03 rapid omega-3 oil profiling laboratory with joint-venture partner Phoenix Energy in Asia.


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Source: BioMed Reports


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