All posts in Peptide News from Biotech and Pharma

Source: https://globenewswire.com/
NOVATO, Calif., July 14, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced positive topline data from the pivotal Phase 3 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome). The study met its primary endpoint of reducing urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, demonstrating a reduction from baseline of 64.8 percent (p<0.0001). The data are being presented at the 14th International Symposium on MPS and Related Diseases. “Treatment with rhGUS showed a rapid and sustained reduction in urinary GAG excretion, as well as signs of clinical improvement in this heterogeneous patient population,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We look forward to working with the FDA and EMA to file these data with the goal of bringing this potential treatment to patients with MPS7 who currently have no other options.” The study provides evidence of clinical improvement with rhGUS treatment. The Multi-domain Responder Index (MDRI) score at 24 weeks of treatment, a secondary endpoint, demonstrated an overall mean improvement (±SD) of +0.5 domains (±0.80) (p=0.0527). Six of the 12 patients had an improvement in their MDRI score of +1 or more. Five patients demonstrated no worsening of this progressive disease, or an MDRI score of 0. One patient had an MDRI score of -1. The MDRI is a summation of scores from each of the following domains: the six-minute walk test (6MWT), forced vital capacity (FVC), shoulder flexion, visual acuity, and the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) fine motor and gross motor function. For each domain, patients received a score of +1 when they demonstrated improvement of a magnitude equal or greater than a pre-defined minimally important difference (MID), -1 for worsening of one MID or greater, and 0 for any change that was less than 1 MID. As expected, many patients were unable to perform one or more tests due to physical or cognitive limitations and were not evaluated on those tests. Read more: https://globenewswire.com/news-release/2016/07/14/856153/0/en/Ultragenyx-Announces-Positive-Topline-Data-from-Phase-3-Study-of-Recombinant-Human-Beta-Glucuronidase-in-Mucopolysaccharidosis-Type-7.html

Source: http://www.businesswire.com/
NEW YORK--(BUSINESS WIRE)--Synergy Pharmaceuticals Inc. (NASDAQ:SGYP) today announced it has reached the Food and Drug Administration (FDA) mid-cycle review milestone for the plecanatide new drug application (NDA) in chronic idiopathic constipation (CIC). Additionally, the company is providing an update on the ongoing irritable bowel syndrome with constipation (IBS-C) program.
We are pleased with the progress and ongoing dialogue with the FDA,” said Gary S. Jacob, Chairman and CEO of Synergy Pharmaceuticals, “We remain optimistic about the potential approval of plecanatide in CIC by the PDUFA date of January 29, 2017. I am also very pleased with the progress our commercial, medical affairs and supply chain teams have made in recent months as we anticipate bringing this important new treatment option to CIC patients and their caregivers early next year.”

Furthermore, the company has decided to continue patient enrollment for its two ongoing phase 3 clinical trials with plecanatide in IBS-C. The decision to continue enrollment comes after trial monitoring demonstrated a slower enrollment pace combined with an increase in the number of patients not meeting randomization criteria after the screening period prior to starting treatment.

“Our first priority is to ensure high quality trials that reflect our rigorous standards and expectations," said Dr. Jacob, "To that end, we are updating our timing for top-line data in both trials to the fourth quarter of this year and intend to file the plecanatide NDA in IBS-C in the first quarter of 2017. We remain confident that plecanatide will continue to deliver outstanding clinical results consistent with previous trials, ultimately providing an important treatment option for patients suffering from IBS-C.”
Read more: http://www.businesswire.com/news/home/20160715005112/en/Synergy-Pharmaceuticals-Update-Ongoing-FDA-Review-Plecanatide

Source: http://www.marketwired.com/

LA JOLLA, CA--(Marketwired - Jul 14, 2016) - Panther Biotechnology, Inc. (OTC PINK: PBYA) (Panther), a biotechnology company specializing in the development of enhanced therapeutics for the treatment of neoplastic and autoimmune disorders, is pleased to announce today that it has initiated the formal drug development process for its lead compound, Transferrin Doxorubicin (TRF-DOX). In April 2015, Panther entered into a definitive agreement with privately held Faulk Pharmaceuticals, Inc. to acquire Faulk's pharmaceutical technology assets which included TRF-DOX. The transaction provided Panther with a proprietary, multi-nationally patent protected, ligand-drug conjugate technology platform as well as a pipeline of drug product candidates that address unmet medical needs in oncology, autoimmune, antiviral and other disease indications. Panther was particularly interested in the TRF-DOX conjugate as it has shown promising safety and preliminary efficacy in a randomized controlled study of ovarian cancer outside the US.
Panther has executed an agreement with Dr. Patricia D. Williams of IND Directions, LLC in Washington DC to lead all aspects of the drug development process for TRF-DOX including regulatory, preclinical, manufacturing and clinical development activities. Dr. Williams has over 30 years of experience designing and implementing high quality drug development programs for small molecules, biologics, and gene therapies. Dr. Williams has held various positions of increasing responsibilities at major pharmaceutical and biotechnology companies (Bristol-Myers, Eli Lilly, American Cyanamid, Ligand, Biochem Pharma) as well as contract research and consulting organizations (SRA Life Sciences, TherImmune, Gene Logic and Summit Drug Development Services). In addition, Dr. Williams has already worked on TRF-DOX with Faulk Pharmaceuticals in the past. Teams under Dr. Williams' leadership have advanced over 50 small molecules and biologics into clinical development. Under Dr. Williams' leadership, Panther has initiated the preparation of a formal Pre-Investigational New Drug (IND) submission to the US Food and Drug Administration (FDA). This step will result in FDA's input into Panther's Phase 2 clinical plans with TRF-DOX as well as the preclinical and manufacturing data that will support clinical trials with TRF-DOX.

Read more: http://www.marketwired.com/press-release/panther-biotechnology-announces-initiation-formal-development-transferrin-doxorubicin-otc-pink-pbya-2142485.htm

Source: http://www.businesswire.com/
NEW HAVEN, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) announced today that researchers presented new data from an ongoing open-label, Phase 1/2 trial of intravenous (IV) SBC-103 (rhNAGLU enzyme), an investigational enzyme replacement therapy, in children with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo syndrome type B), a genetic, progressive, and devastating rare lysosomal storage disease. Preliminary evidence, based on brain scans (MRI) and neurocognitive assessments at 24 weeks, showed the potential for disease stabilization in patients with MPS IIIB treated with SBC-103. These data were presented at the 14th International Symposium on MPS and Related Diseases in Bonn, Germany.

MPS IIIB is caused by genetic mutations that result in a marked decrease in N-acetyl-α-D-glucosaminidase (NAGLU) enzyme activity, leading to abnormal accumulation of heparan sulfate (HS) in the brain and other organs, as well as progressive brain atrophy with cortical gray matter (CGM) volume loss. This results in severe neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. At present there is no treatment for this disorder. MPS IIIB typically presents in children during the first few years of life, and these children have a greater than 50 percent mortality rate by 17 years of age.

“MPS IIIB is a devastating and life-threatening disorder, with no available treatments, and has a severe and progressive impact on the cognitive function of children suffering with the disease,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “These new data presented today suggest the potential of SBC-103 to cross the blood-brain barrier when administered intravenously and provide preliminary evidence of potential dose-dependent disease stabilization at 24 weeks in children with MPS IIIB.”

Read more: http://www.businesswire.com/news/home/20160714005680/en/Alexion-Presents-SBC-103-rhNAGLU-enzyme-Phase-12

Source: http://www.prnewswire.com/

BOONTON, N.J., July 13, 2016 /PRNewswire/ -- Enteris BioPharma, Inc., a biotechnology company developing innovative drug products built around its proprietary delivery technologies, announced today the initiation of its clinical program for Tobrate™, an oral tablet formulation of tobramycin, for the treatment of uncomplicated urinary tract infections (uUTIs). Tobrate™ utilizes the Company's proprietary Peptelligence™ platform, a novel formulation technology that enables oral delivery of molecules that are typically injected, including peptides and BCS class II, III, and IV small molecules.

"Initiation of the Tobrate™ clinical program is a significant event for Enteris as we now have the opportunity to advance a potentially high-value and highly differentiated therapeutic for the treatment of uUTI, a condition that affects approximately 10 million U.S. women each year," said Joel Tune, chief executive officer and executive chairman of Enteris. "Moreover, Tobrate™ is an expansion of our internal drug pipeline, which includes Ovarest™, a Phase 2a ready oral peptide for endometriosis."

As detailed in the Investigational New Drug (IND) application, which was accepted by the U.S. Food and Drug Administration (FDA) in April, Tobrate™ will initially be evaluated in a randomized, double-blind, single ascending dose/multiple ascending dose (SAD/MAD) Phase 1 pharmacokinetic (PK) study in 24 healthy volunteers. Data from the Phase 1 study is expected in the first quarter of 2017.

Read more: http://www.prnewswire.com/news-releases/enteris-biopharma-initiates-clinical-program-for-tobrate-oral-tobramycin-tablet-in-uncomplicated-urinary-tract-infection-300297880.html

Source: https://globenewswire.com/

REDWOOD CITY, Calif., July 11, 2016 (GLOBE NEWSWIRE) -- Coherus BioSciences, Inc. (NASDAQ:CHRS), a leading global biosimilars company with late-stage clinical products, today reported topline results from its follow-on pharmacokinetic and pharmacodynamic (PK/PD) clinical study of CHS-1701, a pegfilgrastim (Neulasta®) biosimilar candidate. This study met all of its co-primary endpoints for PK, Cmax and Area Under the Curve (AUC), and PD, absolute neutrophil count, and ANC (ANCmax and ANC AUC). For both endpoints, the 90% Confidence Intervals (CI) for the Geometric Mean Ratio (GMR) were well contained within the pre-specified margins of 80% to 125%.
This randomized, single-blind, three-sequence, three-period crossover study in healthy subjects assessed PK, PD, and safety (including immunogenicity) of a 6 mg subcutaneous (SC) injection of CHS-1701 compared to 6 mg SC dose of Neulasta. A total of 122 healthy volunteer subjects were randomized to one of three treatment sequences, each with three treatments. Subject inclusion criteria, procedures and study design, as well as other measures, reflected modifications addressing findings in the previous study CHS-1701-03, successfully decreasing subject variability and eliminating the extreme subject outliers previously observed.

“As has been seen, pegfilgrastim is a relatively biologically complex molecule. Our novel and innovative study design allowed us to evaluate and successfully control for the contribution of subject variability to the PK/PD parameters, validating our hypothesis regarding the outliers seen in the earlier study. Based on the totality of the data, including this clinical result as well as those from the previous PK/PD study and the successful immunogenicity study, we are confident that CHS-1701 is highly biosimilar to the reference product, Neulasta, and will produce the expected clinical effect in patients,” said Barbara Finck, M.D., Chief Medical Officer of Coherus BioSciences.

Read more: https://globenewswire.com/news-release/2016/07/11/854984/0/en/Coherus-Announces-Positive-Topline-Results-for-CHS-1701-Pegfilgrastim-Biosimilar-Candidate-Pharmacokinetic-and-Pharmacodynamic-Biosimilarity-Study.html

Source: https://globenewswire.com/
NEW YORK and LONDON, July 06, 2016 (GLOBE NEWSWIRE) -- Akari Therapeutics (NASDAQ:AKTX), an emerging growth, clinical-stage biopharmaceutical company, today announced positive interim data from the first cohort in its Phase Ib trial which demonstrates maintenance of complement inhibition using once-daily dosing. The Phase Ib trial in normal healthy volunteers is designed to investigate the maintenance dose of subcutaneous Coversin needed to maintain complement inhibition on a once daily basis. The company believes near-complete complement inhibition is necessary to maintain adequate control in patients with certain conditions including Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS).

“Subcutaneous dosing with Coversin will represent an important advance for patients in complement therapy,” said Dr. Gur Roshwalb, Chief Executive Officer of Akari Therapeutics. “Patients and key opinion leaders have expressed to us their preference for a subcutaneous, self-administered therapy. We will use this dosing regimen in our upcoming Phase II PNH trial.”

In this double-blind, randomized Phase Ib trial, each cohort of six normal healthy volunteers is given either a loading dose of subcutaneous placebo twice a day for two days followed by five days of a single daily placebo dose (n=2) or, in this first cohort, a loading dose of 30 mg of subcutaneous Coversin twice a day for two days followed by five days of a single daily subcutaneous maintenance dose of 30mg (n=4). Data from this first cohort demonstrated that subcutaneous Coversin achieved complete complement inhibition (Elisa CH50 < 8 Eq/ml, lower limit of quantification) within the first day, and demonstrated complete complement inhibition at the end of dosing on day seven. To date, there have been no injection site reactions reported in the trial. One volunteer receiving the Coversin dose stopped dosing on day three due to a non-serious adverse event possibly related to antibiotics administered for meningitis prophylaxis. The trial is being conducted at Hammersmith Medicines Research Ltd, in London. It is expected that further data from this trial will be presented at future scientific forums. Read more: https://globenewswire.com/news-release/2016/07/06/853912/0/en/Akari-Therapeutics-Announces-Positive-Interim-Update-from-Phase-Ib-Trial-Demonstrating-Sustained-Complement-Inhibition-Using-Once-Daily-Subcutaneous-Maintenance-Dosing-with-Coversi.html

Source: http://news.genzyme.com/
CAMBRIDGE, Mass.--(BUSINESS WIRE) - Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first adult patient has enrolled and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B).

ASMD is one of a group of lysosomal storage disorders that affect cellular metabolism and are caused by genetic mutations. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase resulting in accumulation of sphingomyelin in multiple organs of the body. Common clinical manifestations include enlarged liver and spleen, liver dysfunction, infiltrative lung disease, bleeding complications, cardiovascular and bone disease, and growth delay. There are currently no approved treatment options for patients with ASMD.

ASCEND is a Phase 2/3 multi-national, multi-center, double-blinded, placebo-controlled trial to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in adult patients with ASMD, specifically NPD B. The Phase 2/3 trial will assess the effect of olipudase alfa on spleen size, lung function and other important clinical parameters. Thirty-six patients are expected to be enrolled in the study and receive olipudase alfa or a placebo. Upon completion of the 52 week primary analysis period, all patients will receive treatment in an extension period.

Read more: http://news.genzyme.com/press-release/sanofi-genzyme-begins-pivotal-phase-23-trial-olipudase-alfa-adult-patients-acid-sphing

Source: Shire plc & reported by http://www.prnewswire.com/
Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that its Phase 2 study evaluating an investigational protein replacement, SHP607, did not meet its primary endpoint of reducing the severity of retinopathy of prematurity (ROP), a rare eye condition. The study, however, demonstrated clinically relevant effects in secondary endpoints related to the development of severe bronchopulmonary dysplasia (BPD), a chronic lung disease, and severe intraventricular hemorrhage (IVH), a type of brain injury, both of which have lifelong negative implications for normal development.

SHP607 is a recombinant human version of the naturally-occurring protein complex of insulin-like growth factor 1 (IGF-1) and its most abundant binding protein, IGF binding protein-3 (IGFBP-3).

The Phase 2 study included 121 extremely premature infants (born between 23 weeks and 27 weeks +6 days) randomized at birth to either SHP607 or standard neonatal care, and treated continuously until an equivalent gestational age of 30 weeks. IGF-1 is a growth factor that plays a major role in the development of the growing fetus in the uterus. It is supplied by the mother until about 30 weeks of gestation when the fetus begins producing the growth factor on its own. Levels of IGF-1 dramatically decrease in infants born extremely premature (before 28 weeks of gestation), thereby increasing the risk for complications related to the lungs, brain, eyes, and other organs.

Read more: http://www.prnewswire.com/news-releases/shire-announces-top-line-results-for-phase-2-trial-of-shp607-in-extremely-premature-infants-585008131.html

Source: http://www.prnewswire.com/
OXFORD, England, June 28, 2016 /PRNewswire/ - Glide Technologies today announced results from a successful clinical proof-of-concept study that demonstrated its novel solid formulation of the most widely-used dose of octreotide (100 mcg) achieved bioequivalence to the currently marketed immediate release liquid injectable product (Sandostatin®). Glide's formulation was delivered using the company's proprietary needle-free Solid Dose Injector (SDI®), which provides the potential for room temperature stability and patient-friendly administration. Octreotide is approved for use in the treatment of acromegaly and certain neuroendocrine tumours, with the estimated market for immediate release liquid formulations exceeding $150 million.

The clinical study compared the tolerability, pharmacokinetics and bioavailability of 100 mcg of Sandostatin® delivered by needle and syringe with Glide's octreotide solid dose formulation delivered via SDI®. The results demonstrate that Glide's formulation achieved bioequivalence, matching Sandostatin® on both maximum peak concentration (Cmax) and area under the curve (AUC). In addition, the study subjects (n=20) confirmed the suitability of the SDI® for self-administration and reported an overwhelming preference for the Glide SDI® over needle and syringe. The Glide delivery system was well tolerated by the subjects who rated injection comfort as 4.45 on a scale of 0-5 (with 5 being most comfortable).

Read more: http://www.prnewswire.com/news-releases/glide-technologies-announces-successful-clinical-proof-of-concept-study-with-its-novel-octreotide-solid-dose-formulation-delivered-via-sdi-584642901.html