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Source: http://www.businesswire.com/
SAN DIEGO--(BUSINESS WIRE)--La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today reported positive results from its Phase 1 study of LJPC-401 in patients at risk of iron overload.
The Phase 1 study is a multi-center, open-label, dose-escalation study evaluating the safety, tolerability, and effect on serum iron of a single dose of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), thalassemia, and sickle cell disease (SCD). Fifteen patients were dosed at escalating dose levels ranging from 1 mg to 20 mg.

LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved. There were no significant changes in serum chemistries or hematology other than serum iron parameters.

A dose-dependent, statistically significant reduction in serum iron was observed (p=0.008 for dose response; not adjusted for multiple comparisons). At the 20 mg dose level, LJPC-401 reduced serum iron by an average of 58.1% from baseline to hour 8 (p=0.001; not adjusted for potential regression to the mean effect), and serum iron had not returned to baseline through day 7 (21.2% reduction from baseline to the end of day 7).

Read more: http://www.businesswire.com/news/home/20160907006780/en/La-Jolla-Pharmaceutical-Company-Reports-Positive-Results

Source: BioMarin Pharmaceutical Inc. & reported by http://www.nasdaq.com/press-release/

SAN RAFAEL, Calif., Sept. 06, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced a program update for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease. CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments, where the majority of affected children lose their ability to walk and talk by approximately six years of age. After 81 weeks, patients treated with cerliponase alfa continue to have motor-language (ML) scores representing substantial attenuation of disease progression compared to natural history. These data are consistent with the data at 48 weeks submitted with the original Biologics License Application (BLA), and demonstrate durable and consistent treatment response. During their initial review of the BLA, the U.S. Food and Drug Administration (FDA) requested an updated efficacy data cut from the ongoing extension study, which the company provided. The FDA designated this submission as a major amendment to the application, thus extending the PDUFA action date by three months to April 27, 2017.

Read more: http://www.nasdaq.com/press-release/biomarin-announces-update-to-brineura-cerliponase-alfa-program--for-treatment-of-cln2-disease-a-20160906-01176#ixzz4KXHZUbGD

Source: Soligenix, Inc. & reported by http://www.prnewswire.com/
Princeton, N. J., Sept. 6, 2016 /PRNewswire/ -- Soligenix, Inc. (OTCQB: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the United States (US) Patent Office granted the patent entitled "Peptides for Treating and Preventing Immune-Related Disorders, Including Treating and Preventing Infection by Modulating Innate Immunity." The newly issued patent claims composition of matter of analogs of dusquetide (research name: SGX94), the Company's lead development compound. Dusquetide recently demonstrated positive preliminary results in a Phase 2 clinical trial for the treatment of oral mucositis in head and neck cancer patients. The recently issued patent broadens the protection around dusquetide and provides further protection for the underlying innate defense regulator (IDR) technology platform. Similar claims have been granted and/or are being pursued in jurisdictions worldwide.

IDRs are first-in-class small peptides with a novel mechanism of action. IDRs function by modulating the response of the innate immune system at a key convergence point in the intracellular signaling pathways. The innate immune system is a non-specific system which responds to a variety of insults, including infections and tissue damage. Because IDRs act by modulating the response of the innate immune system, they are agnostic as to either the cause of tissue damage (e.g., chemotherapy, radiation or trauma) or infection (e.g., antibiotic sensitive or antibiotic resistant infections). A summary of the IDR technology can be found in the following audio presentation entitled, "Dusquetide: Innate Defense Regulation as a Novel Approach to Antibiotic-Resistant Infection": https://www.youtube.com/watch?v=ef0-wQod-2E&feature=youtu.be

Read more: http://www.prnewswire.com/news-releases/soligenix-announces-issuance-of-new-composition-of-matter-patent-for-dusquetide-analogs-300321894.html

Source: Dipexium Pharmaceuticals, Inc. & reported by http://www.prnewswire.com/
NEW YORK, Sept. 7, 2016 /PRNewswire/ -- Dipexium Pharmaceuticals, Inc. (Nasdaq: DPRX), a late-stage pharmaceutical company focused on the development and commercialization of Locilex® (pexiganan cream 0.8%), a novel, broad-spectrum, topical antimicrobial peptide, today announced that the European Patent Office has issued a new Locilex patent in the European Union (EU). The patent claims are directed to a novel formulation of Locilex, a topical antimicrobial peptide formulated as a topical cream, and the use of Locilex as a method of treating skin or wound infections. The issued patent has an expiry date in June 2033.

"This constitutes the first patent granted by the European Union, the second largest pharmaceuticals market in the world," stated David P. Luci, President & Chief Executive Officer of Dipexium, "this is an important building block for our commercialization strategy for Locilex in Europe." Mr. Luci continued, "Dipexium now has issued patents for Locilex in the US, EU, and Japan, the three largest pharmaceuticals markets in the world, as well as Australia and New Zealand. In addition, Dipexium has obtained notice of allowance for patents in Hong Kong and Korea. We anticipate the issuance of additional patents for Locilex in other major international markets throughout 2016 and 2017."

Read more: http://www.prnewswire.com/news-releases/dipexium-pharmaceuticals-announces-issuance-of-locilex-patent-by-european-union-300323725.html

Source: http://www.marketwired.com/
SAN DIEGO, CA--(Marketwired - August 30, 2016) - Aegis Therapeutics LLC announced today that it has been awarded Hong Kong Patent No. HK1164059 providing fast acting formulations for triptans, a class of drugs that are effective in treating migraine headaches which include sumatriptan, zolmitriptan, naratriptan, rizatriptan, eletriptan, almotriptan and frovatriptan. Human clinical trials have shown that the Intravail® formulation of sumatriptan achieves therapeutic drug levels at approximately 2 to 3 minutes, 20 to 30 times faster than the currently marketed non-injectable sumatriptan products. For comparison, the most widely used triptan formulations, namely sumatriptan nasal spray or tablet formulations, both reach maximum blood levels of drug in about 60 to 120 minutes thus delaying onset of relief.
The enabling Aegis Intravail formulation technology is broadly applicable to a wide range of small molecule and biotherapeutic drugs to increase noninvasive bioavailability by the oral, nasal, buccal, and sublingual routes and to speed attainment of therapeutic drug levels in cases where speed is important to the patient, for example in the treatment of pain, nausea, emesis, convulsive disorders, spasticity, and the like.
Aegis technology is commercialized by our licensees who conduct the actual product development and marketing activities. Aegis' licensees include seven of the top 10 largest pharmaceutical companies and two of the top 10 largest multinational generics companies, along with many public and private biotech companies. Aegis growing patent portfolio currently has more than seventy-five issued and pending drug formulation patents covering noninvasive delivery and stabilization of biotherapeutics, biosimilars, and small-molecule drugs.
Read more: http://www.marketwired.com/press-release/aegis-awarded-9th-patent-for-fast-acting-migraine-nasal-spray-treatment-2154203.htm

Source: http://www.businesswire.com/
ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris: ERYP), the French biopharmaceutical company developing ‘tumor starvation’ treatments for acute leukemia and other oncology indications with unmet medical needs, today announced that it has reached full patient enrollment in the Phase 2b trial of eryaspase, also known as ERY-ASP or GRASPA®, for the treatment of acute myeloid leukemia (AML).

The open-label, randomized, multi-center clinical trial, which is being conducted at more than 20 sites in Europe, has completed enrollment of a total of 123 patients and is on track for reporting of primary data in the second half of 2017. Patients enrolled in the trial are over the age of 65, newly-diagnosed with AML, and unable to receive intensive chemotherapy. The primary endpoint is overall survival (OS) at one year.

“We are pleased to have reached this important clinical milestone of complete enrollment in our trial for AML and expect to reporting primary data from the trial in the second half of 2017,” said Gil Beyen, Chairman and CEO of ERYTECH. “AML is a very aggressive cancer. We are developing eryaspase with the goal of contributing to the treatment of these patients, many of whom may respond to L-asparaginase, but have difficulty with the side effects associated with the current available forms. Therefore, we believe the increased tolerability profile obtained through the encapsulation of L-asparaginase in the red blood cells could result in a new innovative approach to treatment of AML patients.”
Read more: http://www.businesswire.com/news/home/20160828005031/en/ERYTECH-Completes-Patient-Enrollment-Phase-2b-Trial