Monday, December 17, 2018
Avrobio treatment of Fabry Disease granted FDA orphan designation
The FDA granted orphan designation to Avrobio’s treatment of Fabry Disease, according to a post to the agency’s website.
https://thefly.com/landingPageNews.php?id=2838165
Innovate to Start Phase 3 Celiac Trial in 1H 2019, Announce Top-line NASH Data
Preliminary top-line data from the alcoholic steatohepatitis (ASH) collaboration with Massachusetts General Hospital shows positive effect of larazotide on ethanol-induced permeability in vitro
Innovate Biopharmaceuticals Inc. (Nasdaq: INNT), a clinical stage biotechnology company focused on developing novel medicines for autoimmune and inflammatory diseases, is evaluating various funding proposals for commencing the first ever Phase 3 registration trial for celiac disease. Celiac disease affects approximately 1% of the U.S. population, more than 3 million Americans, and is a high unmet need with no FDA approved treatments available.
Christopher Prior, Ph.D., CEO of Innovate, stated, “We believe that during 2018 we have laid the foundation for an exciting 2019 by completing much of the preparatory work to launch our Phase 3 trial for celiac disease and to expand our pipeline in areas of liver disease.” Dr Prior added, “We are continuing our efforts to secure financing for this trial on favorable terms with the goal of benefiting our stockholders and bringing important medicines to patients where there are no currently available therapies.”
As previously presented, larazotide demonstrated decreased deterioration of gut permeability, in the DIAMOND NASH mouse model, where leaky gut developed by 16 weeks. With ongoing work in drug combinations with Novo Nordisk A/S’s OZEMPIC®(semaglutide) approved for type 2 diabetes and Intercept Pharmaceuticals Inc.’s OCALIVA® (obeticholic acid) approved for primary biliary cholangitis (PBC), Innovate expects to release data by EASL 2019.
Through its recent collaboration with Massachusetts General Hospital (MGH) on alcoholic steatohepatitis (ASH), Innovate is announcing preliminary top-line data showing positive effects by larazotide on ethanol-induced permeability in vitro. The MGH model shows larazotide markedly protects and rescues cells from increased ethanol-induced permeability, a known pathologic effect in ASH. Innovate plans to present further details, including transepithelial electric resistance (TEER), and timing of these ethanol induced permeability effects, at an upcoming medical meeting in 2019. Alcoholic liver disease (ALD), including acute-on-chronic forms, such as alcoholic hepatitis, has been shown to lead to hospitalizations (1% of hospital admissions in 2010)1 and high mortality rates (20–30% at 1 month and 40–70% at 6 months).2
Peter Traber, M.D., an experienced drug developer in NASH and GI, said, “The fact that larazotide is capable of restoring permeability following three different insults, namely gluten, high fat diet and alcohol relevant to celiac disease, NASH and ASH, respectively, underscores the potential broad application for this unique mechanism of action.”
Zynerba: Clinical Update; Announces Two New Clinical Indications
– Company Will Focus on Four Childhood Neuropsychiatric Disorders: FXS, DEE, Autism Spectrum Disorder and 22q11.2 Deletion Syndrome –
– Cash Runway Now Extends into the Second Half of 2020 –
– Conference Call to be Held Today at 8:30 am Eastern Time –
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided an update on its clinical progress.
- Zynerba remains on track to report top line results from the CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) study in Fragile X Syndrome in the second half of 2019;
- The Company has completed enrollment in its Phase 2 BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) clinical trial in children and adolescents with developmental and epileptic encephalopathies (DEE). Top-line results will be reported in the third quarter of 2019;
- Zynerba has expanded its pipeline with the addition of two new childhood neuropsychiatric clinical targets for ZYN002: Autism Spectrum Disorder (ASD) and 22q11.2 Deletion Syndrome (22q Deletion Syndrome; 22q), a rare genetic syndrome leading to significant impairments, including neuropsychiatric disorders. The Company expects to initiate open label Phase 2 studies in these indications in the first half of 2019 and report top-line results in first half 2020;
- The Company has postponed the initiation of its clinical trial in adult epilepsy until after the completion of the four childhood neuropsychiatric studies; and
- As a result, Zynerba expects to extend its cash runway into the second half of 2020.
“Our aspirations and expectations are clear: To work closely with the U.S. Food and Drug Administration to expand the opportunity for pharmaceutically-developed CBD treatments that meet their rigorous medical and manufacturing standards, and in doing so, continue toward our goal of addressing significant unmet medical needs in neuropsychiatric disorders,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “With the new indications of ASD and 22q, in addition to FXS and DEE, we now have four shots on goal to show the clinical benefit of ZYN002 transdermal gel in patient populations that have few, if any, therapeutic options available.”
Conference call information
Zynerba management will host a live conference call and webcast today at 8:30 am Eastern Standard Time to provide a corporate update. The call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345 (international) and referencing conference ID 6892856. To access the live webcast or the replay, visit the investor page of the Company’s website at http://ir.zynerba.com/. The webcast will be recorded and available on the Company’s website for 30 days.
Zynerba management will host a live conference call and webcast today at 8:30 am Eastern Standard Time to provide a corporate update. The call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345 (international) and referencing conference ID 6892856. To access the live webcast or the replay, visit the investor page of the Company’s website at http://ir.zynerba.com/. The webcast will be recorded and available on the Company’s website for 30 days.
Sanofi Meets with FDA Panel on Type 1 Diabetes Med
TIME: 8 a.m.
EVENT: Health and Human Services Department (HHS); Food and Drug Administration(FDA) (F.R. Page 61642) holds a meeting of the Endocrinologic and Metabolic Drugs Advisory Committee to discuss new drug application 210934 for sotagliflozin oral tablet, sponsored by Sanofi-Aventis U.S. LLC, for the proposed indication: Adjunct to insulin therapy to improve glycemic control in adults with type 1 (T1) diabetes mellitus.
DATE: January 17, 2019
LOCATION: FDA White Oak Campus, 10903 New Hampshire Avenue, Building 31 Conference Center, Room 1503, Silver Spring, Md.
CONTACT: LaToya Bonner, 301-796-9001, EMDAC@fda.hhs.gov
UnitedHealth: will pay $500M less for doctor group
In hopes of winning regulatory approval for a deal that was first announced a year ago, the purchase price in UnitedHealth Group’s proposed acquisition of DaVita Medical Group has shrunk by more than $500 million, according to a regulatory filing Monday.
The deal, which would significantly expand the push by Minnetonka-based UnitedHealth into the clinic business, is expected to close during the first quarter of 2019, according to a filing from Colorado-based DaVita Inc. that also cites “underlying business performance” in explaining the new price.
Last year, UnitedHealth Group agreed to purchase DaVita Medical Group for $4.9 billion, but the filing lists a new purchase price of $4.34 billion.
“As a result of underlying business performance and in an effort to expedite the process to obtain [Federal Trade Commission] approval of the proposed transaction, the parties agreed to amend the purchase agreement,” DaVita said in the regulatory filing. “The parties are working together to close the proposed transaction as expeditiously as possible and expect to close in the first quarter of 2019.”
Merit Medical acquisition ‘another prudent solid tuck-in,’ says Piper Jaffray
Piper Jaffray analyst Matt O’Brien views Merit Medical Systems’ acquisition of Vascular Insights as “another prudent solid tuck-in” for the company. Vascular’s assets, used for the treatment of varicose veins, complement an “already substantial” vascular portfolio and should allows Merit’s “large global salesforce” to address this $700M global market, O’Brien tells investors in a research note. He reiterates an Overweight rating on the shares with a $75 price target.
https://thefly.com/landingPageNews.php?id=2838099
Piper says Sophiris data ‘as expected,’ remains buyer of shares
Piper Jaffray analyst Christopher Raymond keeps an Overweight rating on Sophiris Bio with a $7 price target following this morning’s update from the Phase 2b localized prostate cancer trial. As expected, Sophiris reported six-month biopsy data for patients receiving a second administration of topsalysin, but no further clinically significant changes to prostate lesions were observed, Raymond tells investors in a research note. However, the analyst thinks the Phase 3 trial design and regulatory feedback remain key catalysts for the stock given the “compelling” six-month data from the single administration of topsalysin. He remains a buyer of Sophiris Bio shares, which are down 42% in late morning trading to $1.20.
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