Monday, October 8, 2018
Scientists use CRISPR to cure mice disorder in womb, STAT reports
Scientists reported today that they used CRISPR to alter the DNA of laboratory mice in the womb, eliminating a deadly liver disease before the animals had even been born, Sharon Begley of STAT reports. CRISPRing human fetuses is still years away, but the success in mice paves the way for curing genetic diseases before birth, the writer adds. Companies developing CRISPR-based therapies include Editas Medicine (EDIT), Intellia Therapeutics (NTLA), and Crispr Therapeutics (CRSP).
https://thefly.com/landingPageNews.php?id=2801091
Tetraphase Presents Positive Data from Phase 3 Anti-infective Trials
XERAVA Demonstrated Efficacy in Treating Secondary Bacteremia in Patients with Complicated Intra-Abdominal Infections in Post-Hoc Analysis of Phase 3 Clinical Trials
TP-6076 Demonstrated Favorable Pharmacokinetic and Safety Profile in Phase 1 Trial, Supporting Advancement into a Bronchopulmonary Disposition Study
Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, announced data from a pooled analysis of two Phase 3 studies of XERAVA in complicated intra-abdominal infection (cIAI) and a Phase 1 multiple-ascending dose study for its pipeline candidate TP-6076. These data were presented at the Infectious Disease Society of America’s (IDSA) Infectious Disease Week (IDWeek) 2018, held October 3-7 in San Francisco.
“The data presented at IDWeek demonstrate high clinical cure rates and microbiological eradication with XERAVA among patients with cIAI and concurrent bacteremia,” said Guy Macdonald, President and Chief Executive Officer of Tetraphase. “These data confirm the efficacy of XERAVA in a subgroup of cIAI patients who may be at higher risk for poor outcomes, and this new analysis comes at an exciting time as we make final preparations for the commercial launch of XERAVA in the coming weeks.”
Mr. Macdonald added, “We are also encouraged by the positive safety, tolerability and pharmacokinetic data from the multiple-ascending dose Phase 1 study evaluating a seven-day dosing regimen for intravenous (IV) TP-6076. We plan to advance TP-6076 to a bronchopulmonary disposition study beginning in the first quarter of 2019 to confirm appropriate therapeutic levels of TP-6076 in the lungs to treat infections caused by Acinetobacter baumannii and other MDR pathogens. In previously completed in vitrotesting, TP-6076 MIC90 values were as much as 64-fold lower than those for tigecycline against MDR Gram-negative pathogens, including Acinetobacter baumannii, suggesting that TP-6076 could be a potent treatment for these difficult-to-treat bacteria. We are enthusiastic about the potential for TP-6076 and the future of this program.”
FDA Panel to Review of Mallinckrodt Abuse-Deterrent Oxycodone Formulation
Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) has confirmed it will hold a joint meeting of its Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee on Nov. 14, 2018.
The Committees will assess the 505(b)2 new drug application submission for MNK-812, Mallinckrodt’s abuse-deterrent formulation of immediate-release, single-entity oxycodone tablets with a proposed indication for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. Mallinckrodt is advancing this reformulation of Roxicodone®(oxycodone hydrochloride tablets USP) with properties designed to deter intravenous and intranasal abuse.
“We look forward to engaging with the Advisory Committees to discuss the potential benefits our new abuse-deterrent formulation technology can bring in helping to mitigate opioid abuse and misuse,” said Matt Harbaugh, Executive Vice President, Chief Financial Officer and President, Specialty Generics for Mallinckrodt.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Nov. 16, 2018, to complete its review of Mallinckrodt’sproduct.
Ampio Ampion update
Ampio Pharmaceuticals, Inc. (NYSE MKT: AMPE) today provided an update of the ongoing Company activities required to file a Biologics License Application (BLA) for Ampion for the treatment of severe Osteoarthritis of the knee (OAK).
Michael Macaluso, Ampio’s CEO, noted that ‘During the time that AP-003-C, the second of our two required pivotal phase III clinical trials, was designed and executed, the FDA underwent a major reorganization. As a consequence, Ampio was overseen by three different review groups representing two different review offices within the Center for Biologics Evaluation Research (CBER). Currently, Ampion is being reviewed by CBER’s newly formed Office of Tissue and Advanced Therapies (OTAT).’
The company is currently focused on completing the following regulatory requirements established by the FDA
Perform two pivotal clinical trials of Ampion that CBER accepts as adequate and well-controlled studies that demonstrate the safety and efficacy of Ampion. These two pivotal trials are not required to be identical but must meet pre-determined endpoints and provide substantial replication of results.
All Ampion clinical studies must provide evidence in support of a BLA.
FDA must accept the Chemical and Manufacturing Controls (CMC) program for the investigational product.
Ampio Corporate Update
The Company is reporting the following milestones in support of the three objectives outlined above: On August 16, 2018, OTAT reaffirmed that ‘FDA considers AP-003-A to be an adequate and well-controlled clinical trial that provides evidence of the effectiveness of Ampion. AP-003-A can contribute to the substantial evidence of effectiveness necessary for the approval of a BLA.’ Over 2,000 patients now support the substantial evidence necessary for the BLA.
The AP-003-C OLE study was designed to support the safety and tolerability of repeat injections of Ampion (up to five injections) over the course of at least one year. No serious drug-related adverse events have been reported, and all patients demonstrated a sustained reduction in pain over the course of the trial.
On September 27, 2018, OTAT provided a favorable response, with no areas of dispute, in support of CMC for the Ampion BLA. Agreement was obtained ‘on the CMC data requirements for BLA and on the data presented for analytical characterization, commercial release specifications, and potency of Ampion drug product.’
The Company is continuing discussions with the FDA to determine if the pivotal AP-003-C study is the final trial required to support the Ampion BLA. Should another trial be required, the Company will move quickly to meet the FDA requirements under a Special Protocol Assessment (SPA).
Biotech week ahead, Oct. 8
As we step into the final quarter of 2018, biotech stocks have recorded double-digit gains for the year-to-date period.
Will the run up continue? Stay tuned to the following events of the upcoming week.
Conferences
- 10th Annual Conference on Stem Cell and Regenerative Medicine – Oct. 8-9, in Zurich, Switzerland
- 9th International Summit on Clinical Microbiology – Oct. 8-9, in Zurich
- 3rd International Conference and Expo on Optometry & Vision Science – Oct. 8-9, in Edinburgh, Scotland
- 2nd International Conference on Craniofacial Surgery – Oct. 8-9, in London
- 3rd European Otolaryngology-ENT Surgery Conference – Oct. 8-10, in London
- 3rd World Kidney Congress – Oct. 08-10, in Dubai, UAE
- 5th International Conference on Gynecology and Obstetrics – Oct. 8-10, in Hilton Zurich Airport Zurich
- The European Committee for Treatment and Research In Multiple Sclerosis, or ECTRIMS, 2018 Congress – Oct. 10-12, in Berlin, Germany.
- 21st European Biotechnology Congress – Oct. 11-12, in Moscow, Russia
- 36th World Cancer Conference – Oct. 11-13, in Zurich
Adcom Meeting Schedule
The Oncologic Drugs Advisory Committee will meet Oct. 10 to discuss Celltrion’s BLA for CT-P10, a proposed biosimilar to Roche Holdings AG Basel ADR RHHBY 0.44% unit Genentech, Inc.’s Rituxan, for treating CD20-positive, B-cell Non–Hodgkin’s Lymphoma.
The Anesthetic and Analgesic Drug Products Advisory Committee of the FDA will discuss Oct. 11 Trevena Inc TRVN 4.08%‘s NDA for oliceridine 1 milligram/milliliter injection for the management of moderate-to-severe acute pain in adult patients for whom an intravenous opioid is warranted. The committee will evaluate safety, efficacy as well as benefit-risk considerations. Trevena shares have been trending up in recent sessions.
The Anesthetic and Analgesic Drug Products Advisory Committee will also review AcelRx Pharmaceuticals Inc ACRX 8.14%‘s NDA for sufentanil sublingual tablets for treating moderate-to-severe acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. The committee will look at risk-benefit considerations and also recommend approvability of the candidate. The Adcom meeting is scheduled for Oct. 12.
AcelRx’s NDA was handed down a CRL in October 2017 following which a resubmission was made, with the new PDUFA date set for Nov. 3.
Clinical Trial Results
TG Therapeutics Inc TGTX 4.43% is scheduled to present final Phase 2a data for TG-1101, its treatment candidate for relapsing form of multiple sclerosis, at the ECTRIMS Congress on Oct. 11.
Pending Releases From Q3
Acorda Therapeutics Inc ACOR 2.01% is due to release Phase 2 data for its primary sclerosing cholangitis treatment option BTT1023.
GALAPAGOS NV/S ADR GLPG 3.72% is due to release Phase 2 data for its combo treatment GLPG 2451+2222+2737 for cystic fibrosis in homozygous F508del patients.
Novo Nordisk A/S (ADR) NVO 2.34% will release Phase 2 data for its Concizumab that is being evaluated as a treatment option for hemophilia A.
Cytokinetics, Inc. CYTK 4.42% will release interim analysis of Phase 1b data for its CK-2127107 for limited mobility. The company will also release results of a Phase 2 study of the same candidate for treating COPD.
October Releases (with no specified date)
Entera Bio Ltd NASDAQENTX is due to release Phase 2 pharmacokinetic/pharmacodynamic data for EB612 that is being evaluated for hypoparathyroidism.
Esperion Therapeutics Inc ESPR will release Phase 3 data for its hypercholesterolemia treatment bempedoic acid.
Soligenix, Inc. SNGX 0.49% is set to release interim analysis of Phase 3 data for SGX301, its treatment candidate for cutaneous T-cell lymphoma.
Auris Medical Holding AG EARS 53.19% is scheduled to release Phase 1 data for its AM-125, which is being studied for vertigo.
IPOs
Allogene Therapeutics, which develops CAR T cell cancer therapies, is set to offer 16 million shares in an IPO, with the shares priced between $16 and $18. The company seeks to list the shares on the Nasdaq under the ticker symbol ALLO.
Equillium, a developer of monoclonal antibody therapies for aGVHD and cGVHD, will offer 4.67 million shares, with an estimated price range of $14-$16. The shares are to be listed on the Nasdaq under the ticker symbol EQ.
Osmotica Pharma, which develops extended-release neurology and women’s health drugs, is due to offer 8.3 million shares, priced between $14 and $16. The shares will be listed on the Nasdaq under the ticker symbol OSMT.
IPO Quiet Period Expiry
Principia Biopharma Inc PRNB 3.54%
DaVita Inpatient Hospital Services Plan Reaccredited for Ambulatory Care
DaVita Kidney Care, a division of DaVita Inc. (NYSE: DVA), a leading independent provider of integrated health and kidney care services in the United States, today announced DaVita Hospital Services has received its second reaccreditation for Ambulatory Health Care from The Joint Commission.
“Our hospital partners ask us to bring our operations excellence and demonstrated quality of patient care to the inpatient care setting, and we’re thrilled to be recognized for delivering on that promise,” said Tina Livaudais, Vice President, Clinical Services for DaVita Hospital Services Group. “Our care team is one of the best in kidney care, and I’m pleased to have the chance today to acknowledge their enormous contributions to our reaccreditation.”
The reaccreditation, effective 2018, is a symbol of quality earned by DaVita Hospital Services for meeting or exceeding performance standards set by The Joint Commission. Reaccreditation focuses on enhancing the quality and safety of patient care through improved clinical outcomes and performance metrics, risk management and survey preparedness.
DaVita was the first inpatient dialysis provider to be accredited by The Joint Commission in 2013. The Joint Commission collaborated to outline national industry standards by which safety and quality of inpatient kidney disease and apheresis therapies would be measured in the future. Having set standards allowed DaVita to better measure performance and increase alignment with its hospital partners.
“Joint Commission accreditation provides ambulatory care organizations with the processes contributing to improvements in a variety of areas from the enhancement of staff education to the demonstration of leading practices within the ambulatory setting,” said Pearl Darling, MBA, Executive Director, Ambulatory Care Services, The Joint Commission. “We commend DaVita and its staff for achieving this pinnacle demonstrating a commitment to patient safety and quality. Your passion, dedication and tenacity can ultimately improve patient care. Thank you for your commitment to patient safety and entrusting The Joint Commission to assist you.”
Citron positive on Pyxus International
Shares of Pyxus International (PYX +30.8%) extend on their earlier gains after Citron Research weighs in on the debate.
“$PYX would not short…speculative long. Real management and tight float. The stock could double from here as long as investors are Cannabis crazy,” tweets Citron.
Previously: Pyxus International rallies to new high (Oct. 8)
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