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Monday, April 8, 2019

Translate Bio Presents Preclinical Data at Society for Inherited Metabolic Disorders

— MRT5201 was efficiently delivered to the liver of a mouse model of OTC deficiency —
— Preclinical data demonstrated a single IV dose of MRT5201 was sufficient to protect against hyperammonemia for up to 4 weeks —
 Translate Bio, Inc. (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced the presentation of preclinical data for MRT5201, an mRNA therapeutic designed to treat patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder. The poster presentation, entitled “Treatment of Ornithine Transcarbamylase Deficiency with a Targeted mRNA Therapeutic (MRT),” includes data demonstrating that a functional OTC mRNA encapsulated in a lipid nanoparticle (LNP) was successfully delivered to the liver in a preclinical mouse model of OTC deficiency via intravenous (IV) administration. These data were featured in a poster presentation at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 7, 2019 in Bellevue, Washington.
“MRT5201 is designed to treat patients with OTC deficiency by intravenous delivery of mRNA encoding fully functional OTC enzyme to the liver to enable hepatocytes to produce the normal OTC enzyme,” said Dr. Ann Barbier, chief medical officer of Translate Bio. “These data demonstrate MRT5201’s ability to prevent hyperammonemia in a relevant disease model and support further study of MRT5201 as a possible treatment for OTC deficiency.”

Revance to Present Trial Results at Aesthetics & Anti-Aging Conference

– Unprecedented clinical results for DaxibotulinumtoxinA for Injection (DAXI), Revance’s long-acting neuromodulator, were highlighted at the 17th Aesthetics & Anti-Aging Medical World Congress in Monte-Carlo –
Revance Therapeutics, Inc. (Nasdaq: RVNC), a biotechnology company developing next-generation neuromodulators for use in treating aesthetic and therapeutic conditions, today announced podium and poster presentations of its SAKURA 3 Phase 3 open-label, long-term safety study of DaxibotulinumtoxinA for Injection (DAXI) for the treatment of glabellar (frown) lines at the 17th Aesthetics & Anti-Aging Medical World Congress (AMWC). The leading international conference was held April 4 – 6 in Monte-Carlo, Monaco. SAKURA 3 was the culmination of the largest aesthetic clinical program of an aesthetic neuromodulator, which consistently demonstrated unprecedented efficacy, safety and duration across 65 sites and 3,800 treatments. The SAKURA 3 trial was specifically designed to evaluate the long-term safety of DAXI following both single and repeat treatment administration in adults 18 years of age and over.
“I was excited to present the SAKURA 3 study data on DAXI at the largest aesthetics meeting in the world, with more than 2,000 people in attendance,” said cosmetic dermatologic surgeon Sabrina Guillen-Fabi, MD, of Cosmetic Laser Dermatology in San Diego, Calif. “Colleagues were impressed with the consistent and predictable response rates, the safety profile and, most importantly, the duration of effect.”

Kiniksa to present preclinical KPL-404 data at Keystone Symposia

Kiniksa announced that it will present preclinical data for KPL-404, a monoclonal antibody inhibitor of the CD40 co-stimulatory receptor, at the Keystone Symposia.

Bio-Rad Labs (BIO) Receives U.S. FDA Clearance for IH-500

Bio-Rad Laboratories, Inc. (NYSE: BIO), a global leader of life science research and clinical diagnostic products, today announced that it has received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for Bio-Rad’s IH-500, an automated random access system for blood typing and screening.
The IH-500 system was designed for small- to medium-size laboratories, offering automated blood typing and screening based on gel card technology with minimal user intervention. The system can process Bio-Rad’s entire range of IH gel cards for ABO blood grouping, reverse testing, phenotype, Rh-subgroups, antibody screening, antibody identification, single antigen testing, direct AHG testing (DAT), and crossmatch, to deliver safe and accurate results.
“We are pleased to receive FDA clearance for our IH-500 and look forward to extending our reach in the U.S. transfusion medicine market,” said John Hertia, Bio-Rad Executive Vice President and President, Clinical Diagnostics Group. “This addition rounds out our offering, specifically benefitting mid and smaller volume laboratories using gel technology to test blood in an automated way,” he said.

Zogenix hit after FDA rejects filing

Zogenix Inc. ZGNX, -0.29% shares dropped in the extended session Monday after announcing that the Food and Drug Administration did not accept a marketing application for one of its drugs. Following a brief halt, Zogenix shares fell 25% after hours, following a 0.3% decline to close at $51.85 in the regular session. The developer of rare-disease therapies said the FDA rejected a marketing application for its drug Fintelpa to treat seizures from Dravet syndrome because they were “not sufficiently complete to permit a substantive review.” Zogenix said it would seek immediate guidance from the FDA to respond to issues raised in the rejection.

FDA OKs ViiV Healthcare’s two-drug HIV combo

The FDA approves Viiv Healthcare’s Dovato (dolutegravir and lamivudine) for the treatment of HIV-1 infection in adults who have not received prior antiretroviral treatment, the first fixed-dose two-drug combo regimen available in the U.S. for these patients.
ViiV Healthcare is the HIV-focused joint venture between GlaxoSmithKline (GSK +0.2%), Pfizer (PFE +0.2%) and Shionogi (OTCPK:SGIOY -0.4%).

Apple healthcare opportunity seen growing to as much as $300B+ in decade

A Morgan Stanley analyst sees healthcare as a $15B-$313B revenue opportunity for Apple by 2027
Alexa is moving into healthcare, with Amazon (AMZN) announcing a program allowing select developers to create and launch HIPAA-compliant healthcare skills for the company’s voice assistant. Amazon was one of the technology companies identified by participants in a recent Morgan Stanley survey as potentially having the largest impact on healthcare over the next five years, alongside Google (GOOGL; GOOG) and Apple (APPL). However, analyst Katy Huberty argued that participants may be underestimating the iPhone maker as she believes Apple is building a healthcare ecosystem and is “poised to emerge as a leader in consumer-centric healthcare.”
ALEXA TO TRACK CONSUMER HEALTHCARE: Over the weekend, The Wall Street Journal’s Melanie Evans reported that Amazon is positioning its AI assistant Alexa to track users’ prescriptions and relay personal health information in an effort to involve the technology in everyday healthcare. Five companies including Cigna (CI), Livongo Health and some major hospital systems have developed Alexa features for consumers using federal protocol, the publication added. This follows Amazon’s announcement last week that “the Alexa Skills Kit now enables select Covered Entities and their Business Associates, subject to the U.S. Health Insurance Portability and Accountability Act of 1996, HIPAA, to build Alexa skills that transmit and receive protected health information as part of an invite-only program. Six new Alexa healthcare skills from industry-leading healthcare providers, payors, pharmacy benefit managers, and digital health coaching companies are now operating in our HIPAA-eligible environment. In the future, we expect to enable additional developers to take advantage of this capability.”
APPLE OPPORTUNITY UNDERAPPRECIATED: Apple is building a healthcare ecosystem and is “poised to emerge as a leader in consumer-centric healthcare,” Morgan Stanley’s Huberty told investors in a research note on Monday. The analyst pointed out that the center of gravity is shifting in U.S. healthcare, as wearables, electronic medical records, and proposed regulations are enabling data to flow more freely between silos and stakeholders. Huberty sees healthcare as a large, greenfield services opportunity for Apple, which has the potential to lead digital disruption, much like what iTunes did for music or the App Store did for mobile services. The company’s healthcare strategy appears to aim for an ecosystem that puts the consumer at the center, she contended, noting that the Apple Watch has evolved into a “health monitoring guardian and guru.”
Further, Huberty argued that armed with the Apple Watch and the iPhone, Apple is emerging as “an agent of change” in healthcare and a “leader among tech companies entering the space.” While the analyst sees room for contribution from many technology companies in reducing the estimated $1.2T of wasted U.S. healthcare spending while at the same time improving outcomes, she believes Apple’s advantages include its outsized user base, particularly in wearables; a proven services platform; and trust as a steward of data privacy. Investors are aware of Apple’s healthcare efforts, but the analyst believes they “may be missing the big picture and underappreciating the magnitude of the opportunity.” Huberty noted that her firm’s recent survey of 30 meeting participants, three-quarters of them C-level executives from leading healthcare and technology companies, showed that those polled see Amazon, Google and Apple as the technology companies that will have the largest impact on healthcare over the next five years, in that order of concentration of votes. However, they seem to be underestimating what Apple can accomplish, she thinks.
Overall, the analyst told investors that she sees healthcare as a $15B-$313B revenue opportunity for Apple by 2027. Huberty believes there are two clear paths to monetization, namely wearables and services. Sustained 40% growth in wearables alone implies Apple would reach the low end of that $15B-$313B target by 2021, she said, while access to free applications increases the value of Apple’s platform and helps the company sell more devices and its own services. Additionally, the analyst sees iCloud possibly becoming the go-to cloud service for storing individual’s health data. Huberty reiterated an Overweight rating on Apple’s shares.