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Monday, January 7, 2019

Lupin Orphan Drug Gets EC OK for the Treatment of Myotonia


Lupin is pleased to announce that the European Commission (EC) has approved NaMuscla® (mexiletine) for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders. Non-dystrophic myotonic disorders are a group of rare, inherited neuromuscular disorders which cause the inability to relax muscles following voluntary contraction. NaMuscla® reduces myotonia symptoms in adult patients, resulting in a significant improvement in patient quality-of-life and other functional outcomes1,5.
Lupin has partnering discussions ongoing for commercialization of NaMuscla® in European territories outside Germany and the UK.
The EC approval follows the positive opinion which was issued by the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), in October 2018 and will apply to all 28 countries of the European Union, Norway, Iceland and Liechtenstein. The approval makes NaMuscla®, which recently had its orphan drug designation ratified by the EMA’s Committee for Orphan Medicinal Products (COMP), the first treatment to be licensed throughout the EU for the symptomatic treatment of myotonia in adults with NDM disorders.
Lupin is preparing for the launch of NaMuscla®, which will occur in the initial markets of Germany and the UK in Q1 2019.

All the cool wearables and health tech at CES 2019


The 52nd CES is the place to be if you are interested in next-generation innovations. The show runs from January 8th till the 11th, with press conferences set to start a couple of days earlier. Over 180,000 people are expected to descend on the city, known primarily for its gambling, shopping, fine dining, entertainment and nightlife.
CES stands for the Consumer Electronics Show. In the 50 years it’s been running, thousands of products have been announced including many that have transformed the lives of people around the world.
The 2.75 million-square-foot convention will host some 4,400 exhibiting companies, including manufacturers, developers and suppliers of consumer technology hardware and content. This will be accompanied by a conference program featuring more than 1,000 speakers.
Most big wearable tech companies are here, apart from Apple that is. But don’t let this come to you as a surprise. The Cupertino outfit has been a no-show for years now, instead preferring to hold its own product launch events.
Our full coverage of the extravaganza can be seen on this link. The feed can also be seen below.

CES 2019 – Healthcare innovations to look out for


The Consumer Electronics Show (CES) is an annual event that attracts over 180,000 attendees each year, showcasing the most cutting edge, revolutionary technology imaginable. Of interest to readers of PHP however, will be the Digital Health Summit, which focuses on how technology can revolutionize healthcare and wellness. In fact, healthcare has become so ingrained in CES, that attendees can now earn CME (continuing medical education) credits for some of the sessions. I’ve highlighted a few of these innovations below; it remains to be seen how well they work in the real world, and if patients are interested in this sort of technology.

Improvements to existing fitness trackers

While current fitness trackers can measure activity, improvements to them, as well as improvements to their ecosystem, will allow for patients to track activity, as well as blood glucose, blood pressure, weight, and other biologic measurements. Being able to track your overall health, and see how it is trending over time, will allow those with multiple conditions to track their health and identify risk factors before they escalate.

Pria by Black and Decker: Medication management made easy

The PRIA by Black+Decker is an automated medication management and home health assistant (Photo from their Facebook Page)
Medication management is an ongoing struggle for patients, especially when dosing is irregular, or when different medications require different dosing scheduled. The PRIA™ by BLACK+DECKER™ Home Care Companion is an automated medication management and home health assistant that allows a caregiver to monitor an individuals medication and healthcare schedule. It is able to schedule different medication doses and schedules, provide reminder alerts, and track visits (source).

Addison: Your healthcare virtual assistant

Similar to the above, but take even further is Addison Care. This is a virtual assistant created by Electronic Caregiver. Not only is it capable of reminding users to take medications (and then being able to track adherence), it can also measure health performance. It’s a very ambitious project that was several years in development, but as you can see below, the system is very interactive, using voice activation, cameras, and other feedback mechanisms to get a more in-depth understanding of someones health state. The video below provides more detail on how it can assess a patients risk of falling:

Thought leadership in healthcare

In addition to the device above, CES also has a series of interesting talks scheduled, including:
  • Boldly Going Where No Tech’s Gone Before: A session focused on incorporating the patient perspective into healthcare, ranging from clinical trials to hospital care
  • Patient Decade Commences: Tear Down These Walls!: Continuing the patient theme, this session will be focused on patient generated health data, and includes a patient advocate as part of the panel
  • The Solution to the Opioid Crisis No One Is Talking About: A panel that sounds like something out of Star Trek will be discussing “… a bright light we refer to as Neuromodulation—a discreet, FDA-approved, implantable neural device holding powerful capabilities to intervene in the adoption of opioids by confronting the root of the cause—PAIN.”
  • Gamechanger Seqster Unites Health Data & Genomics: The idea of a single health care record owned by the patient is something of a pipedream for patient privacy advocates. However, in this session, the CEO of Seqster proposes their solution for this incredibly difficult problem.
It’ll be interesting to see what else is announced at CES; this is what I could find before the conference starts, but it remains to be seen what other innovations come about afterwards. In addition, I’m curious to see how well these innovations work in the real world, and how companies respond to concerns around patient privacy, and what feedback systems they put in place to ensure patient safety is paramount.

CES 2019: AARP trialing virtual reality for remote healthcare


Can virtual reality serve as a remote medical diagnostic tool for seniors, who often have difficulty accessing quality healthcare? An ongoing collaboration between VRHealth, a healthcare technology company specializing virtual reality solutions and data analysis, and AARP Innovation Lab is testing the concept.
The idea is to use sensors and VR technology to enable remote health monitoring. VRHealth’s telehealth platform, available in the Oculus store, enables seniors to grant key participants in the medical process, like family members or physicians, access to healthcare data collected during VR therapy sessions. Caregivers are then able to make adjustments to treatment, potentially avoiding the need for a doctor’s visit.
Putting aside the low hanging jokes about my parents and their somewhat strained relationship with technology, the idea has a lot to recommend it. Mobility issues, money, and location constraints disproportionately conspire against seniors when it comes to accessing quality healthcare.
According to Pew Research, “nearly one-in-five (19%) Americans ages 65 and older say they had a medical problem but did not visit a doctor, skipped a medical test or a treatment recommended by a doctor, did not fill a prescription or skipped doses of their medicine because of cost constraints.”
While not a replacement for in-person treatment, a virtual connection to a healthcare provider could help mitigate some of the cost and mobility difficulties associated with establishing ongoing care.
“Our telehealth platform is a crucial step in the healthcare process because it enables patients to engage in a healthcare routine in the comfort of their own home while providing access to their data directly to their doctors,” VRHealth CEO Eran Orr says. “Any adjustments that need to be made to a patient healthcare regime can be adjusted based on the data in the platform.”

Potential pitfalls involved in beaming medical data over a network include the obvious digital privacy considerations, especially with vulnerable populations like older adults. There’s also the concern that caregivers could get an incomplete picture based on inbound data, resulting in improper treatment.
VRHealth is hoping to mitigate that likelihood with robust data collection and analysis. The company’s platform uses sophisticated tracking tools while the patient is engaged in VR therapy around several different use cases, including brain health applications, memory span and cognitive skills, neck exercises, and pain management techniques. As patients engage in directed activities in an immersive, 360-degree VR environment, vital data is collected, analyzed, and communicated with caregivers.
VRHealth has a residency at AARP Innovation Lab and is demoing the tele-health platform at AARP’s CES booth.
“AARP’s presence at CES is about highlighting how technology can increase social connections and improve people’s health, wealth and personal fulfillment,” said Andy Miller, AARP’s senior vice president of innovation and product development. “VR Health is exhibiting with us at CES since its VR platform helps foster crucial connections that seek to allow physicians, patients and their families to receive critical health information in real-time in order to provide the best possible care.”

Frequency Nabs $42M for Hearing Loss Drugs, Clinical Data On The Way


Hearing loss drug development continues to attract investment, with Frequency Therapeutics announcing today that it has brought in $42 million in a Series B financing to help it move its treatment through clinical testing.
Boston area-based Frequency is working on small molecule drugs that stimulate the growth of dormant stem cells in the body, and its lead drug, FX-322, targets key sensory cells in the inner ear. The drug, which is injected into the ear, is in an early-stage clinical trial, and the company says it will announce results in the first half of this year.
The new funding round was led by Taiwania Capital Management and Axil Capital, with Yonjin Capital and DF Investments also joining in as new investors. The round included investments from previous backers Polaris Founders Capital, Alexandria Venture Investments, Cobro Ventures, Korea Investment Partners, and Emigrant Capital.
Two other companies pursuing hearing loss treatments also secured funding in recent months. Decibel Therapeutics closed a $55 million Series C round last year, and says it should enter clinical testing this year with its drugs that protect against hearing loss that’s caused by other drugs. The company is also working on gene therapy for hearing loss.
Decibel isn’t alone in the gene therapy race. Akouos announced a $50 million funding round last year to finance its gene therapy for hearing loss, and the company’s leaders hope to start clinical trials in the next three years.

Alector Files for IPO to Fund Studies of Alzheimer’s, Dementia Drugs


Alector, a biotech company focusing on the immune system to treat degenerative brain disorders such as Alzheimer’s disease, has filed to go public to fund human tests of its experimental therapies.
South San Francisco, CA-based Alector set a preliminary $150 million target for its IPO. The company has applied for a listing on the Nasdaq exchange under the stock symbol “ALEC.”
In its prospectus, Alector challenges longstanding theories that point to problem proteins—amyloid beta and tau in Alzheimer’s, alpha-synuclein in Parkinson’s, and TDP-43 in frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS)—as the root causes of neurodegeneration. Alector contends that the underlying cause of neurodegeneration is a problem with immune cells in the brain.
Alector focuses on microglia, a type of immune cell that provides nourishment to neurons and “eats” cellular debris and pathological proteins. These cells can deteriorate with age, which in turn diminishes their ability to perform their beneficial and protective role, according to Alector. The company says its drugs target mutated genes linked to neurodegeneration, which could slow or even reverse the deterioration of the brain’s immune cells.
“By restoring healthy immune function in the brain, we believe we can simultaneously counteract the multiple pathologies responsible for neurodegeneration,” the company says in the filing.
Last year, Alector advanced two drugs into early-stage clinical testing. AL001 has been dosed in 42 FTD patients; the company reported no serious side effects or problems that would limit the dose of the drug. The company plans use the IPO proceeds to move on to a Phase 1b study, with data expected in the first half of this year. The second Alector drug, an experimental Alzheimer’s treatment called AL002, started a Phase 1 dose-escalating study in healthy patients.
Alector says the IPO proceeds will also be used to advance two other drugs into human testing: the Alzheimer’s drug AL003 and AL101, which is being assessed as a treatment for multiple neurodegenerative disorders.
The Alector Alzheimer’s drugs, AL002 and AL003, are being developed under a partnership with AbbVie (NYSE: ABBV). Under the agreement, AbbVie paid Alector $205 million up front and made a $20 million equity investment in its partner. Alector could gain up to $985 million more in milestone payments, depending on the progress of the Alzheimer’s drugs. Alector is responsible for Phase 1 and 2 testing. If AbbVie exercises its options on the drugs, the pharmaceutical giant will take over development and commercialization commercialization of the treatments, if they are approved for the market.
Alector, which was founded in 2013, has raised more than $210 million in financing, most recently a $133 million Series E round last year. Alector’s largest shareholders include affiliates of OrbiMed, which own 21.4 percent of the company, and affiliates of Polaris Venture Partners, which own 21.7 percent.

Prepping US move, OrbiMed-backed Apollomics bags $100M for I/O combos


Guo-Liang Yu has big plans mapped out for Apollomics in 2019: Move the biotech’s headquarters from Hangzhou, China to Foster City, CA; scout new assets to add to its pipeline, currently led by c-Met/PD-1 combo; and build up the team to run a trans-Pacific operation. With support from a well-heeled Chinese backer, he now has $100 million to bankroll all that.

OrbiMed Asia, where Yu is a partner, got the company started in 2016 with a modest $9.75 million Series A, back when it was named CBT Pharmaceuticals.
Like a number of players in the increasingly crowded checkpoint field, Apollomics is banking on a combination approach to immuno-oncology with partners including Beijing Pearl Biotechnology, Zhejiang Bossan Pharmaceutical, Genor Biopharma and Chia Tai TianQing.
The new cash will fund, and likely add to, the more than 10 ongoing clinical trials for these combos, which will be helped by an R&D facility and manufacturing capabilities built in Hangzhou in the past year.
Kexiang Zhou, managing director of CMBI, is joining the board.