17 Mar

Alzheon Files for IPO to Fund Studies of Amyloid Alzheimer’s Drug

Alzheon is turning to the public markets to finance a new round of testing for its experimental Alzheimer’s disease drug.

The Framingham, MA, company set a preliminary $80 million target for its initial public stock offering. That figure is expected to change as Alzheon determines how many shares to sell and at what price. But the company may need more than $80 million. FDA approval could require two Phase 3 studies. Two years ago, Alzheon pegged that cost at more than $100 million.

Alzheon’s drug, ALZ-801, is a reworked version of tramiprosate, a drug that was meant to block the… Read more »

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16 Mar

Teva’s Aycardi Named Chief Medical Officer of Xenon Pharma

Xenon Pharmaceuticals (NASDAQ: XENE) announced that Ernesto Aycardi will become the company’s chief medical officer. Aycardi, a neurologist, comes to Burnaby, British Columbia-based Xenon from Teva Pharmaceutical (NYSE: TEVA), where he was a vice president. His experience also includes positions at EMD Serono, Biogen Idec, and Merck (NYSE: MRK). Xenon is developing treatments for neurological disorders.

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16 Mar

Research Headlines – European museums in an age of migration

[Source: Research & Innovation] People move from one country or continent to another for many reasons, and the pace of migration is speeding up. An EU-funded project looked at how museums can reflect the impact of these flows in today’s interconnected, multicultural world.

16 Mar

President’s Cancer Panel Recommends Shift Towards Value

This week, the President’s Cancer Panel published a new report entitled “Promoting Value, Affordability, and Innovation in Cancer Drug Treatment” that examines the price of cancer treatments in America and their value to the patients who need them. With health care costs on the rise, adopting an approach that rewards for quality – not quantity – is garnering support from stakeholders across the board. And as the Panel makes clear, the need to ensure drug prices are aligned with value is “urgent”:

“An ideal framework would integrate information on clinical outcomes, toxicities, impact on quality of life, and costs. It would inform negotiations between drug manufacturers and payers and also could guide development of value-based payment models and benefit designs that promote selection of high-value drugs by physicians and patients. Value assessments also could inform shared decision making among patients and providers and potentially improve patient outcomes,” the group writes.

This is a message that is consistent with what BIO has been saying for some time. In an opinion piece featured in The Hill, Jim Greenwood, BIO’s President and CEO, underscores the importance of a value-based model and discussed the need for expanding this approach across the entire health care system.

“Outcomes-based pricing is already happening”

For starters, Mr. Greenwood explains, doctors and hospitals are increasingly being paid not for the quantity of care they provide, but for the outcome or quality of care patients receive. Our health care system is shifting towards paying for what works, opposed to how much is administered.

“Future treatments will be more accessible and affordable”

Second, because drugmakers are accountable for patient outcomes, a value-based approach encourages insurers to ease coverage restrictions on more costly, innovative medicines. As a result, patients would have access to the affordable treatments they need and greater opportunity to enjoy healthier lives.

“This will bend the cost curve in health care”

Next, by ensuring future cures are accessible and patients adhere to their medications, value-based pricing will lower the trajectory of health care spending. By keeping patients out of the hospital and away for unnecessary doctor visits, we can lower health care costs for everyone.

“Outdated federal policies are standing in the way”

And finally, providing more legal and regulatory flexibility will help expand the use of outcomes-based drug pricing, and more importantly, advance patient health by providing better access to the treatments they need.

While value measurement in health care is not a new concept, groups like the Institute for Clinical and Economic Review (ICER) – who claim to be early advocates for this process – have also proven to lack sufficient input from third-party validators and have not been fully transparent. Echoing similar concerns raised by BIO, the President’s Cancer Panel writes:

“Limitations noted for one or more of these frameworks include lack of patient-centeredness, lack of systemwide perspective, inadequate provisions for updates as new data are obtained, lack of transparency about methodologies, and failure to engage all stakeholders.”

BIO welcomes the efforts by the President’s Cancer Panel and its emphasis to adopt an approach to health care pricing focused on value – not volume. That’s why we continue to highlight the work of the Innovation and Value Initiative (IVI), which recently introduced its Open-Source Value Project. This transparent and holistic approach for assessing value in health care puts patients first, allows for a broad range of perspectives, incorporates the latest available evidence, and considers the full range of scientifically defensible approaches. The promotion of market-based policies will help ensure patients have access to affordable, innovative medicines they need.

15 Mar

Julie Hambleton Named Ideaya Biosciences Chief Medical Officer

Cancer drug developer Ideaya Biosciences has appointed Julie Hambleton to serve as senior vice president and chief medical officer, head of development. Hambleton most recently worked at Bristol-Myers Squibb (NYSE: BMY) where she was a vice president and head of the company’s U.S. medical operations. Her experience also includes executive posts at Five Prime Therapeutics (NASDAQ: FPRX) and Clovis Oncology (NASDAQ: CLVS). Ideaya, which splits its operations between South San Francisco, CA, and San Diego, is developing drugs based on synthetic lethality, an approach that targets genetic interactions of cancer cells as a way of killing them.

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15 Mar

Call for proposals for ERC Synergy Grant

[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: ERC-2018-SyGPillar: Excellent ScienceOpening Date: Deadline: Tue, 14 Nov 2017 17:00:00 (Brussels local time)Modification Date: Thu, 15 Mar 2018Latest information: Attention: Spam messages pretending to come from the Participant Portal:
Some users are reporting spam messages pretending to come from the Participant Portal in which commercial services are being promoted.
Please be on guard against messages which look identical to Participant Portal notifications but which do not stem from the Participant Portal.
We do not have control over messages claiming to be sent ‘on behalf of’ the European Commission.
On this occasion, we would also like to remind you about phishing attempts with the Participant Portal look and feel. Phishing messages typically aim to get your password, ID or bank account details under a fake pretext, for instance inviting you to update your information online.
Please note that the Participant Portal will never ask you to send important personal and financial data via email.
Your first action against phishing is to be vigilant. Protect yourself by keeping the following advices in mind: http://ec.europa.eu/research/participants/portal/desktop/en/support/about.html

15 Mar

National Nutrition Month: The Promise of Gene Editing

One of the leading voices at the intersection of cooking and nutrition is Monica Reinagel or, as active social media users might know her, @NutritionDiva. To kick off National Nutrition Month, Reinagel (a trained chef and nutritionist) wrote a piece about the promise of gene editing, or more specifically CRISPR-Cas9 (“CRISPR”), for the website Food & Nutrition.

As a quick refresher, plants that are genetically edited do NOT carry foreign genes, whereas, the genetically modified foods available in your grocer today are the product of transgenic modification, or the transfer of genes from one organism to another. Not only does gene editing not involve the transfer of genes, but as Reinagel explains, it promises to be even more precise than transgenic modification.

Instead of transferring genetic material from one organism to another, scientists now can edit an organism’s own DNA by deleting a few nucleotide bases (the basic units in DNA) or shifting them slightly. No foreign genetic material is involved. It’s like the difference between using a photo editor to paste one person’s head on another person’s body and editing a few pixels to remove a blemish or fix flyaway hairs in a picture.

And with this new gene editing tool, scientists and farmers alike, are eager about the technology’s potential to enhance crops to solve global issues, like malnutrition.

Genetic researchers working with gene editing, along with farmers and growers, are excited about the potential for CRISPR technology to expedite solutions to a wide array of pressing concerns including climate change, malnutrition and population growth.

Reinagel goes on to note that gene editing, or GenEd as she refers to it, also alleviates concerns shared by opponents of GMOs.

For example, questions about food allergens are largely obviated by CRISPR techniques because no foreign genetic material is introduced in GenEd foods.

Even so, the verdict is still out on how this new technology will be regulated by federal agencies. Reinagel closes with the notion that many scientists agree that the product should be the focus for regulators, not the process for which the product was created.

To the extent that regulations are put in place, scientists are calling for a “product vs. process-based” review system, whereby as new foods are brought to market, they are evaluated according to the attributes and makeup of the food itself, not how these attributes were acquired.

Read the Nutrition Diva’s full piece here.

15 Mar

Solid Discloses More Problems As FDA Halts Duchenne Gene Therapy Study

Solid Biosciences recently completed a big IPO despite disclosing, last-minute, that the FDA had partially suspended a clinical test of its gene therapy for Duchenne muscular dystrophy. Less than two months later, the company has reported a new, and more ominous finding: the FDA has halted testing of the treatment, known as SGT-001, altogether.

Cambridge, MA-based Solid (NASDAQ: SLDB) said late this afternoon that the FDA has placed a study called Ignite DMD, an early-stage test of SGT-001, on clinical hold. Solid said the first patient dosed in the study on Feb. 14 was hospitalized “several days” afterwards when… Read more »

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15 Mar

Cybersecurity

[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: H2020-SU-ICT-2018-2020Pillar: Industrial LeadershipPlanned Opening Date: Deadline: Wed, 14 Nov 2018 17:00:00 (Brussels local time)Modification Date: Thu, 15 Mar 2018Latest information: The submission session is now available for: SU-ICT-01-2018(IA)

14 Mar

Research Headlines – Art to promote dialogue on contentious cultural heritage

[Source: Research & Innovation] EU-funded researchers are developing art-based methods of promoting reflection and dialogue on contentious aspects of Europe’s cultural heritage. Transmission of these methods aims to increase inclusiveness and foster convivial relations, thereby helping to shape European imagination.