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Thursday, March 18, 2021

Gilead Sciences, Novo Nordisk Expand NASH Collaboration

 Gilead Sciences Inc. and Novo Nordisk A/S on Thursday said they are expanding their clinical collaboration in nonalcoholic steatohepatitis, a chronic liver condition commonly known as NASH.

The companies said they plan to conduct a Phase 2b study to investigate the safety and efficacy of Novo's semaglutide paired with a fixed-dose combination of Gilead's investigational compounds cilofexor and firsocostat, alone and in combination in people with compensated cirrhosis due to NASH.

Semaglutide is the active ingredient in Novo's Ozempic injection and Rybelsus pills, which are both approved to treat type 2 diabetes.

Gilead and Novo said the four-arm study in roughly 440 patients will evaluate the impact on liver fibrosis improvement and NASH resolution, with recruitment starting in the second half of 2021.

Gilead, a Foster City, Calif., biopharmaceutical company, and Danish pharmaceutical company Novo announced plans in 2019 to collaborate in NASH.

The companies in November said a Phase 2a proof-of-concept study investigating semaglutide alone and in combination with cilofexor and/or firsocostat in people with NASH and mild to moderate fibrosis met its primary endpoint, showing that all regimens were well tolerated over 24 weeks.

https://www.marketscreener.com/quote/stock/GILEAD-SCIENCES-INC-4876/news/Gilead-Sciences-nbsp-Novo-Nordisk-Expand-NASH-Collaboration-32726271/

Hospitals Must Now Disclose Prices. Here's How to Interpret Them

 Need a knee replacement and wondering what your out-of-pocket cost might be? Under a new regulation from former President Donald Trump and just enacted by the Centers for Medicare & Medicaid Services (CMS) in January, hospitals must now tell you, as well as prices for other "shoppable" services that can be scheduled, from psychotherapy to routine blood tests.

Kind of.

The rule requires nearly all of the nation's 6,000 hospitals post detailed price breakdowns on their websites for at least 300 services, 70 of which are dictated by CMS. The rest are left up to the hospitals' discretion.

But, as with so much of health care, the reality of hospital price transparency is complicated.

Hospitals must post five prices for each of the procedures: gross charges; discounted cash prices; the charges they've negotiated with insurers and the minimum and maximum negotiated prices. Any hospital that doesn't comply could face penalties of up to $300 a day.

The Reasoning Behind the Hospital Prices Rule

The government developed the regulation in the hopes of empowering patients to make smarter health care purchases, while encouraging competition among hospitals and insurers that could ultimately push down health costs. But some experts question whether either goal will be met.

"Out-of-pocket estimate tools can be helpful for patients, but even the savviest consumers will struggle to navigate the big data dump required by the rule," says Ariel Levin, senior associate director for policy at the American Hospital Association (AHA).

That group sued the U.S. Department of Health and Human Services, trying to block the rule by claiming it would encourage health insurers' anti-competitive behavior, potentially hindering efforts to control costs. The AHA lost in federal court.

According to a Wall Street Journal article, some of the biggest national and regional hospital systems (including Mayo Clinic, HCA Healthcare and NewYork Presbyterian) have yet to fully reveal their prices.

Complicating the problem for consumers is that interpreting pricing information from hospitals isn't always straightforward.

For example, if a patient insured by Horizon Blue Cross and Blue Shield goes to the website for Hackensack Meridien Health in New Jersey, clicks on its price estimator tool and searches the cost of a knee replacement at that city's main hospital, she may find an out-of-pocket cost estimate of zero.

That would seem like good news, until she scrolls down the page and finds that the hospital's fees for patients without insurance would range from $38,871 to $60,271.

Then there's a disclaimer saying that people with insurance might have to pay some portion of that cash cost in the form of co-pays, co-insurance or deductibles, which are not automatically factored into the estimate.

What's more, the estimate excludes fees charged by the surgeon, who more than likely doesn't work directly for the hospital and therefore would charge separately for the knee replacement. (The regulation was written specifically to cover hospitals.)

How to Find the Prices

So how can you make sense of whatever pricing information hospitals do provide?

The first challenge is finding it.

Although the regulation requires hospitals to post pricing information on their websites, some are burying it, says Dr. Joel Shalowitz, adjunct professor of health industry management at Northwestern University in Chicago. He suggests Googling the name of the hospital and the words "price transparency" to get directly to the page with the information.

The next challenge: dealing with wide discrepancies in the procedures for which prices are offered.

The 70 required by CMS fall into four categories: evaluation and management; laboratory and pathology; radiology and medicine and surgery. The last of those categories includes a broad range of procedures that must be posted, including cataract surgery, electrocardiogram and gallbladder removal. But it's by no means a comprehensive list.

And hospitals can take liberties in deciding which services beyond the required 70 to include in their price disclosures.

Shalowitz sees even more shortcomings with the coming flood of pricing information."What's not included is any mention of quality," he says.

Add to that the reality that many health care services are ordered, and performed, by physicians who often require their patients go to specific hospitals. In that case, "your ability to make a decision based solely on prices doesn't really exist," Shalowitz says.

The bottom line: don't take the price transparency information provided by hospitals at face value.

Who to Ask for Help Getting Information

To ensure you're getting the most accurate cost, call the hospital, the physician who'll be performing the procedure and your health insurer to confirm any information gleaned from price-transparency tools.

"The hospital won't make it easy, but don't give up. Stick to your guns and say 'I want the real prices,'" advises Marni Jameson Carey, executive director of the Association of Independent Doctors.

The task of determining the cost of shoppable health services may get easier in a few years, Carey adds, thanks to a regulation set to roll out starting in January 2022. It will require health insurers to disclose a slew of pricing information online, including in-network negotiated rates, historical net prices for prescription drugs and rates paid for out-of-network doctors.

Some companies plan to take the new price-transparency data and make it easier for consumers to use. They include Healthcare Bluebook, which provides price-comparison apps for employers and other health plan sponsors. The apps let patients compare prices charged by hospitals and independent health providers such as physicians and imaging centers.

Bill Kampine, co-founder of Healthcare Bluebook, expects the data hospitals will be disclosing will enhance his company's apps. "The more data the better — it will help us round out a more complete picture" of health care costs, he says.

So, will all of this transparency ultimately bring down health costs? Don't count on it, says Dr. David Blumenthal, president of the health care research organization Commonwealth Fund.

"Shoppable services are a small fraction of total spending," he says. "This may cause the suppliers of those services to look a little more carefully at their cost structure. But does that cause the entire health system to improve? I don't see that."

https://www.nextavenue.org/hospitals-must-now-disclose-prices-heres-how-to-interpret-them/

Adverum Sets Board Slate for 2021 AM, Reiterates Commitment to Stockholder Value Creation

 Responds to Intent to Nominate Directors from Sonic Fund

Sonic’s Attempt to Control the Board Not in the Best Interests of Adverum or the Company’s Stockholders

Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that its Board of Directors is expected to nominate a Board slate consisting of three independent directors – Dawn Svoronos, Reed Tuckson, M.D. and Tom Woiwode, Ph.D. – for reelection at the 2021 Annual Meeting of Stockholders as Class I directors with terms expiring in 2024. Following the 2021 Annual Meeting, Adverum will have a continuing Board of nine directors who bring extensive biotechnology healthcare expertise across R&D, clinical development and regulatory affairs, and commercialization, as well as deep leadership, operational and financial acumen. The date of the Annual Meeting will be shared in the 2021 proxy statement.

https://www.biospace.com/article/releases/adverum-announces-board-slate-for-2021-annual-meeting-and-reiterates-commitment-to-stockholder-value-creation/

Merck Files Registration Statement on Planned Spinoff of Organon & Co.

 Organon Proposed Board of Directors To Have More Women Than Any S&P 500 Healthcare Company

Investor Day Planned for May 3 Ahead of Spinoff in Late Second Quarter 2021

Merck & Co. (NYSE: MRK), known as MSD outside the United States and Canada, today announced that it has filed a Form 10 registration statement with the United States Securities and Exchange Commission (“SEC”) in connection with the intended spinoff of its women’s health, biosimilars and established brands businesses into a standalone, publicly-traded company, Organon. Organon will register its common stock under Section 12 of the Securities Exchange Act of 1934 (the “Exchange Act”).

The Form 10 includes detailed information about Organon, such as historic financial information, as well as a description of Organon’s business and strategy and other legal and financial disclosures.

A copy of the Form 10 is available on the SEC website at www.sec.gov and can also be viewed on www.merck.com/investors/sec-filings.

Completion of the transaction is subject to a number of conditions, including effectiveness of the registration statement filed with the SEC, receipt of an opinion from tax counsel and other customary conditions. The transaction also requires final approval of the Merck Board of Directors. If all conditions are met, the separation is on track for completion late in the second quarter of 2021.

“Merck is confident that the spinoff will deliver significant benefits for both companies, better meet patient and customer needs and create value for Merck shareholders,” said Rob Davis, executive vice president, global services, and chief financial officer, Merck. “The transaction will create two companies with enhanced strategic and operational focus, which is expected to result in accelerated growth, improved agility, simplified and more efficient operating models, and improved resource allocation. Merck and Organon will be well-positioned to pursue distinct strategic agendas that will drive each company’s long-term success.”

Merck will host a virtual investor event on Monday, May 3 beginning at 10 a.m. EDT and lasting approximately two hours. At the meeting, Organon management will present its strategy, opportunities for growth and financial outlook followed by a question and answer session. Details will be provided at a date closer to the event merck.com/investor-relations.

https://www.biospace.com/article/releases/merck-announces-filing-of-form-10-registration-statement-in-connection-with-planned-spinoff-of-organon-and-amp-co-/

Sangamo: EMA Supporting Orphan Designation for Sickle Cell Treatment

 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 study partnered with Sanofi. The Committee’s decision to grant Orphan Designation was based in part on early data from three patients that had 52 weeks, 13 weeks, and 29 days of follow-up, respectively.

In recently published minutes, the Committee considered the preliminary clinical observations of BIVV003 as well as the potential of long-term effects that may obviate the need for frequent treatment suggested a clinically relevant advantage. The Committee’s published minutes report information on select patient characteristics for the first three patients treated, including genotype and history of red blood cell transfusions and vaso-occlusive crises. Sangamo and Sanofi expect to enroll a total of eight patients in the PRECIZN-1 study.

As previously indicated, Sangamo and Sanofi expect to submit updated data from the PRECIZN-1 study for presentation at a medical meeting later this year. At that time, the Companies will also provide an update on the partnered ongoing Thales study evaluating ST-400 in beta thalassemia.

https://www.biospace.com/article/releases/sangamo-announces-ema-releases-details-supporting-orphan-designation-for-bivv003-for-the-treatment-of-sickle-cell-disease/

Merck In New Collab to Advance Neurodegenerative Candidates With Amathus

 Amathus Therapeutics, a biopharmaceutical company working on small molecule modulators across diverse genetically defined diseases, has entered into a strategic collaboration with Merck to develop novel small molecule treatment candidates for neurodegenerative diseases.

In a statement on the collaboration, Amathus President and Chief Scientific Officer Edward Holson noted that the company will continue to place focus on taking advantage of genetically defined pathways and core pathologies in several indications.

“The Amathus team has demonstrated activation of molecular chaperones is possible and the potential to address core mitochondrial dysfunction with specificity holds tremendous therapeutic potential,” Holson said. “The Merck neuroscience team is an ideal partner to translate this novel approach into potential first-in-class, disease-modifying treatments for patients with neurodegenerative diseases.”

The terms of the agreement place responsibility on Amathus to identify and progress novel chaperone activators through the process of preclinical discovery. The agreement terms also give Merck the option to acquire Amathus as well as its treatment pipeline of mitochondrial-targeted drug candidates, all of which have been designed for the management of neurodegenerative disorders and renal diseases.

Merck will provide an upfront payment to Amathus if the former decides to exercise its option. Amathus will be eligible for milestone payments in excess of $500 million for each successfully developed candidate program. Merck, upon exercise of its option, will hold sole responsibility for the clinical development as well as commercialization of the candidates.

“We are excited to be working with the Amathus team to investigate the company’s novel mitochondrial-targeted candidates for the treatment of neurodegenerative conditions,” said Merck Research Laboratories’ Senior Vice President, Head of Discovery Sciences and Translational Medicine Fiona Marshall. “External collaborations that supplement and complement our internal pipeline programs remain a cornerstone of Merck’s discovery strategy.”

This new partnership with Amathus represents just another in a long line of collaborative agreements set forth by Merck. Earlier this year, Merck entered into a research partnership with clinical-stage biotechnology company Symvivo Corporation to advance a proprietary gene delivery platform across different indications. The terms of this agreement give Merck the option to obtain exclusive license of Symvivo’s platform technology to advance the delivery of oral vaccines.

Late last year, Merck also entered into an agreement with A2 Biotherapeutics to advance allogeneic cell therapy for solid tumors. Merck assists A2 with preclinical development of an undisclosed cell therapy candidate and has been given the option to exclusively develop, manufacture and commercialize the treatment following a Phase I research program. A2 receives an upfront payment from Merck under the collab, but specific financial details were not disclosed at the time of the agreement announcement.

In December 2020, Aligos Therapeutics entered into an exclusive license and research collaboration agreement with Merck, allowing both companies to further apply the Aligos-developed oligonucleotide platform in the discovery, research, optimization and development of oligonucleotides directed against a target for non-alcoholic steatohepatitis. Up to one additional target of interest in cardiometabolic disease/fibrosis was also selected under terms of the agreement.

A few days before the Aligos partnership, Germany’s Merck KGaA announced it had also entered into a strategic collaboration agreement with Artios Pharma to discover and develop several precision oncology therapies. According to the terms of the agreement, Merck will pay Artios $30 million in upfront and near-term payments, and Artios will also be eligible to receive an additional $860 million per developed target, pending Merck KGaA’s exercise of the option.

https://www.biospace.com/article/merck-enters-into-new-collab-this-time-to-advance-neurodegenerative-candidates-with-amathus/

Ocugen to Seek EUA for India-Developed COVID-19 Vaccine in US

 Pennsylvania-based Ocugen has struck a deal to sell 100 million doses of a COVID-19 vaccine India’s Bharat Biotech in the United States later this year.

In an interview with Reuters, Ocugen Chief Executive Officer Shankar Musunuri said it intends to launch the vaccine in the U.S. in the second quarter of this year, pending regulatory authorization. If the plan comes to fruition, the first doses will be imported from India and then subsequently manufactured in the U.S. by Ocugen. The two companies first announced plans for the deal in February.

Ocugen and Bharat Biotech have been working together since December to develop COVAXIN, an advanced stage whole-viron inactivated vaccine. COVAXIN, a two-dose vaccine, has already received authorization in India for people ages 12 and above. In February, more than 30 million doses of COVAXIN had been administered to health professionals and front-line workers in India.

When the companies partnered in December, Ocugen said it had assembled a Vaccine Scientific Advisory Board to evaluate the clinical and regulatory path to approval in the U.S. market.

Earlier this month, Bharat announced interim results of its Phase III trial that showed efficacy of 81% for its COVID-19 vaccine. The interim data analysis was based on 43 recorded cases of the disease in the trial of 25,800 people. So far, the U.S. Food and Drug Administration has authorized three different vaccines in the U.S. developed by Moderna, Pfizer and BioNTech and most recently, Johnson & Johnson.

The FDA has held off authorizing AstraZeneca’s vaccine, initially due to concerns over efficacy data. Multiple countries in Europe and Asia that previously authorized the AstraZeneca vaccine have now paused vaccination efforts with that medication due to concerns over clotting issues.

“The evaluation of COVAXIN has resulted in several unique product characteristics including long-term persistence of immune responses to multiple viral proteins, as opposed to only the spike protein, and has demonstrated broad spectrum neutralizing capability with heterologous SARS-CoV-2 strains, thus potentially reducing or eliminating escape mutants. Requiring only a standard vaccine storage temperature of 2-8oC and with the potential to treat all age-groups, COVAXIN may offer an important option to protect lives across America,” Musunuri said in a statement last month when Ocugen and Bharat announced the commercialization agreement.

Ocugen and Bharat hope to become the fourth vaccine authorized in the United States. According to Reuters, the companies will meet with the U.S. Food and Drug Administration in April to press for EUA based on the data from the ongoing Phase III study. Ocugen’s Musunuri said the FDA is “fine with the way the interim analysis is being done” and believes that will make the path toward authorization easier.

Musunuri said COVAXIN had the potential to work against COVID-19 variants and Ocugen could initially focus on children as it was likely to be safe for those over the age of 12, Reuters added.

“Like a polio virus given to babies, this could be safe for all children, high-risk groups, pregnant women and people with comorbidities,” Musunuri said of COVAXIN, according to the report.

https://www.biospace.com/article/ocugen-to-seek-eua-for-india-developed-covid-vaccine-in-the-united-states/