Why Did The FDA Deny Approval For A Monthly HIV Injection Treatment?

Last week, the FDA denied the approval of Cabenuva, the first of its kind monthly HIV injection treatment. Developers hoped that the monthly injection would replace daily pills, thus making life easier for HIV patients and also possibly removing some of the stigma associated with the disease.

According to ABC News, the FDA had no concerns with the drug’s efficacy or safety. Rather the application’s denial was due to concerns regarding the drug’s manufacturing process. In their Complete Response Letter (CRL) regarding the New Drug Application, the FDA stated their rejection was related to “Chemistry, Manufacturing, and Controls (CMC),” EMPR reported.

Cabenuva passed trials and proved to be effective at repressing virus levels in HIV-positive patients. New trials will not be required as the FDA denial was not related to concerns with drug safety. Instead, the manufacturers plan to “work closely and with urgency with the FDA to determine the appropriate next steps for this new drug application,” a drug spokesperson told ABC News.

“Clearly this is disappointing and not the news we hoped for,” said a spokesperson.

MOSCOW, RUSSIA MAY 19, 2019: A doctor collects a blood sample for an HIV test as part of the Stop … [+]

ARTYOM GEODAKYAN/TASS

HIV is a virus that weakens a person’s immune system by interfering with their ability to fight off infection. Although there is no cure, there are certain medications that can help control the disease by slowing the virus’ progression in the body. These drugs may also aid in lowering an individual’s risk of spreading HIV to others by reducing the amount of the virus in your body, the Center for Disease Control and Prevention report.

The Cabenuva injection contains two active ingredients (cabotegravir and Janssen’s rilpivirine). The long-lasting drug is administered every four weeks. For some HIV patients there is a fear of stigma associated with their health status. Release from the dependence of daily pills may save some from this stigma.

In addition to HIV treatment, there are also medications used called PrEP (Pre-Exposure Prophylaxis), which help prevent HIV negative individuals from contracting the virus. The combination of these HIV prevention and treatment medications have helped to slow the rate of HIV transmission in the U.S. and improve the lives of those living with the virus.

Although the FDA cited that there were no concerns with the safety of Cabenuva, doctors express concerns of may come along with a monthly injection versus a daily pill. For example, patients who miss an injection may be at greater risk for the virus to grow in numbers and strength, ABC News reported.

The drug application was filed by ViiC Healthcare, which is majority-owned by GlaxoSmithKine. Pfizer and Shionogi also have small stakes.

https://www.forbes.com/sites/danadovey/2020/12/30/why-did-the-fda-deny-approval-for-a-monthly-hiv-injection-treatment/#606178377e6a

Stem Cell Banking Becomes A Reality For Man’s Best Friend

For millions of Americans, pets are family. And we do almost anything to provide for our loved ones and ensure they age without pain and suffering. So why would our four-legged family members be any different?

With new preventative strategies and access to health technologies, they don’t have to be. In fact, the same cord blood banking system used for human babies is now available for our fur babies. Any new puppy parent in the U.S. can have stem cells from the routine spay and neuter process shipped to Gallant, the first stem cell banking system for dogs nationwide. Just like with people, the intended purpose is to use the body’s own stem cells to address future diseases and health issues in lieu of traditional medications – which often only mitigate symptoms and have side effects – or surgeries.

“We have the opportunity to advance the entire science, by working with pets first,” contends the founder and CEO of Gallant, Aaron Hirschhorn. According to him the regulatory landscape around pets is a lot more flexible than humans, creating a potential opportunity to not only improve the quality and length of life for man’s best friend, but also to advance the knowledge around stem cell therapies across the spectrum.

It’s estimated that pet owners will spend more than $75 billion on pets, and more than $18 billion on vet care in 2019 alone. Additionally, more than 1 million pets will be spayed or neutered every month in the U.S – highlighting how big the existing pet health market already is, and the frequency of routine procedures. With tens of billions spent and millions of veterinary visits, Gallant emphasizes that the process for collecting pet stem cells can be seamlessly integrated into the current pet care system. Instead of throwing away the young stem cells, they are simply cryopreserved for later use in prospective treatments for future ailments.

However, the Gallant kits are not in vet clinics just yet. They have to be ordered directly by the pet parent, and can set you back hundreds of dollars for the collection, medical couriering, and storage. But, because the collection happens during the routine spay and neuter process, any vet can store stem cells in the Gallant kit. And, the team argues the costs are 80%-to-90% less than comparable treatments across the lifespan of a pet.

While there has been a lot of conflicting information about stem cell therapy success in humans, some stem cell therapies have been demonstrated in clinical studies to help dogs with numerous ailments including osteoarthritis, atopic dermatitis, torn ligaments, and chronic dry eye. “In my experience with clinical trials and evaluating dogs with debilitating arthritis, I’ve seen first-hand how cell therapy can change lives,” said Dr. Linda Black, Chief Scientific Officer at Gallant. “I’m committed to developing therapies that dramatically improve the quality of life for dogs.”

Gallant’s research team claims it is developing first-of-its kind biotechnologies and treatment methods – having committed to spend millions on R&D pursuing both autologous therapies (which use a dog’s own stem cells) and allogeneic therapies (which any dog can use). And they have a lot of new resources and partners to follow through on that commitment.

Gallant on Shark Tank

DISNEY

Puppies And Sharks

Gallant recently appeared on Shark Tank, striking a deal with both 23andMe’s Anne Wojcicki and Lori Greiner, with the Sharks agreeing to invest $500k in exchange for 5% of the business. Through this partnership, the team believes that Anne’s expertise in biotech and Lori’s experience bringing products to the consumer market will help continue consumer education on the power of stem cell therapy.

But the animal biotech team at Gallant has been making other moves to round out company expertise and influence. The board and advisors include heavy hitters from the human cord blood banking world as well as the former president of PetSmart Charities. The scientific team also boasts more than 100 years of collective experience in regenerative medicine. In tandem, the company has been able to raise $11 million in funding, not counting the Shark Tank money.

And yet, possibly the biggest step toward making the company a nationwide success is the recent acquisition of the vet division of Cook-Regentec, including its intellectual property, existing stem cell banking operations, and its pipeline of cell therapy products derived from reproductive tissue. Meaning, vets from the division have treated hundreds of cats and dogs with their own banked stem cells.

With a potentially huge market, wide-ranging expertise, and funding, the future of Gallant is bright. But its impact is yet to be determined. If it delivers on its promises, it stands to change how we think about caring for our four-legged family members – and how early we begin to think about preventative health care. It also brings about the possibility that our companions can teach us a thing or two about science through stem cell research.

https://www.forbes.com/sites/nicolefisher/2020/12/31/stem-cell-banking-becomes-a-reality-for-mans-best-friend/#64880e981b48

Will Walgreens Partnerships Pay Off In 2020?

The myriad partnerships Walgreens Boots Alliance has signed in recent years will be closely watched in 2020 amid speculation about the retail drugstore giant’s next big move.

Rumors have swirled that Walgreens may be taken private in a massive leveraged buyout involving private equity firms like KKR & Co., according to reports in the last two months. This is speculation Walgreens hasn’t confirmed nor publicly entertained as a strategy.

In the meantime, Walgreens observers are anxiously awaiting some financial results from the myriad partnerships that the retail pharmacy chain has formed with health insurers like Humana, medical care providers like VillageMD and UnitedHealth Group’s and Optum’s MedExpress urgent care unit and the Kroger grocery chain. Most of these efforts have been considered tests and have yet to be scaled nationally.

“We believe all these partnerships aim at increasing traffic to (Walgreens) stores and thus boost sales,” Mizuho Securities’ Ann Hynes said in a report last month, echoing her fellow analysts who regularly ask about the partnerships on earnings calls and investor meetings. “However, we view so far these partnerships have not yielded material financial benefits to (Walgreens) yet.”

In some of his most recent public comments, Walgreens CEO Stefano Pessina reiterated at the 2019 Forbes Healthcare Summit earlier this month in New York that partnerships remain the most cost-effective way for the global drugstore chain to remain competitive and grow in the future.  “The partnership is the best and cheapest way to transform the industry you are working in,” Pessina told Forbes Chairman and Editor-in-Chief Steve Forbes in an interview at the summit.

Walgreens strategy contrasts with that of rival CVS Health, which a year ago spent almost $70 billion buying Aetna, the nation’s third largest health insurer.

CVS observers see Aetna’s more than 20 million health plan subscribers increasingly being guided to CVS drugstores for lower cost retail health services over Walgreens. CVS is enticing customers with lower cost personal care items and primary care services via its MinuteClinics and a new “HealthHub” format that will be in 1,500 stores by the end of 2021.

Should Walgreens entertain the idea of going private via leveraged buyout, there is concern new ownership could slow capital expenditures on the partnerships in favor of boosting profits and cash flow. Walgreens also has more than 9,200 stores and management already decided in recent months to raise its annual cost-cutting target to “at least $1.8 billion by fiscal 2022,” Walgreens global chief financial officer James Kehoe said two months ago.

Still, Walgreens will have to continue to pursue ways to bring in new customers and any new owner or management team won’t change plans to shift from a healthcare strategy.

“We’re strongly focused on creating neighborhood health destinations around a more modern pharmacy,” Alex Gourlay, Walgreens co-chief operating officer said in October. “We have made significant steps during the year to develop our primary care business.”

https://www.forbes.com/sites/brucejapsen/2020/12/31/will-walgreens-partnership-strategy-begin-2020-payoff/#1390d470103e

Medicare For All Could Die In 2020 As Private Insurers Add Seniors

Health insurers are expected to make it more difficult on Presidential candidates pushing single payer versions of “Medicare for All” after they added hundreds of thousands of seniors to their private Medicare Advantage plans this year.

New benefits for seniors under Medicare Advantage began Wednesday, the first day of the 2020 New Year, in what has already been a record for the number of health plans participating in a program that offers seniors the same benefits as traditional Medicare plus extras like preventative care and outpatient healthcare services.

This new Medicare Advantage enrollment comes as most Democrats running for their party’s nomination for the Presidency back off a single payer version of Medicare for All that would uproot the private insurance industry.

In Iowa, for example, Sen. Bernie Sanders of Vermont continues to push single payer Medicare, but former Vice President Joe Biden has launched ads in recent weeks in touting an effort to build on existing coverage under the Affordable Care Act, which offers subsidies for private coverage and has expanded Medicaid, mostly through private insurers. Meanwhile, Sen. Elizabeth Warren, once considered a single payer “purist” has backed off moving all Americans to a government-run healthcare system in favor of first bolstering the ACA and introducing a public option, as the Washington Post reported less than two months ago.

The lack of momentum for a single payer version of Medicare for All among Democrats vying to challenge President Donald Trump should Republicans re-nominate him isn’t lost on health plans signing up seniors to Medicare Advantage.

“You deal with the facts as they are today,” Centene CEO Michael Neidorff said last month in an interview at the 2019 Forbes Healthcare Summit in New York. “Things like Medicare for All, I view as a sound bite. Even some of the candidates are backing off of it.”

Centene is poised to gain a stronger foothold in the Medicare Advantage business once its acquisition of WellCare Health Plans is approved this year. Centene’s rivals in the health insurance business including Anthem, the Aetna unit of CVS Health, Cigna, Humana and UnitedHealth Group. All of these big health insurers as well as smaller plans and startups are also expected to see big gains in Medicare Advantage enrollment this year.

These insurers have been selling perhaps their richest benefit packages for Medicare Advantage, thanks to new rules that allow Medicare Advantage plans to offer more benefits to seniors. Medicare Advantage plans contract with the federal government to provide extra benefits and services to seniors, such as disease management and nurse help hotlines with some also offering vision, dental care and wellness programs.

Beginning this month, health insurers are going to begin reporting their fourth quarter 2019 earnings and updated outlooks for 2020, which are expected to include robust numbers from Medicare Advantage enrollment. UnitedHealth Group, the nation’s largest health insurer, will be the first to report on Jan. 15.

Given the expansions of many established health plans into new regions and an increasing number of new entrants and startups selling Medicare Advantage, enrollment for 2020 is expected to eclipse 2019’s record. Enrollment last year in Medicare Advantage plans surpassed 22 million, which is 35% of total Medicare beneficiaries and it’s expected to reach 24 million for the 2020 plan year. 

And any additional enrollment for 2020 will be difficult to uproot as candidates are finding out on the Presidential campaign trail.

https://www.forbes.com/sites/brucejapsen/2020/01/01/medicare-for-all-could-die-in-2020-as-insurers-add-seniors/#5b85e8045e01

IMO 2020 – a major shake-up for oil and shipping

Tougher rules on sulphur emissions from ships came into effect on Wednesday, in the biggest shake-up for the oil and shipping industries for decades.

IMO 2020

From Jan. 1, United Nations shipping agency the International Maritime Organization (IMO) will ban ships from using fuels with a sulphur content above 0.5%, compared with 3.5% now.

The regulations are aimed at improving human health by reducing air pollution.

Only ships fitted with sulphur-cleaning devices, known as scrubbers, will be allowed to continue burning high-sulphur fuel. Ship owners can also opt for other sources of cleaner fuel such as liquefied natural gas (LNG).

Failure to comply with the global regulations will result in fines or vessels being detained and in some jurisdictions the risk of imprisonment, which could affect vital requirements such as insurance cover.

Enforcement will be policed by flag and port states rather than the IMO and industry officials are still unsure about whether there will be full compliance when it kicks in.

“There are still questions of what will happen at sea and how will this be enforced in terms of how compliance will be measured. While there is great momentum, there are still more questions than answers,” said Paul Taylor, global head of shipping & offshore with France’s Societe Generale CIB.

Refineries separately face significant costs to adapt to the new fuel specifications.

WILL THERE BE ENOUGH LOW SULPHUR FUEL?

Oil majors including BP and Royal Dutch Shell have announced they are producing very low sulphur fuels that meet the 0.5% requirements.

While major fuel bunkering ports such as Singapore, Fujairah in the United Arab Emirates and Rotterdam in the Netherlands have compliant-fuel supplies, analysts and shipping firms are still unclear what will happen at smaller ports given the need for ships to plan their sailing routes.

ARE THERE ANY SAFETY ISSUES WITH THE NEW FUEL?

It remains unclear what impact there will be with mixing very low sulphur fuels of 0.5% together, which at this stage have not been fully tested on ship engines. One of the risks is that the level of sediment created could damage engines at sea.

While guidance has been issued on avoiding mixing different fuel batches, the issue continues to raise worries especially after a major problems with bunker fuel contamination in 2018.

“We still have concerns over safety and the availability of compliant fuels in every port worldwide. This is a pressing issue,” said Guy Platten, secretary general of the International Chamber of Shipping trade association.

WHAT ABOUT SCRUBBERS?

There is still a question over whether jurisdictions and ports could restrict the use of certain types of scrubbers due to uncertainty over the effects of the waste water that gets pumped into the sea.

Last year, ten environmental groups called on the IMO to impose an immediate ban on the use of scrubbers.

Users of the devices argue that there is no conclusive scientific research showing that discharges from open loop scrubbers – which wash out the sulphur – cause environmental harm and their use was safe. Analysts say there is still the possibility of tighter restrictions, which would add to the costs of those investing in them.

The IMO has encouraged further study into the impact of scrubbers on the environment.

https://www.marketscreener.com/SOCIETE-GENERALE-4702/news/IMO-2020-a-major-shake-up-for-oil-and-shipping-29784756/

With Scrutiny of Insulin Drugmakers, Is Novo Nordisk a Buy for 2020 Investors?

The last three months have been good ones for investors in the pharmaceutical and biotech markets and with a new year upon us, some analysts are already picking potential winners in the market for 2020. One of those companies could be Denmark-based Novo Nordisk.

Over the last 12 months, shares of the Danish drugmaker have steadily increased by 24% to today’s price of $57.68, as of 10:04 a.m. Novo Nordisk is one of the biggest manufacturers of diabetes drugs and insulin in the world. While insulin manufacturers, including Eli Lilly and Sanofi, are under fire by lawmakers in the United States due to the high prices, one analyst has pegged the company as a buy in 2020, especially with the approval of Rybelsus, an oral-semaglutide. In September, the U.S. Food and Drug Administration approved Rybelsus, a once-daily glucagon-like peptide-1 (GLP-1) receptor agonist, approved for type 2 diabetes. Writing in The Motley Fool, analyst Prosper Junior Bakiny said “the addressable market” for Rybelsus “makes its prospects mouth-watering.”

The FDA is continuing to review Rybelsus for another indication to reduce the risk of major adverse cardiovascular events (MACE) such as heart attack, stroke, or cardiovascular death in adults with type 2 diabetes and established cardiovascular disease. Novo Nordisk said it anticipates a decision on that indication in the first quarter of 2020.

“Thus, sales of Novo Nordisk’s GLP-1 drugs will likely continue leading its revenue on an upward trajectory, and the company’s shares could continue climbing in 2020,” Bakiny said after rating the company a “buy” for 2020.

Not only did the FDA approve Rybelsus, in October, the regulatory agency also expanded the label for Novo Nordisk’s Fiasp (insulin aspart injection) to include use in insulin infusion pumps for the improvement of glycemic control in adults with type 1 or type 2 diabetes. Fiasp, a rapid-acting insulin, was first approved by the FDA in 2017 for use by intravenous infusion under supervision by a healthcare professional or by subcutaneous multiple daily injections in adults with diabetes.

In addition to the approval of Rybelsus and the expanded label for Fiasp. Novo Nordisk has spent the past several months forging collaborations with various companies as it looks to expand its pipeline in new directions. In October, the company entered into an R&D deal with bluebird bio to develop next-generation genome editing therapies for genetic diseases, including hemophilia. The initial focus of the three-year deal will be on correcting FVIII-clotting factor deficiency. Then, in November, Novo Nordisk partnered with Dicerna Pharmaceuticals to discover and develop therapies for liver-related cardio-metabolic diseases. The two companies will leverage Dicerna’s GalXC RNAi technology platform to explore more than 30 liver cell targets. The indications for the R&D pact are broad, including chronic liver disease, non-alcoholic steatohepatitis, type 2 diabetes, obesity, and rare diseases.

https://www.biospace.com/article/is-novo-nordisk-a-buy-in-2020-/

Acceleron and Fulcrum Strike Deal to Address Pulmonary Diseases

Acceleron Pharma and Fulcrum Therapeutics are entering 2020 as partners to tackle pulmonary diseases. On Monday, the two companies forged a collaboration and license agreement that has the potential to be worth more than $450 million.

The Cambridge, Mass.-based companies inked a deal to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space. Fulcrum has a product engine and target identification platform that uses computational biology, as well as a proprietary relational database that will help in the identification of small molecules that control the expression of certain genes. Acceleron will use Fulcrum’s platform to focus on genes that are known to impact specific pathways associated with pulmonary disease, the companies said. Fulcrum has also used the platform for drug discovery in a range of genetically defined muscle, central nervous system and blood disorders. Acceleron will be responsible for all development and commercialization activities for any potential therapeutics identified via this platform.

Habib Dable, chief executive officer of Acceleron, said the collaboration brings in Fulcrum’s skill in identifying drug targets that are based on the modulation of genetic pathways associated with disease. That knowledge, Dable said, will be paired with Acceleron’s “deep expertise in TGF-beta superfamily signaling.” The different skill sets the companies bring together will be aimed at generating what they hope will become disease-modifying therapies.

For Acceleron, the deal comes several months after its mid-stage asset sotatercept received Orphan Drug Designation from the U.S. Food and Drug Administration as a potential treatment in pulmonary arterial hypertension (PAH). Sotatercept is being evaluated in two Phase II trials in patients with PAH, with top-line results from one of those trials expected in the first quarter of 2020. Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH, a rare progressive disorder that is characterized by the constriction of pulmonary arteries and elevated blood pressure in the pulmonary circulation.

“With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase II trials in pulmonary arterial hypertension—and ACE-1334, we underscore our growing commitment to the development of novel therapies for patients with pulmonary diseases of high unmet medical need,” Dable said in a statement.

Under terms of the agreement, Acceleron will provide Fulcrum with an upfront payment of $10 million and will reimburse the company for relevant R&D costs. Fulcrum will also R&D and commercial milestone payments of up to $295 million for the first product to be approved as a result of this collaboration. Also, Fulcrum will receive up to $143.5 million in additional milestone payments for all subsequent products commercialized, the companies said.

Fulcrum CEO Robert J. Gould said the collaboration with Acceleron builds on the “proven potential” of the company’s platform to identify therapies that can address the root cause of diseases. He noted that the platform led to the discovery of Fulcrum’s mid-stage asset losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD) in patients with a genetically confirmed diagnosis of FSHD.

“This new opportunity to screen and identify pulmonary disease-specific therapies is another reflection of the broad potential applications of the Fulcrum platform in gene modulation,” Gould said in a statement.

https://www.biospace.com/article/acceleron-and-fulcrum-tie-up-to-develop-therapies-for-pulmonary-diseases/