ViiV files first-in-class HIV drug in Europe

ViiV Healthcare has filed its new HIV drug fostemsavir for approval in Europe, a few weeks after the US, as a rescue therapy for people who can’t control their infection with current drugs.

The HIV specialist – which is majority-owned by GlaxoSmithKline – is seeking approval from the EMA of fostemsavir as an add-on to other antiretroviral therapy (ART) drugs in adults with multidrug resistant HIV.

The first-in-class HIV attachment inhibitor – which binds to glycoprotein 120 (gp120) on the envelope of HIV and stops the virus latching on to host cells – will be targeted at patients for whom an effective virus-suppressing regimen can’t be selected because of “resistance, intolerance or safety considerations.”

Its mechanism of action means there is no cross-resistance to other classes of  ART drug, according to ViiV.

HIV’s notorious ability to mutate and develop resistance to current drugs means that while most people with HIV can expect long-term suppression with drug therapy, some experience treatment failures that progressively limit the drugs they can use to control the infection.

“Although these individuals make up a small percentage of the total number of people who live with HIV, their unmet treatment needs are life-threatening and we are committed to addressing them,” said ViiV’s chief executive Deborah Waterhouse.

The FDA filing is based on the phase 3 BRIGHTE study, which showed that fostemsavir was significantly better than placebo when added on to current therapy at reducing viral load in this hard-to-treat patient population.

Fostemsavir is just one of a series of new drugs that ViiV is hoping will help it compete in the coming years with Gilead Sciences, the market leader in HIV treatment and its arch-rival in the category.

It’s not expected to be a massive earner for the company – analysts have predicted peak sales could be around $400 million a year – but its clinical importance to patients starting to lose control of their infection is profound.

ViiV’s new generation of two-drug oral therapies like Juluca (dolutegravir/rilpivirine) and long-acting injectable ART therapies are the key to its battle with Gilead, which is in ascendency at the moment with its single-tablet three-drug product Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide fumarate).

Launched in 2018, Biktarvy made almost $3.2 billion in sales in the first three quarters of 2019, becoming Gilead’s top-selling HIV therapy ahead of four-drug combo Genvoya (elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide fumarate).

Meanwhile, ViiV suffered a setback last month when the FDA rejected its once-monthly injectable Cabenuva, based on integrase inhibitor cabotegravir and Johnson & Johnson’s non-nucleoside reverse transcriptase inhibitor (NNRTI) rilpivirine, after finding fault with manufacturing data for the drug.

https://pharmaphorum.com/news/viiv-files-first-in-class-hiv-drug-in-europe/

Glaxo CEO Walmsley aiming for six new approvals in 2020: #JPM20

GlaxoSmithKline’s CEO Emma Walmsley has said the company hopes to get at least six new drug approvals in the next year.

In an interview at the JP Morgan Healthcare Conference in San Francisco with CNBC, Walmsley said there is a lot of data expected in 2020.

Walmsley said in the interview that the next year could see “at least six approvals of new medicines or new indications”.

GSK is asking for approval for belantamab mafodotin, a treatment for multiple myeloma, a potential first-in-class drug that binds to the B cell maturation antigen (BCMA) that is also targeted by CAR-T therapies in clinical development.

A look at GSK’s pipeline shows its novel two-drug HIV treatment is top of the list, although this last month suffered a knock-back from the FDA.

The combination of cabotegravir and rilpivirine developed by its ViiV Healthcare joint venture with Pfizer and Shionogi is unlikely to need further trials for approval as the FDA cited issues with manufacturing controls in its rejection letter.

Also in registration phase is GSK’s asthma triple therapy Trelegy (fluticasone furoate+umeclidinium+vilanterol), which last year produced results in the CAPTAIN study showing it was better at improving lung function in uncontrolled asthma than the company’s two drug combo Relvar/Breo (fluticasone+vilanterol).

At the time of the results last May GSK’s R&D chief Hal Barron said Trelegy was on course for a filing in uncontrolled asthma.

Also in registration stage is Zejula (niraparib), the PARP inhibitor added to GSK’s portfolio following its $5.1 billion acquisition of Tesaro.

This is already approved in late line ovarian cancer by the FDA since October, with other regulators possibly following suit soon.

In Japan daprodustat for anaemia associated with chronic renal disease is under review, and there are around a dozen other drugs in late stage development, ranging from therapies for bacterial infections, rheumatoid arthritis, and HIV.

Walmsley added that despite the focus on a mean-but-lean R&D operation, the company would still focus on bringing returns to shareholders.

She said: “We want to make sure that that innovation is being brought to patients but our commitment to the dividend remains extremely strong and there’s no change to our policy around that.”

GSK’s CEO Walmsley aiming for six new approvals in 2020

#JPM20: UPittsburgh Med Center venture arm in $1B life sciences commitment

University of Pittsburgh Medical Center’s venture capital arm is committing $1 billion in investments toward developing new drugs, diagnostics and devices by 2024.

The investments will be targeted to complement the work already underway in Pittsburgh, including a $200 commitment toward an immunotherapy-focused partnership with the University of Pittsburgh, officials said in a statement.

UPMC Enterprises’ initial focus has been on the use of immunotherapies for cancer, transplantation and diseases related to aging. But the group is expanding to look at retinal and respiratory diseases, autoimmune diseases and neuroinflammation.

“The common link among our investments will be that each has a direct and powerful impact on how we care for our patients, while generating a significant financial return,” said Executive Vice President Jeanne Cunicelli in a statement. She previously served as managing director at Bay City Capital in San Francisco.

UPMC has invested more than $800 million in its entrepreneurial efforts to date, primarily in digital solutions, which have returned more than $1.5 billion, officials said.

Since 2018, UPMC Enterprises has formed five companies in the translational sciences sector and invested in biotech company Werewolf Therapeutics as well as 30 internal research projects.

Companies and internal programs funded by UPMC Enterprises have access to what Cunicelli calls “industry-grade” infrastructure.

They pointed to the example of a startup named Generian—co-founded by UPMC’s Toren Finkel to tackle diseases related to aging—which was able to access “top business and legal talent,” a high-throughput facility for screening potential drug targets and access to chemists worldwide, officials said.

Other investments have included BlueSphere Bio, co-founded by physician-scientists Mark and Warren Shlomchik and focused on rapid, personalized T-cell therapies for cancer; TTMS, with a mission to discover the next generation of effective immunotherapy targets; and Abound Bio, which is developing antibody libraries to generate biologic therapeutics, officials said.

https://www.fiercehealthcare.com/hospitals-health-systems/upmc-enterprises-announces-1b-life-sciences-investment-commitment

#JPM: Esperion tees up for cholesterol-busting meds—at affordable prices

With approvals looming for two cholesterol-busting drugs, Esperion is getting ready to rumble. It’s building a team of 300 sales reps to make sure doctors know to prescribe the new drugs to patients who struggle to control their cholesterol. And it plans to sell the drugs at a price that ensures patients prescribed them can actually afford them.

When asked how the biotech landed on that price for bempedoic acid and its pill combining bempedoic acid with ezetimibe, a cholesterol-busting drug often prescribed with statins, Chief Commercial Officer Mark Glickman said Esperion worked with managed care organizations to determine a price in an affordable tier.

“Getting the best formulary position was the goal, to make it affordable to the healthcare system and the patient,” Glickman said at the J.P. Morgan Healthcare Conference. “Believe it or not, we still do OK too. It doesn’t have to be win-lose.”

We don’t know the price yet, but if Esperion keeps its promise, it’ll be a far cry from the trend of ever-rising prices in the biopharma industry—a pattern that longtime biotech investor Alexis Borisy just started a new company to combat.

“The biggest mistake that pharma companies made over the last several years as they asked managed care what would be a good price … and then ignored them,” Glickman said. “I promise you, nobody told Sanofi and Regeneron that $14,000 was a good price for a PCSK9.”

Esperion expects the FDA to rule on its drugs in February. It has also filed the duo for approval in Europe. Its pivotal program included four studies for bempedoic acid and one study of the combo pill in patients who have atherosclerosis—plaque buildup in their arteries—or who are at risk of developing the condition. By JPM 2021, Glickman is confident Esperion will be the “little company that could.”

“We will be looking back at the story of how many patients we helped this last year,” he said. “It will be a story about what we’ve done well: made sure physicians are educated about this wonderful medicine, but the dire state of getting LDL cholesterol to goal.”

Despite the success of multiple statins—a class of drugs that Esperion CEO Tim Mayleben thinks has no comparator in any other disease area—between 10% and 20% of people who take them still struggle to lower their cholesterol to a level deemed healthy.

“Think about the 40 million people taking a statin today. That’s probably 9 million or 10 million people who can’t get their bad cholesterol to a healthy level on statins alone,” Mayleben said. Some of those people can’t take statins at all because they suffer side effects, while others can’t take enough of a statin to reduce their cholesterol to a healthy level.

If one statin doesn’t quite make a dent in a person’s cholesterol, their doctor can’t just add a second statin to their treatment.

“That would be toxic, so people need a nonstatin means of lowering bad cholesterol. That’s where our drug comes in,” Mayleben said.

If approved, Esperion reckons bempedoic acid will be the first once-daily, non-statin pill to lower LDL, or “bad,” cholesterol in about 20 years.

Several big names in pharma have tried to bring a non-statin drug to market: Pfizer, Roche, Eli Lilly and Merck poured money into a class of drugs called CETP inhibitors that turned out to be a bust. That string of failures may have turned off would-be cholesterol busters.

“The drug class was not successful. Bit companies spent lots of money on this and were unsuccessful. I think it’s a little bit like the Alzheimer’s area today; after a number of failures, people start to give up and say, ‘let’s not keep putting good money after bad until we figure something out,” Mayleben said.

https://www.fiercebiotech.com/biotech/jpm-esperion-tees-up-to-launch-cholesterol-busting-meds-at-affordable-prices

Boston Scientific down 6% on preliminary 2019 revenue

Boston Scientific (BSX -6.3%) reports preliminary Q4 and FY 2019 sales.

Q4 revenue is expected to be ~$2.9B, representing growth of ~13.4% on a reported basis, compared to previous guidance range of 13% to 15%.

FY 2019 sales of ~$10.74B, growth of ~9.3% on a reported basis, compared to prior guidance of 9% to 9.5%.

Q4 and FY 2019 Endoscopy sales of $500M and $1.89B, respectively.

Q4 and FY 2019 Urology and Pelvic Health sales of $380M and $1.41B, respectively.

Q4 and FY 2019 Rhythm and Neuro sales of $820M and $3.14B, respectively.

Q4 and FY 2019 Cardiovascular sales of $1.15B and $4.21B, respectively.

Q4 and FY 2019 Specialty Pharmaceuticals of $60M and $80M, respectively.

The company estimates that it will exceed its previous EPS guidance of $0.22 to $0.25 for Q4 and $0.72 to $0.75 per share for FY 2019, on a GAAP basis.

The company’s non-GAAP EPS will be within its previous guidance range of $0.42 to $0.45 per share for Q4 and a range of $1.55 to $1.58 per share for FY 2019.

Boston Scientific will webcast its conference call discussing financial results and business highlights for Q4 and FY 2019 on Wednesday, February 5, 2020 at 8:00 a.m. EST.

#JPM20

https://seekingalpha.com/news/3531683-boston-scientific-down-6-on-preliminary-2019-revenue

Biocept validates liquid biopsy tests in cerebrospinal fluid

Nano cap Biocept (BIOC +19.4%) is up on more than double normal volume in early trade in response to its announcement that its Target Selector liquid biopsy tests can now be used to assess the presence of circulating tumor cells and biomarkers in the cerebrospinal fluid of breast or lung cancer patients suspected of brain or central nervous system metastases.

Liquid biopsy tests are typically performed with peripheral blood samples.

https://seekingalpha.com/news/3531692-biocept-validates-liquid-biopsy-tests-in-cerebrospinal-fluid-shares-up-19

Alexion to launch study of Ultomiris in ALS

Alexion Pharmaceuticals (ALXN -2%) plans to launch a late-stage study, CHAMPION-ALS, this quarter evaluating Ultomiris (ravulizumab-cwvz) in patients with amyotrophic lateral sclerosis (ALS).

The primary endpoint of the 350-subject study will be the change from baseline in a scale called ALSFRS-R.

Ravulizumab is a complement inhibitor currently approved in the U.S. for atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria.

https://seekingalpha.com/news/3531701-alexion-to-launch-study-of-ultomiris-in-als