Top Stories for Week of August 24, 2020

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Top FDA official in charge of overseeing new COVID-19 vaccines says he’ll resign if agency is pressured into a premature approval.

A top US health regulator who will help decide the fate of a coronavirus vaccine has vowed to resign if the Trump administration approves a shot before it is shown to be safe and effective, Reuters has learned. Peter Marks, director of the Food and Drug Administration’s Center for Biologics Evaluation and Research, made the statement in response to concerns raised on a conference call late last week of government officials, pharmaceutical executives and academics who serve on a vaccine working group organized by the National Institutes of Health, according to three sources familiar with the matter.

When contacted by Reuters, Marks confirmed the account. Scientists, public health officials and lawmakers are worried that the Trump administration will pressure the FDA to authorize a COVID-19 vaccine in advance of the November presidential election, even if data from clinical trials do not support its widespread use.

Marks told Reuters he has not faced any political pressure and that the FDA would be guided by science alone.

Should that change, “I could not stand by and see something that was unsafe or ineffective that was being put through,” Marks said. “You have to decide where your red line is, and that’s my red line,” he said. “I would feel obligated (to resign) because in doing so, I would indicate to the American public that there’s something wrong.”

He added that he would equally object if someone sought political gain by holding up approval of a vaccine that was shown to work, and that was safe. Michael Caputo, assistant secretary for public affairs at the Department of Health and Human Services, which oversees FDA and NIH, said the government aims to identify a safe and effective vaccine by January 2021.

Speculation about the FDA approving a vaccine under political pressure “only undermines confidence in the public health system,” Caputo said in a statement. “I’ve never met one FDA regulator who wouldn’t resign over improper pressure, and that’s how America knows their seal of approval is the gold standard.”

The FDA declined to comment on the meeting. President Donald Trump’s approval ratings have fallen sharply in the wake of a pandemic that has killed more than 176,000 Americans and infected over 5.6 million. The race to produce a vaccine has become the centerpiece of his administration’s response.

Earlier this month, Trump said a vaccine was possible before the Nov. 3 vote. The FDA has scheduled a meeting of its advisory committee of outside experts on coronavirus vaccines on Oct. 22.

Separately, according to Johns Hopkins Medical, as of Saturday, Aug. 22, there were over 23 million COVID-19 global cases confirmed, and some 801,000 deaths. Confirmed cases in the US totaled 5.6 million with over 176,000 deaths. Both of the US figures comprise 24% and 22%, respectively, of the global totals. The US population is about 4% of the global total.

COVID-19 Addenda: Moderately ill COVID-19 patients saw their condition improve after a 5-day course of Gilead Sciences Inc.’s (Foster City CA) remdesivir, but the drug did not significantly shorten hospital stays and a 10-day course did not show a benefit, according to new data. It’s currently sold under an emergency use authorization from the US Food and Drug Administration.

The drug, which was shown in a trial of severely ill COVID-19 patients to shorten their hospital recovery time, has been at the forefront of the battle against the pandemic. The 600-patient analysis, published on Friday by the Journal of the American Medical Association, found that moderately ill patients treated with the antiviral drug for up to 5 days had significantly higher odds of improvement in certain areas but the clinical importance of the benefit for those patients was uncertain.

Elsewhere, the failure of California’s infectious disease monitoring system for a stretch of at least 20 days in July and August triggered potentially deadly fallout that continues to reverberate across the state, says Kaiser Health News. The fallout has been most severe in heavily populated counties, which rely primarily on a statewide electronic information system to guide their pandemic response. Local health departments couldn’t clearly see where the coronavirus was spreading, dramatically slowing their efforts to trace and track new infections–leading to more death and disease, public health officials said.

“By the time you get those cases, even if you do your best to trace the contacts, you might be too late,” said David Campos, deputy Santa Clara County executive. “Individuals who were positive didn’t know they were positive, so they ended up spreading the virus to other people unknowingly.”

Mass testing of Russia’s first potential COVID-19 vaccine to get domestic regulatory approval will involve more than 40,000 people and will be overseen by a foreign research body when it starts next week, backers of the project said on Thursday. These were the first details on the shape and size of the upcoming late-stage trial of the vaccine given by its developers, who are aiming to allay concerns among some scientists about the lack of data provided by Russia so far.

The vaccine, called “Sputnik V” in homage to the world’s first satellite launched by the Soviet Union, has been hailed as safe and effective by Russian authorities and scientists following two months of small-scale human trials, the results of which have not been made public yet. But Western experts have been more skeptical, Reuters reports, warning against its use until all internationally approved testing and regulatory steps have been seen to be taken and proved a success.

The FDA has placed on hold an emergency authorization for the use of plasma as a COVID-19 treatment following the intervention of top federal health officials who claimed the available data were too weak, according to a report in The New York Times. The National Institute of Allergy and Infectious Diseases (NIAID) clinical director H. Clifford Lane confirmed the hold to the Times, adding plasma may still be authorized sometime in the future.

Much has been made about the prospect of utilizing donated plasma from recovered COVID-19 patients to treat those currently infected with the disease. The theory goes that antibodies in the plasma can help reduce the time patients spend in hospitals as well as mortality rates if the treatment is administered quickly. One of the biggest studies into adapting the technique for COVID-19 came from the Mayo Clinic the prior week, which showed statistically significant improvements in mortality rates among the most severe cases.

And there’s new evidence suggesting that the White House is about to promote a new, unproven and fuzzy coronavirus treatment, CNN reports. Mike Lindell, the CEO of Minnesota-based MyPillow and an enthusiastic Trump supporter, says so. He told CNN he took part in a July meeting with President Trump to discuss oleandrin as a potential treatment for coronavirus. But such early enthusiasm for the compound, made using a toxic shrub, is not only strange, but disturbing, three infectious disease specialists told CNN. It’s only been tested in laboratory dishes and is very unlikely to end up as a treatment for the infection, they said.

“This is really just nonsense and a distraction,” Dr. Jonathan Reiner, CNN medical analyst and a professor of medicine at George Washington University, told CNN’s Anderson Cooper.

Reiner likened it to President Trump’s previous support of hydroxychloroquine, now shown to be unhelpful in treating coronavirus.

J&J inks $6.5 billion deal for autoimmune disease specialist Momenta.

Johnson & Johnson (New Brunswick NJ) agreed to pay $6.5 billion in cash to acquire Momenta Pharmaceuticals Inc. (Cambridge MA), a maker of autoimmune-disease drugs, in the largest pharmaceutical- industry merger this year. Though the price tag falls short of some of the industry-shifting takeovers of recent years, the deal is the latest sign drug companies are looking for ways to bulk up even as the coronavirus pandemic upends other businesses, Bloomberg reports.

At $52.50 a share, the deal represents a premium of more than 70% to Momenta’s closing price prior to the deal’s announcement. The biotechnology company had seen its shares surge 56% this year after encouraging clinical-trial data for its treatment for a rare blood disorder that affects fetuses and newborns. That treatment has received an orphan-drug designation from the US Food and Drug Administration.

Autoimmune diseases have been an area of increasing interest for big drugmakers. Earlier last week, French drugmaker Sanofi SA (Paris) agreed to acquire US biotech company Principia Biopharma Inc. (South San Francisco) for about $3.4 billion. The all-cash deal gives Sanofi treatments for multiple sclerosis and a range of other disorders. Drugmakers weren’t shielded from disruptions caused by the outbreak of COVID-19 in the US early this year.

A sharp decline in elective medical procedures helped curb demand for cancer drugs and other high-priced therapies that have become the industry’s main profit engines in recent years. However, many pharmaceutical executives indicated during the most recent earnings season that they believe the most serious effects of the pandemic have passed. And investors have been eager to snap up stocks of drugmakers, especially smaller biotech companies that could become takeover targets.

J&J said in a statement that acquiring Momenta would bolster its Janssen Pharmaceutical unit’s leadership in immune-mediated diseases. Momenta said in June that its experimental drug nipocalimab hit its primary goal in a Phase 2 trial as a treatment for myasthenia gravis, an autoimmune neuromuscular disorder. Stifel analyst Derek Archila wrote in a note to clients that it is unlikely other bidders will emerge for Momenta. Shares of Momenta closed the week up 80% at $52.32, while J&J shares rose 3% to $152.76.

Pfizer, BioNTech spell out Phase 3 priority for COVID-19 vaccine candidate.

Shares of Pfizer Inc. (NYC) and US-listed shares of partner BioNTech SE (Mainz DEU) rose last week after they released additional data for their experimental coronavirus vaccine and reiterated they were on track to seek a regulatory review in October. Pfizer has said repeatedly since June that it was targeting October for its FDA application and the companies started a large late-stage study last month of the candidate vaccine, one of the few globally in later stages of development. Reuters reports that on Thursday they reported data from previous early-stage trials of the vaccine, BNT162b2, that showed it induced similar immune responses and had milder side effects than prior data on another candidate.

“In both younger and older adults, the two vaccine candidates elicited similar dose-dependent SARS−CoV−2−neutralizing geometric mean titers (GMTs), comparable to or higher than the GMT of a panel of SARS−CoV−2 convalescent sera,” the team wrote. “BNT162b2 was associated with less systemic reactogenicity, particularly in older adults,” they added.

Specifically, no older adults in the BNT162b2 groups reported redness or swelling, although they experienced the expected injection site pain. Systemic reactions such as fever were milder in response to BNT162b2 than to BNT162b1, and even though some younger patients reported fatigue, headache, chills, muscle pain and joint pain, none of the older BNT162b2 recipients did.

“Overall, systemic events reported by 65 to 85-year-olds who received BNT162b2 were similar to those reported by those who received placebo after Dose 1,” investigators wrote.

Pfizer stock, down 1% this year, has trailed the glittering rise in value of major pharmaceutical companies this year. Moderna Inc. (Cambridge MA) and Britain’s AstraZeneca PLC are among those to have moved into late-stage trials. Pfizer shares closed the week up 2% to $38.88 while BioNTech gained 6% to $73.02.

IPO Sector: Outset Medical Inc., which provides a hemodialysis system for kidney diseases, filed on Friday with the SEC to raise up to $100 million in an initial public offering. The company’s Tablo Hemodialysis System is designed as a single enterprise solution that can be utilized across the continuum of care. To date, the company has focused primarily on the acute care setting, for which Tablo was cleared in 2014. It is in the early stages of commercializing in the home market, for which the device was cleared in March 2020. The San Jose, CA-based company was founded in 2003 and booked $29 million in sales for the 12 months ended June 30, 2020. It plans to list on the Nasdaq under the symbol “OM.” BofA Securities and Morgan Stanley are among the joint bookrunners on the deal. No pricing terms were disclosed.

Elsewhere, Universe Pharmaceuticals Inc., a Chinese pharmaceutical specializing in traditional Chinese medicine derivatives, registered up to $30 million in an IPO. The Jiangxi, China-based company plans to offer 5 million shares at a price range of $5 to $7. At the midpoint of the proposed range, Universe Pharmaceuticals would command a market value of $126 million. The company manufactures, markets, and distributes traditional Chinese medicine derivative (TCMD) products targeting the elderly. It currently produces 13 varieties of TCMD products, which are sold in approximately 249 cities and 30 provinces in China. Universe Pharmaceuticals was founded in 1998 and booked $31 million in revenue for the 12 months ended March 31, 2020. It plans to list on the Nasdaq under the symbol “UPC.” Univest Securities is the sole bookrunner on the deal.

Kymera Therapeutics Inc., a preclinical biotech developing protein degraders for oncology and immunology, raised $174 million by offering 8.7 million shares at $20, above the range of $16 to $18. The Watertown, MA-based company uses its proprietary targeted protein degradation platform, Pegasus, to discover highly selective small molecule protein degraders with potent activity against disease-causing proteins throughout the body. Kymera expects to file an IND for KT-474, an orally bioavailable IRAK4 degrader, in the 1H 2021. If approved, the company will initiate a Phase 1 trial in adult healthy volunteers and hidradenitis suppurativa and atopic dermatitis patients. Kymera was founded in 2015 and booked $9 million in collaboration revenue for the 12 months ended June 30, 2020. It lists on the Nasdaq under the symbol “KYMR.” Morgan Stanley, BofA Securities and Cowen are among the joint bookrunners on the deal. Shares closed the week up 66% at $32.26.

Inhibrx Inc., a Phase 1 biotech developing single domain antibodies for cancer and rare diseases, raised $119 million by offering 7 million shares at $17, the midpoint of the range of $16 to $18. The company offered 1 million more shares than anticipated. The La Jolla, CA-based company plans to raise $102 million by offering 6 million shares at a price range of $16 to $18. At the midpoint of the proposed range, Inhibrx would command a fully diluted market value of $620 million. The company’s lead candidate INBRX-109, is a tetravalent death receptor 5, or DR5, agonist being evaluated in patients diagnosed with chondrosarcoma and mesothelioma, two difficult-to-treat cancers. INBRX-109 is currently in a Phase 1 trial and has enrolled 75 patients to date. Inhibrx was founded in 2017 and booked $6 million in revenue for the 12 months ended June 30, 2020. It lists on the Nasdaq under the symbol “INBX.” Jefferies, Evercore ISI and Credit Suisse are the joint bookrunners on the deal. Shares closed the week up 20% at $20.43.

Harmony Biosciences Holdings Inc., a commercial stage biotech developing therapies for narcolepsy, raised $128 million by offering 5.3 million shares above the proposed range at $24. At IPO, Harmony Biosciences raised 28% more in proceeds than anticipated. The Plymouth Meeting, PA-based company’s sole marketed product, WAKIX (pitolisant), was approved for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019. The company expects to initiate a Phase 3 trial for EDS and cataplexy in pediatric patients in the 2H21. It also plans to begin Phase 2 trials for EDS and other key symptoms in patients with Prader-Willi Syndrome in the 2H20 and in adult patients with myotonic dystrophy in the 1H21. Harmony Biosciences was founded in 2017 and booked $64 million in net product revenue for the 12 months ended June 30, 2020. It lists on the Nasdaq under the symbol “HRMY.” Goldman Sachs, Jefferies and Piper Sandler are the joint bookrunners on the deal. Shares closed the week up 50% at $36.

Nano-X Imaging Ltd., which is developing affordable medical imaging systems, raised $165 million by offering 9.2 million shares at $18, the high end of the range of $16 to $18. The Neve Ilan, Israel-based company had previously filed to offer 5.9 million shares at the same range. Insiders intend to purchase $80 million worth of shares in the offering. The company is developing lower cost medical imaging technology which it believes can achieve the same functionality as legacy X-ray imaging. Nano-X’s Nanox System is comprised of its Nanox.ARC hardware, which uses a microelectromechanical system semiconductor cathode as opposed to X-ray analog cathodes, and its cloud-based imaging software Nanox.CLOUD. Nano-X Imaging was founded in 2012 and lists on the Nasdaq under the symbol “NNOX.” Cantor Fitzgerald, Oppenheimer & Co., Berenberg and CIBC are the joint bookrunners on the deal. Shares closed the week up 21% at $21.70.

And Sun BioPharma Inc., a clinical-stage biotech developing small molecule therapies for pancreatic cancer, announced terms for its IPO on Thursday. The Waconia, MN-based company plans to raise $10 million by offering 1.1 million shares at $8.75, the last close of its shares on the OTCQB (SNBP). The company is also offering warrants to purchase 1.1 million shares at an assumed exercise price of $10.94 per share (125% of assumed offer price). Insiders intend to purchase $50,000 worth of shares and warrants in the offering. At the proposed price, Sun BioPharma would command a fully diluted market value of $88 million.  Sun BioPharma was founded in 2011 and plans to list on the Nasdaq under the symbol “SNBP.” Craig-Hallum Capital Group is the sole bookrunner.

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