Top Stories for Week of September 7, 2020

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Pfizer sees data for COVID-19 vaccine’s efficacy by end of October.

Amid the frantic race for a COVID-19 vaccine, Pfizer may have early efficacy results in just a matter of weeks. During a digital event Thursday, Pfizer CEO Albert Bourla said the company has enrolled about 23,000 people for its Phase 3 coronavirus vaccine trial so far. The company expects initial results in late October, according to FiercePharma.

If the results are positive, the company would be ready to ask the FDA to authorize vaccinations as soon as possible. In fact, the company is already preparing its application to submit quickly if the vaccine shows promise, he added. The FDA has set an advisory panel meeting for October 22 to discuss COVID-19 vaccine progress. In October, “the truth will be revealed,” Bourla said in a Washington Post interview.

Bourla’s timeline would have been unthinkable at the beginning of the year, when the novel coronavirus started spreading and researchers got to work on the first vaccine candidates. Moderna Inc. (Cambridge MA) entered the clinic in record time, while partners Pfizer and BioNTech SE (Mainz DEU) started US human testing in early May.

At the start of the R&D process, experts predicted a vaccine could be available in 12 to 18 months if all went smoothly. Now, experts are still skeptical that a vaccine could be ready in late October. As the programs raced ahead, Americans grew wary about getting a shot developed and approved in a short time.

In a recent survey conducted by Stat and Harris Poll, more than 80% of people responded that they’d worry about safety for a hastily approved vaccine. Nearly 80% see politics driving the approval process rather than science. After controversial decisions by the FDA on hydroxychloroquine and convalescent plasma, some medical experts have raised concerns over politics entering science.

One prominent physician, Dr. Eric Topol, last week called on FDA commissioner Stephen Hahn to “tell the truth or resign” after the plasma move. Trump and allies have said they’re pushing for a vaccine by the end of the year or earlier, apparently seeking a political boost from COVID immunizations.

Bourla’s briefing comes right after news that the Centers for Disease Control and Prevention is prepping for possible vaccine deliveries by November 1. In a note to states the previous week, CDC director Robert Redfield asked them to consider waiving restrictions to ensure vaccine distribution sites could be up and running in a matter of months. All of this comes as the US election is just two months away.

Still, in speaking to CNN on Wednesday, NIH director Francis Collins said it remains “unlikely” a vaccine will be ready in late October. Dr. Anthony Fauci said the same on Thursday, but he said it’s not impossible. Meanwhile, a group of biotech CEOs last week wrote that COVID data releases should only come through academic conferences or peer-reviewed journals, not press releases. It’s unclear how long that process–or a potential FDA review–would take under the circumstances.

Separately, according to Johns Hopkins Medical, as of Saturday, Sept. 5, there were nearly 26.7 million COVID-19 global cases confirmed, and some 876,000 deaths. Confirmed cases in the US totaled 6.2 million with over 188,000 deaths. Both of the US figures comprise 23% and 21%, respectively, of the global totals. The US population is about 4% of the global total.

COVID-19 Addenda: Russia’s “Sputnik-V” COVID-19 vaccine produced an antibody response in all participants in early-stage trials, according to results published on Friday by The Lancet medical journal that were hailed by Moscow as an answer to its critics. The results of the two trials, conducted in June-July this year and involving 76 participants, showed 100% of participants developing antibodies to the new coronavirus and no serious side effects, The Lancet said.

Russia licensed the two-shot jab for domestic use in August, the first country to do so and before any data had been published or a large-scale trial begun.

“The two 42-day trials–including 38 healthy adults each–did not find any serious adverse effects among participants, and confirmed that the vaccine candidates elicit an antibody response,” The Lancet said. “Large, long-term trials including a placebo comparison, and further monitoring are needed to establish the long-term safety and effectiveness of the vaccine for preventing COVID-19 infection,” it said.

Elsewhere, results of a meta-analysis published last Wednesday in JAMA suggest that the use of systemic corticosteroids in critically ill patients with COVID-19 is associated with lower 28-day all-cause mortality compared to usual care or placebo. Study author Jonathan Sterne noted that the benefit was seen regardless of age, sex or how long patients had been ill.

“Steroids are a cheap and readily available medication, and our analysis has confirmed that they are effective in reducing deaths amongst the people most severely affected by COVID-19,” Sterne remarked.

FirstWord Pharma reports that the latest findings follow results from the RECOVERY study released in June which found that low-dose dexamethasone reduced the risk of death by up to one third in hospitalized patients with severe respiratory complications of COVID-19. When broken down by treatment, dexamethasone produced the strongest benefit with a 36% drop in deaths compared with usual care or placebo, followed by hydrocortisone and methylprednisolone, which reduced mortality by 31% and 9%, respectively.

With COVID-19 vaccine propaganda intensifying each day—including last week, with new comments from Pfizer Inc.’s (NYC) CEO–the head of the US government’s research program said it’s “extremely unlikely” a vaccine will be available in early November. Moncef Slaoui, the Operation Warp Speed head, told NPR it remains “extremely unlikely, but not impossible” a vaccine will be ready by early November.

Operation Warp Speed, a multibillion-dollar research program aimed at delivering 300 million doses of a COVID-19 vaccine by January, is performing “even better than I was hoping,” he said. With that, “there is a very, very low chance that the trials that are running as we speak could read before the end of October” and help score an emergency use authorization or approval.

Slaoui’s comments diverge from those of Pfizer CEO Albert Bourla, who last week said his company expects enough data in late October to make a decision about an FDA submission. In fact, the drug giant is already preparing its submission in case the vaccine shows early efficacy in the trial, Bourla said.

President Trump last week cast doubt on the value of Anthony Fauci to the White House coronavirus task force, saying in an interview with Fox News that he “inherited” the government’s top infectious disease specialist.

“I disagree with a lot of what he said,” Trump told Laura Ingraham when asked if he would put Fauci “front and center” in the pandemic response if he could do it again. “I get along with him, but every once in a while, he’ll come up with one that I say, ‘Where did that come from?’” Trump continued. “I inherited him. He was here. He was part of this huge piece of machine.”

The president reiterated his claim that Fauci opposed his decision to restrict travel from China in January. Fauci indicated at the time he did not think it was a good idea, though he later said it had bought the US time to fight the virus. Trump has repeatedly undermined and criticized Fauci. Last month, he retweeted a message that said Fauci “has misled the American public on many issues, but in particular, on dismissing #hydroxychloroquine and calling Remdesivir the new gold standard.” He also has said Fauci is a nice man but has “made a lot of mistakes.” (Ref: The Hill)

And a panel of the US National Institutes of Health (NIH) concluded that “there are insufficient data to recommend either for or against the use of convalescent plasma for the treatment of COVID-19.” The review by the COVID-19 Treatment Guidelines Panel comes shortly after the FDA issued an emergency-use authorization (EUA) for convalescent plasma to treat hospitalized patients with COVID-19 amid concerns by some senior health officials that emerging data for the treatment were too weak. The panel said it reviewed all available evidence from published and unpublished data on convalescent plasma for the treatment for COVID-19, including the FDA analyses that supported the EUA.

Based on the review, the panel determined that convalescent plasma should not be considered standard of care for the treatment of patients with COVID-19, echoing comments made by FDA chief scientist Denise Hinton when the EUA was announced. The NIH panel found no difference in seven-day survival overall between patients who received convalescent plasma with high titers of SARS-CoV-2 neutralizing antibodies and those who received plasma with low titers.

Sanofi, GlaxoSmithKline start Phase 1/2 study of COVID-19 vaccine candidate.

Vaccine giants Sanofi SA (Paris) and GlaxoSmithKline PLC (London) are joining the ranks of COVID-19 vaccine makers testing their candidates in people, launching a large Phase 1/2 clinical trial Thursday that will take place at 11 sites across the US. The trial, which is expected to be completed by early December, would pave the way for a pivotal Phase 3 efficacy trial to start the same month, if the experimental vaccine proves to be safe, tolerable, and appears to be generating enough of an immune response to proceed, says STAT News.

Sanofi, which is the lead partner, intends to enroll more than 400 people in the Phase 1/2, an unusually large number for a first-in-human trial of a vaccine. In the first phase of the trial, healthy subjects 18 to 49 years of age will be vaccinated to establish the correct dose for the vaccine. Later, a broader group of participants, including at least 140 adults aged 50 and older, will be enrolled.

“We think we can move very rapidly to expansion from the very first subjects that get the vaccine,” John Shiver, Sanofi’s global head of vaccines research and development, told STAT.

While several other experimental COVID-19 vaccines are already in Phase 3 trials, this candidate is the first outside of China to use a vaccine approach for which there is already a licensed vaccine. The approach used is the same as that used for Sanofi’s Flublok vaccine. The Sanofi vaccine is combined with an adjuvant–a compound that enhances the immune response to the vaccine–produced by GSK. Like most of the COVID-19 vaccines currently in production, it will be a two-dose vaccine.

The three candidates already in Phase 3 trials in the US use vaccine platforms that have not yet produced licensed vaccines. Moderna and Pfizer use a messenger RNA approach, while a vaccine being developed by Oxford University and AstraZeneca uses a harmless adenovirus onto which genetic material from the SARS-CoV-2 virus–which causes COVID-19–has been fused. The adenovirus, which doesn’t trigger illness, induces the immune system to generate a protective response to SARS-2. Unlike the mRNA vaccines, which need to be transported and stored frozen, the Sanofi vaccine will be shipped in liquid form and can be stored at refrigerator temperatures, Shiver said. The Pfizer vaccine must be stored at -70 Celsius, which will complicate delivery and distribution.

Sanofi is also working on a second vaccine, an mRNA vaccine, which it is making in partnership with Translate Bio Inc. (Lexington MA). Human trials with this vaccine are expected to start in November. It is possible one or more vaccines could already be licensed or at least in use under an Emergency Use Authorization before either of Sanofi’s vaccines begins its Phase 3 trial. But Shiver seemed unfazed.

“We’re not that far behind,” he said, noting the global need for COVID-19 vaccines cannot be filled by a few vaccine manufacturers. “The threat of this virus and pandemic are large enough that the world needs multiple types of approaches for it.”

Abbott inks $760 million HHS contract for rapid COVID-19 testing kits.

Five Department of Defense facilities will participate in the Phase 3 trial of a COVID-19 vaccine, the Pentagon announced Thursday. According to the Department of Defense, researchers are still seeking volunteers for the next phase of testing for AZD1222, a COVID-19 vaccine candidate under development by AstraZeneca PLC (London). UPI reports that the upcoming trial will take place at Naval Medical Center in San Diego, Joint Base San Antonio, Wilford Hall Ambulatory Surgical Center in San Diego, Walter Reed National Military Medical Center in Bethesda, MD, and Fort Belvoir Community Hospital in Fort Belvoir, VA.

“The Department of Defense continues to play a key role in the development of a potential COVID-19 vaccine,” Tom McCaffery, assistant secretary of defense for health affairs, said. “Now that vaccines have passed the first phases of testing for safety, dosing and response, we are ready to move into the next phase where volunteers are needed to join large clinical studies,” McCaffery added.

AZD1222 is one of several vaccine candidates under development under Operation Warp Speed, the US federal government’s effort to create and distribute a vaccine against the coronavirus that causes COVID-19. The vaccine is the result of a partnership between AstraZeneca and Oxford Vaccine Group, with funding from the Biomedical Advanced Research and Development Authority, a branch of the US Department of Health and Human Services, as well as the British government.

The trial is assessing efficacy and safety of AZD1222 in all participants, while local and systemic reactions and immune responses will be assessed in  3,000 people. The study joins ongoing late-stage trials in the UK, Brazil and South Africa, with further studies planned to start in Japan and Russia. The Phase 3 program for the vaccine, which is being co-developed with the University of Oxford, will enroll a total of up to 50,000 participants globally with results expected later this year.

In July, interim results from the ongoing Phase 1/2 COV001 trial were published in The Lancet showing that AZD1222 was tolerated and generated robust immune responses against the SARS-CoV-2 virus in all evaluated participants. Mene Pangalos, EVP of biopharmaceuticals R&D at AstraZeneca, remarked “we are pleased that AZD1222 demonstrated safety and immunogenicity across all adult age groups.”

IPO Sector: Included among recent SEC filings for initial public offerings, Orphazyme A/S, a Danish late-stage biotech developing protein therapies for rare neurodegenerative diseases, registered up to $115 million worth of common stock The company is currently listed on the Nasdaq Copenhagen under the symbol “ORPHA.” Orphazyme is developing heat shock proteins to develop novel therapies for neurodegenerative orphan diseases. The company submitted an NDA for product candidate arimoclomol with the FDA for Niemann-Pick disease Type C (NPC) in July 2020, and it plans to submit a marketing authorization application to the EMA in the 2H20. In its Phase 2/3 trial of arimoclomol in NPC, the company observed evidence of slowing of disease progression. The Copenhagen-based company was founded in 2009 and plans to list on the Nasdaq under the symbol “ORPH.” BofA Securities, Cowen and Guggenheim Securities are the joint bookrunners on the deal. No pricing terms were disclosed.

Elsewhere, Taysha Gene Therapies Inc., an early stage biotech developing AAV-based gene therapies for CNS disorders, filed with the SEC to raise up to $100 million in an IPO. Founded in partnership with The University of Texas Southwestern Medical Center (UT Southwestern), Taysha Gene Therapies is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations. One of its most advanced candidates, TSHA-101, is being developed for the treatment of GM2 gangliosidosis. The company plans to initiate a Phase 1/2 trial for TSHA-101 under a CTA in Canada by the end of 2020. In addition, it plans to submit INDs for four additional programs to the FDA by the end of 2021. The Dallas, TX-based company was founded in 2019 and plans to list on the Nasdaq under the symbol “TSHA.” Goldman Sachs, Morgan Stanley, Jefferies, and Chardan Capital Markets are the joint bookrunners on the deal. No pricing terms were disclosed.

Lixte Biotechnology Holdings Inc., a Phase 2 biotech using biomarker technology to develop protein inhibitors for cancer, filed on Thursday with the SEC to raise up to $11 million in an IPO. The company is currently listed on the OTC under the symbol “LIXT.” The company has developed two series of pharmacologically active drugs, the LB-100 series and the LB-200 series. The LB-100 series targets several types of cancer, with potential for vascular and metabolic diseases. The LB-200 series may be useful for the treatment of chronic hereditary diseases. The company completed a Phase 1 trial of LB-100 demonstrating antitumor activity in humans and is currently in a Phase 1b/2 trial, with results expected in 2023. The East Setauket, NY-based company was founded in 2005 and plans to list on the Nasdaq under the symbol “LIXT.” WestPark Capital is the sole bookrunner on the deal.

PainReform Ltd., a Phase 2 Israeli biotech developing therapies for post-operative pain relief, raised $20 million by offering 2.5 million units at $8, the low end of the range of $8 to $10. Each unit consists of one share of common stock and one warrant, exercisable at $8.80. The company offered 50,000 less units than anticipated. It originally planned to offer 2.6 million shares at the same range before revising its offering to units. Herzliya, Israel-based PainReform was founded to focus on the reformulation of existing therapeutics using its proprietary extended release drug delivery system. Management is led by Acting CEO and Chief Technology Officer Prof. Eli Hazum, who has been with the firm since 2012 and was previously Head of the Department of Receptor Research and Metabolic Diseases at Glaxo. PainReform lists on the Nasdaq under the symbol “PRFX.” Maxim Group LLC and Joseph Gunnar acted as lead managers on the deal. Shares closed the week off 16% at $6.73.

And COMPASS Pathways PLC, a British Phase 2 biotech developing a psilocybin-based therapy for depression, filed on Friday with the SEC to raise up to $100 million in IPO. The company is focusing on using its proprietary formulation of psilocybin, COMP360, in conjunction with psychological support as a way to help individuals who have treatment-resistant depression, or TRD, a subset of major depressive disorder, or MDD. COMPASS is currently evaluating COMP360 in conjunction with psychological support in a Phase 2b trial and plans to report data from this trial in late 2021. The Cheshire, UK-based company was founded in 2015 and plans to list on the Nasdaq under the symbol “CMPS.” COMPASS Pathways filed confidentially on July 2, 2020. Cowen, Evercore ISI and Berenberg are the joint bookrunners on the deal. No pricing terms were disclosed.

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