Top Stories for Week of September 28, 2020

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J&J presents Phase 1/2a data indicating single-dose vaccine provokes strong immune response.

A single dose of Johnson & Johnson’s (New Brunswick NJ) experimental COVID-19 vaccine produced a strong immune response against the novel coronavirus in an early-to-mid stage clinical trial, according to interim results published on Friday. The vaccine, called Ad26.COV2.S, was equally well-tolerated at two different doses, the results showed.

A single shot, versus a rival two-dose approach being tested by Moderna Inc. (Cambridge MA) and Pfizer Inc. (NYC), could simplify distribution of the vaccine, according to Reuters. However, it is unclear whether elderly people, one of the populations most at risk from the virus, will be protected to the same degree as younger people with the J&J vaccine. The trial in close to 1,000 healthy adults, which is backed by the US government, began after the J&J vaccine was found in July to offer strong protection in a single dose to monkeys. Based on the current results, J&J last Wednesday kicked off a final 60,000-person trial, which could pave the way for an application for regulatory approval. J&J said it expects results of that so-called Phase 3 trial by the end of the year or early next year.

The results, released on the medical website medRxiv, have not been peer-reviewed. Researchers, including those from J&J’s unit Janssen Pharmaceuticals, said 98% of participants with data available for the interim analysis had neutralizing antibodies, which defend cells from pathogens, 29 days after vaccination. However, immune response results were available from only a small number of people–15 participants–over 65 years old, limiting the interpretation. In participants older than 65, the rate of adverse reactions such as fatigue and muscle aches was 36%, much lower than the 64% seen in younger participants, the results showed, suggesting the immune response in older people may not be as strong.

The researchers said more details on safety and effectiveness will follow when the study is completed. For now, the results justify why more studies are needed in larger numbers to look for serious adverse effects, Dr. Barry Bloom, a professor at Harvard T.H. Chan School of Public Health who was not involved in the J&J trial, told Reuters. “Overall, the vaccine is doing what you would expect it to do if you were to move it to Phase 3 trials,” Bloom said.

Separately, according to Johns Hopkins Medical, as of Saturday, Sept. 26, there were 32.6 million COVID-19 global cases confirmed, and some 990,000 deaths. Confirmed cases in the US totaled over 7 million with 204,000 deaths. Both of the US figures comprise 21.5% and 20.6%, respectively, of the global totals.

COVID-19 Addenda: Anthony Fauci, MD, says talk about a second wave of the coronavirus is premature because the US is still dealing with the first one. The idea of a second wave is based on the 1918 flu pandemic, when many cases were seen in the spring, he says. The spring cases “literally disappeared” and were followed by a spike in flu cases in the fall, he told CNN’s Sanjay Gupta, MD, in an online conversation organized by Emory University.

“Rather than say, ‘A second wave,’ why don’t we say, ‘Are we prepared for the challenge of the fall and the winter?’” said Fauci, director of the National Institute of Allergy and Infectious Diseases and a member of the White House coronavirus task force.

Flu shots are an important measure to help the US get through the winter, he said. He and other healthcare professionals have observed that the Southern Hemisphere has had a very light flu season, probably because measures to curb the coronavirus, such as social distancing and mask-wearing, have limited the spread of flu.

Elsewhere, Fugifilm Holdings Corp. (Tokyo) announced last Wednesday that a Phase 3 study of Avigan (favipiravir) in COVID-19 patients with non-severe pneumonia met its primary endpoints. The trial, which compared Avigan to placebo, both in addition to standard treatment, was conducted in Japan and began in March. The study’s primary efficacy endpoint is time to negative conversion of detectable SARS-CoV-2 viral RNA in RT-PCR assays, and to alleviation of symptoms, including body temperature, oxygen saturation and chest images.

Results from an analysis of 156 individuals showed that participants randomised to receive Avigan had a significantly shorter time to resolution, with those given the antiviral improving after a median of 11.9 days compared to 14.7 days for placebo. Fujifilm noted that no new safety concerns were seen in the trial, adding that it will conduct a detailed analysis of the data and work to seek approval in this indication as early as October. Avigan, which selectively inhibits RNA polymerase necessary for influenza virus replication, is currently approved in Japan for the treatment of influenza.

US President Donald Trump indicated that the White House could block plans by the FDA to release tougher standards for granting emergency-use authorisation (EUA) of eventual COVID-19 vaccines, claiming that such a move by the agency appears politically motivated. A recent news report said the FDA was expected to unveil stricter EUA guidelines for COVID-19 vaccines, possibly this week, that would make it unlikely one could be authorised before the US elections in early November.

The FDA previously said a coronavirus vaccine needs to be at least 50% effective in order for the agency to consider granting approval. According to the new report published earlier last week, the FDA is now expected to tell vaccine developers that, before applying for an EUA, they must monitor participants in late-stage clinical trials for at least two months, starting after volunteers receive a second vaccine dose. The agency is also looking for at least five severe cases of COVID-19 in the placebo group for each trial, as well as some cases of the disease in older people, as a sign the vaccine works.

And problems with the body’s inborn response to infection may explain severe illness and death from the coronavirus in roughly 14% of patients, according to two studies published on Thursday in the journal Science. These problems occur more often in men than in women, offering a potential explanation for why the virus seems to affect men more severely. Both studies focused on Type I interferon, a set of 17 proteins that appear when the body is confronted with a virus.

Interferons are produced within hours of exposure and signal to the rest of the immune system that there is an intruder. Genetic flaws in some people undermine the Type 1 interferon response, according to one study. The virus provokes the production of “auto-antibodies”–molecules that misguidedly attack and destroy Type I interferons, instead of the virus, which buys the virus crucial time to gain a foothold and wreak havoc. The researchers found auto-antibodies in 101 of 987 people with severe COVID-19, but in none of the 663 people with mild or asymptomatic illness. The auto-antibodies were seen predominantly in men: 95 of the 101 patients in the study were men.

Novavax starts Phase 3 coronavirus vaccine trial in UK.

Novavax Inc.’s (Gaithersburg MD) stock price surged Friday after the biotech firm started a key late-stage trial of its experimental coronavirus vaccine. MarketWatch said company shares jumped 5% on the week to $113.56 following its Thursday announcement that it kicked off the Phase 3 study in the United Kingdom, which is battling a surge in COVID-19 infections. The trial aimed at proving whether Novavax’s vaccine is safe and effective will enroll up to 10,000 people over the next four to six weeks, the company said. The participants will range in age from 18 to 84 years old, with half getting two shots of the vaccine, known as NVX-CoV2373, and the other half receiving a placebo.

“With a high level of [coronavirus] transmission observed and expected to continue in the UK, we are optimistic that this pivotal Phase 3 clinical trial will enroll quickly and provide a near-term view of NVX-CoV2373’s efficacy,” Dr. Gregory M. Glenn, Novavax’s president of research and development, said in a statement.

Data from the trial will support Novavax’s applications for regulatory approval in the UK, the European Union and other countries, Glenn added.

Novavax said it plans to publish the study’s full protocol in the coming days. Phase 3 trials are currently underway in the US and Europe for four other potential coronavirus vaccines, including candidates from AstraZeneca PLC (London), Pfizer Inc. (NYC) and Johnson & Johnson (New Brunswick NJ). Pfizer and Moderna Inc. have said they could know by October or November whether their vaccines work, but US officials don’t expect a shot to be widely available until next year.

China’s Sinovac expects interim final-stage trial data on coronavirus vaccine this year.

A Chinese pharmaceutical company said Thursday the coronavirus vaccine it is developing should be ready by early 2021 for distribution worldwide. Yin Weidong, the CEO of Sinovac Biotech Co. Ltd. (Beijing), said he personally has been given the experimental vaccine according to euronews.

“At the very beginning, our strategy was designed for China and for Wuhan. Soon after that in June and July we adjusted our strategy, that is to face the world,” Yin said, referring to the Chinese city where the virus first emerged. “Our goal is to provide the vaccine to the world including the US, EU and others,” Yin said.

Stringent regulations in the US, European Union, Japan and Australia have historically blocked the sale of Chinese vaccines. But Yin said that could change. Sinovac is developing one of China’s top four vaccine candidates—-Sinovac–along with state-owned SinoPharm, which has two in development, and military-affiliated private firm CanSino.

More than 24,000 people are participating in clinical trials of CoronaVac in Brazil, Turkey, and Indonesia, with additional trials scheduled for Bangladesh and possibly Chile, Yin said. Sinovac chose those countries because they all had serious outbreaks, large populations and limited research and development capacity, he said. He spoke to reporters during a tour of a Sinovac plant south of Beijing.

Built in a few months from scratch, the plant is designed to enable Sinovac to produce half a million vaccine doses a year. The bio-secure facility was already busy on Thursday filling tiny bottles with the vaccine and boxing them. The company projects it will be able to produce a few hundred million doses of the vaccine by February or March of next year. Sinovac is also starting to test small doses of CoronaVac on children and the elderly in China after noticing rising numbers of cases globally among those two groups.

Yin said the company would prioritize distribution of the vaccine to countries hosting human trials of CoronaVac. While the vaccine has not yet passed the Phase 3 clinical trials, a globally accepted standard, Sinovac has already injected thousands of people in China under an emergency use provision. Yin said he was one of the first to receive the experimental vaccine months ago along with researchers after Phase 1 and 2 of human trials showed no serious adverse effects. He said that self-injecting showed his support for CoronaVac.

IPO Sector: Virios Therapeutics Inc., a Phase 2 biotech developing novel antiviral therapies for fibromyalgia and other diseases, announced terms for its IPO on Friday. The Alpharetta, GA-based company plans to raise $30 million by offering 3 million shares at a price range of $9 to $11. At the midpoint of the proposed range, Virios Therapeutics would command a fully diluted market value of $78 million. The company’s lead candidate, IMC-1, is a novel combination antiviral therapy designed to synergistically suppress Herpes Simplex Virus-1activation and replication, with the end goal of reducing viral mediated disease burden. In a Phase 2a proof-of-concept trial for fibromyalgia, IMC-1 provided statistically significant improvement versus placebo in the primary endpoint of pain reduction. The company plans to enter a Phase 2b trial in the 1Q21, with topline data expected in the 1Q22. Virios Therapeutics was founded in 2012 and plans to list on the Nasdaq under the symbol “VIRI.” ThinkEquity is the sole bookrunner on the deal.

Elsewhere, Graybug Vision Inc., a Phase 2 biotech developing transformative medicines for ocular diseases, raised $90 million by offering 5.6 million shares at $16, the midpoint of the $15 to $17 range. The company’s lead candidate, GB-102, is an intravitreal injection being developed as a once-every-six months treatment for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). In its Phase 1/2a trial, GB-102 was well-tolerated in wet AMD patients. GB-102 is currently in a Phase 2b trial for wet AMD, with topline data expected in the 1H21. Graybug Vision was founded in 2015 and lists on the Nasdaq under the symbol “GRAY.” SVB Leerink, Piper Sandler, Needham & Co. and Wedbush PacGrow are the joint bookrunners on the deal. Shares closed the week up 3% to $16.50.

Orphazyme A/S, a Danish late-stage biotech developing protein therapies for rare neurodegenerative diseases, announced terms for its IPO. The Copenhagen, Denmark-based company plans to raise $100 million by offering 7.6 million ADSs at $13.13, the as-converted September 17th close of its shares on the Nasdaq Copenhagen (ORPHA). At the proposed price, Orphazyme would command a fully diluted market value of $455 million. 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. Orphazyme 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.

PMV Pharmaceuticals Inc., an early stage biotech developing targeted therapies for cancer, announced terms for its IPO. The Cranbury, NJ-based company plans to raise $125 million by offering 7.4 million shares at a price range of $16 to $18. At the midpoint of the proposed range, PMV Pharmaceuticals would command a fully diluted market value of $714 million. The company’s lead candidate, PC14586, is designed to potently and selectively correct p53 misfolding caused by a specific p53 mutation while sparing wild-type p53. The company is initially pursuing a tumor-agnostic development strategy and submitted an IND for PC14586 in August 2020, with a Phase 1/2 trial planned for the 2H20. PMV Pharmaceuticals was founded in 2013 and plans to list on the Nasdaq under the symbol “PMVP.” Goldman Sachs, BofA Securities, Cowen and Evercore ISI are the joint bookrunners on the deal.

Prelude Therapeutics Inc., a Phase 1 biotech developing small molecule therapies for cancer, raised $158 million by offering 8.3 million shares at $19, the high end of the range of $17 to $19. The company’s first clinical candidate, PRT543, is currently in a Phase 1 trial in select solid tumors and myeloid malignancies in patients who are refractory to or intolerant of established therapies. Interim Phase 1 results indicate dose-dependent increases in exposure and target engagement, and the company has observed early signs of clinical activity. The company expects clinical data in the 1H21. Prelude was founded in 2016 and lists on the Nasdaq under the symbol “PRLD.” Morgan Stanley, Goldman Sachs and BofA Securities are the joint bookrunners on the deal. Shares closed the week up 38% to $26.20.

Greenwich LifeSciences Inc., a Phase 3 biotech developing immunotherapies for breast cancer, raised $7 million by offering 1.3 million shares (100% primary) at $5.75, the low end of the downwardly revised range of $5.75 to $6.75. The company originally planned to offer 2.7 million shares (37% primary) at $7.50 to $8.50 before revising its terms earlier this month. Greenwich LifeSciences lists on the Nasdaq under the symbol “GLSI.” Aegis Capital Corp. acted as a lead manager on the deal. Shares closed the week down 13% to $5.00.

Included among recent SEC filings for an initial public offering, Tarsus Pharmaceuticals Inc., a late stage biopharmaceutical company focused on the development of therapeutics for ophthalmic conditions, registered up to $86 million in an initial public offering. The company’s lead product candidate, TP-03, is a novel therapeutic being developed for the treatment of blepharitis caused by the infestation of Demodex mites, which is referred to as Demodex blepharitis. To date, the company has completed four Phase 2 trials for TP-03 in Demodex blepharitis, all of which met their primary, secondary and/or exploratory endpoints. The company commenced a Phase 2b/3 trial in September 2020, and intends to commence a Phase 3 trial in 2021. The Irvine, CA-based company was founded in 2016 and plans to list on the Nasdaq under the symbol “TARS.” BofA Securities, Jefferies, Raymond James, LifeSci Capital and Ladenburg Thalmann are the joint bookrunners on the deal. No pricing terms were disclosed.

Praxis Precision Medicines Inc., a Phase 2 biotech developing therapies for CNS disorders using genetic insights, registered up to $100 million in an IPO. The company’s pipeline contains its most advanced candidates, PRAX-114 and PRAX-944, which are in Phase 2 development for major depressive disorder/perimenopausal depression and essential tremor, respectively. The company plans to initiate a Phase 2/3 trial for PRAX-114 in the US and Australia in the 4Q20, with topline data expected in the 2H21. The Cambridge, MA-based company was founded in 2015 and plans to list on the Nasdaq under the symbol “PRAX.” Cowen, Evercore ISI and Piper Sandler are the joint bookrunners on the deal. No pricing terms were disclosed.

Aligos Therapeutics Inc., a Phase 1 biotech developing oligonucleotide therapies for viral and liver diseases, registered up to $100 million in an IPO. The company’s lead focus is to develop a functional cure for Chronic Hepatitis B (CHB). The company has developed a portfolio of differentiated drug candidates for CHB, including ALG-010133, an S-antigen Transport-inhibiting Oligonucleotide Polymers (STOPS) molecule. ALG-010133 is currently in a Phase 1 proof of concept trial in New Zealand. The South San Francisco, CA-based company was founded in 2018 and plans to list on the Nasdaq under the symbol “ALGS.” J.P. Morgan, Jefferies, Piper Sandler and Cantor Fitzgerald are the joint bookrunners on the deal. No pricing terms were disclosed.

And Eargo Inc., which manufactures differentiated hearing aid systems, registered up to $100 million in an IPO. Eargo believes its hearing aids are the first and only virtually invisible, rechargeable, completely-in-canal, FDA regulated, exempt Class I device for the treatment of hearing loss. The company has sold over 42,000 hearing aid systems, net of returns, as of June 30, 2020. The Mountain View, CA-based company was founded in 2010 and booked $47 million in sales for the 12 months ended June 30, 2020. It plans to list on the Nasdaq under the symbol “EAR.” J.P. Morgan, BofA Securities, Wells Fargo Securities and William Blair are the joint bookrunners on the deal. No pricing terms were disclosed.

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