Pfizer seeks emergency use of its COVID-19 vaccine in US.

Pfizer Inc. (NYC) and its German partner BioNTech SE (Mainz) on Friday filed an application with the US Food and Drug Administration (FDA) for an emergency use authorization (EUA) of its vaccine against COVID-19, the disease caused by SARS-CoV-2, according to a company news release. It’s the latest step in what has been an unusually fast-paced development and testing process, with the companies having reported interim results of Phase 3 trials on November 9 and final results this past Wednesday, as reported by Medscape. The vaccine, BNT162b2, which uses a messenger RNA-based platform, was ultimately found to have 95% efficacy and more than 94% efficacy in individuals over age 65. “

The process of the speed did not compromise safety [at all], nor did it compromise scientific integrity,” said Anthony Fauci, MD, director of the National Institute of Allergy and Infectious Diseases at White House press briefing Thursday. “We need to put to rest any concept that this was rushed in an inappropriate way,” he said. “This is really solid.”

Pfizer and BioNTech said they believe they have met the FDA’s safety data requirements for the EUA. The agency in October outlined its expectations for safety and efficacy to secure an EUA.

“Filing in the US represents a critical milestone in our journey to deliver a Covid-19 vaccine to the world, and we now have a more complete picture of both the efficacy and safety profile of our vaccine, giving us confidence in its potential,” said Albert Bourla, MD, Pfizer’s chairman and CEO, in its release.

The FDA is expected to hold a meeting of its Vaccines and Related Biological Products Advisory Committee sometime in December to review the safety and efficacy data in the companies’ application. The committee will review: 1)Efficacy data from a total 170 confirmed cases of COVID-19 in the Phase 3 study;

2) Safety data from a randomly assigned subset of 8000 participants 18 years and older; 3) Data on 19,000 enrollees who have been followed for a median of 2 months after the second and final dose; and 4) Data on the manufacturing processes. According to Pfizer, the companies plan to submit the efficacy and safety data to a peer-reviewed journal once they have completed their analysis.

The companies–which funded their own trial–signed an agreement with the US government’s Operation Warp Speed program in July to provide 100 million Doses of its vaccine following FDA authorization or approval in exchange for $1.95 billion. The US government has the option to acquire up to 500 million more doses. Pfizer and BioNTech said they will be able to supply 50 million doses globally in 2020 and up to 1.3 billion doses by the end of 2021.

The vaccine must be given in two doses, spaced 21 days apart. Pfizer expects to be ready to distribute the vaccine within hours after FDA authorization. Pfizer’s vaccine must be shipped and stored at -70°C (-94°F), which has presented logistical and storage issues. The company is testing out delivery methods, including a pilot delivery program in New Mexico, Rhode Island, Tennessee, and Texas that will be active after an FDA authorization. States, hospitals, and pharmacy chains are also buying special freezers.

Separately, according to Johns Hopkins Medical, as of Saturday, November 21, there were 57.9 million global COVID-19 cases confirmed, and some 1.4 million deaths. Confirmed cases in the US totaled 4 million with nearly 255,000 deaths. The US figures comprise 20.7% and 18.2% respectively of the global totals. The US population comprises 4% of the global total.

COVID-19 Addenda: The US passed 12 million total coronavirus cases on Saturday, and new, daily cases are approaching 200,000: on Friday, the country recorded more than 198,500, a record. As the nation reconsiders the usual winter holiday travel and cozy indoor gatherings, new cases are being reported at an unrelenting clip. The seven-day average has exceeded 100,000 cases a day every day for the last two weeks, according to a New York Times database. The country’s death toll from the virus stood at nearly 255,000 as of midday Saturday.

And a Times analysis shows that the US has deaths that are higher than normal in all 50 states, as the pandemic brings unusual patterns of death. The latest virus surge began hastening across much of the country in mid-October. It took just over two weeks for the nation to go from eight million cases to nine million on Oct. 30; going from nine to 10 million took only 10 days. From 10 million to 11 million took just under seven days. Despite near-daily records for both new cases and hospitalizations, there remains bipartisan reluctance toward issuing the sweeping type of stay-at-home orders seen in the earliest days of the pandemic.

Elsewhere, US regulators have allowed emergency use of the first rapid coronavirus test that can be performed entirely at home and delivers results in 30 minutes. The announcement by the Food and Drug Administration represents an important step in US efforts to expand testing options for COVID-19 beyond healthcare facilities and testing sites. However, the test will require a prescription, likely limiting its initial use.

The FDA granted emergency authorization to the single-use test kit from Emeryville, CA-based Lucira Health Inc. The manufacturer’s test allows users to swab themselves to collect a nasal sample. The sample is then swirled in a vial of laboratory solution that plugs into a portable device. Results are displayed as lights labeled positive or negative. To date, the FDA has authorized nearly 300 tests for coronavirus.

The vast majority require a nasal swab performed by a health professional and must be processed at laboratories using high-tech equipment. A handful of tests allow people to collect their own sample at home–a nasal swab or saliva–that’s then shipped to a lab, which usually means waiting days for results.

And the European Union could pay more than $10 billion to secure hundreds of millions of doses of the vaccine candidates being developed by Pfizer Inc./BioNTech SE and CureVac AG, an EU official involved in the talks told Reuters. The source said the bloc has agreed to pay 15.50 euros ($18.34) per dose for the COVID-19 vaccine candidate being developed by Pfizer/BioNTech, according to the official. That would mean an overall price of up to 3.1 billion euros ($3.7 billion) for 200 million doses, rising to 4.65 billion euros if another optional 100 million doses are purchased under the deal, the official said.

The pricing information, previously undisclosed, confirms the EU is paying less per dose than the US for an initial supply of that vaccine, as reported by Reuters the week prior. The deal includes an insurance for EU countries to get compensation if the companies divert doses to the US, according to the source, who requested anonymity due to the sensitivity of the matter. The EU has separately agreed to pay 10 euros ($11.84) per dose for an initial supply of 225 million doses from CureVac, a discount from the 12 euros the company initially set.

Moderna adds more hope; says its COVID-19 vaccine is 94.5% effective.

Moderna Inc.’s (Cambridge MA) announcement last week that its vaccine was more than 94% effective at preventing the coronavirus–according to preliminary trial data–raised global hopes further that a resolution to the pandemic that has killed over 1.4 million could be in sight. The news, reported by CNBC, followed on from Pfizer Inc. (NYC) and BioNTech SE’s (Mainz DEU) equally positive news that their vaccine candidate was over 90% effective. The news from Moderna was hailed as a “game changer” and Pfizer’s CEO described the vaccine achievement as “a great day for science and humanity.”

As the market revelry at the news continued, attention quickly turned to practical matters given the unprecedented logistical challenge posed by producing and distributing vaccines, should they receive final regulatory approval, to a global population of around seven billion people. Vaccines need to be produced and transported in specific (and cold) conditions otherwise they can be rendered ineffective; this poses a huge challenge for global drugmakers when it comes to vaccine distribution.

Swiss drugmaker Lonza AG (Basel) has partnered with Moderna and says it aims to produce 400 million doses of the vaccine annually. The US firm is aiming for 500 million to 1 billion doses in total for 2021. Anyone receiving the vaccine will require two doses, as with Pfizer’s shot, showing how long it could take, with the current manufacturing capacity, to vaccinate internationally. Lonza will produce ingredients within Moderna’s vaccine, formally called mRNA-1273, in facilities in the US and Switzerland, where it is headquartered.

Company Chairman Albert Baehny told CNBC about the “big challenges” facing drugmakers like his when it comes to scaling up production. “We can only produce more than 500 million doses a year if we install additional manufacturing lines, so it is clear that we need additional investments in installation if we want to produce more than 500 million (per year) in the future,” he told CNBC on Wednesday.

Baehny identified more challenges to vaccine production that the company has had to confront since embarking on its partnership with Moderna. “There are a few issues; the first is speed. We only started 10, 11 months ago and now we are producing the first commercial batches of the drug substance in North America, and we are planning the first batch of commercial volume in one or two weeks in Switzerland, so the speed has been a challenge, Baehny added. Temperature, and keeping the vaccines cold enough during transportation, is another big challenge.

Pfizer’s vaccine requires a storage temperature of minus 94 degrees Fahrenheit, or -70 degrees Celsius. By comparison, Moderna said that its vaccine remains stable at 36 to 46 degrees Fahrenheit–the temperature of a standard home or medical refrigerator–for up to 30 days. It can be stored for up to six months at minus 4 degrees Fahrenheit.

WHO advises against Gilead’s remdesivir for all hospitalized COVID-19 patients.

The World Health Organization (Geneva) on Friday issued a conditional recommendation against the use of Gilead Sciences Inc.’s (Foster City CA) Veklury (remdesivir) in hospitalized patients with COVID-19, regardless of disease severity. The WHO’s international guideline development group looked at interim data from the Solidarity trial, as well as three other randomized studies, concluding that there is currently no evidence that the antiviral improves survival and other outcomes in these patients.

Results from the Solidarity trial were released last month, with findings suggesting that Veklury had “little or no effect” on 28-day mortality or the in-hospital course of COVID-19 among hospitalized patients. Data indicated that the mortality rate ratio for the drug versus control was 0.95, although at the time, Gilead questioned the study’s design, while also pointing to positive data from the Phase 3 ACTT-1 trial led by the US National Institute of Allergy and Infectious Diseases.

The WHO’s guideline development group, which includes 28 clinical care experts, four patient-partners and one ethicist, said the evidence suggested that Veklury had “no important effect” on mortality, need for mechanical ventilation, time to clinical improvement and other patient-important outcomes. However, the panel called for more research, particularly to provide “higher certainty of evidence” for specific patient groups. The development group added “especially given the costs and resource implications associated with [Veklury]…the panel felt the responsibility should be on demonstrating evidence of efficacy, which is not established by the currently available data.”

In response to the guidelines, Gilead stated “Veklury is recognized as a standard of care for the treatment of hospitalized patients with COVID-19 in guidelines from numerous credible national organizations.” Veklury gained full approval from the FDA in October to treat hospitalized COVID-19 patients aged 12 years and older and weighing at least 40 kg. The drug was previously authorized by the agency for emergency use, initially for those with severe manifestations of COVID-19, and later for all hospitalized adult and pediatric patients with the infection. (Ref: FirstWord Pharma)

IPO Sector: Included among recent SEC filings for initial public offerings, iSpecimen Inc., which provides an online marketplace platform for biospecimen procurement, registered up to $21 million worth of common stock. The company hopes to accelerate life science research and development with a global marketplace platform that connects researchers to subjects, specimens, and associated data. Its current customer base is primarily comprised of three main segments: biopharmaceutical companies, in vitro diagnostic companies, and government/academic institutions. As of September 30, 2020, there were more than 3,500 external registered users on the iSpecimen Marketplace platform, representing more than 2,700 unique organizations. The Lexington, MA-based company was founded in 2009 and booked $7 million in revenue for the 12 months ended September 30, 2020. It has yet to select an exchange or ticker. ThinkEquity is the sole bookrunner on the deal. No pricing terms were disclosed.

Elsewhere, AbCellera Biologics Inc., which provides an AI powered drug discovery platform for antibody therapies, registered up to $200 million in an IPO. The company offers a full-stack, artificial intelligence-powered drug discovery platform that searches and analyzes a database of natural immune systems to find antibodies that can be developed as drugs. Rather than advancing its own clinical pipeline of drug candidates, AbCellera forges partnerships with drug developers and helps them develop drugs more efficiently. As of September 30, 2020, the company had 94 discovery programs that are either completed, in progress, or under contract with 26 partners. The Vancouver, Canada-based company was founded in 2012 and booked $28 million in revenue for the 12 months ended September 30, 2020. It plans to list on the Nasdaq under the symbol “ABCL.” Credit Suisse, Stifel, Berenberg, SVB Leerink and BMO Capital Markets are the joint bookrunners on the deal. No pricing terms were disclosed.

Nanobiotix SA registered up to $60 million in a US IPO. Nanobiotix is developing proprietary nanotechnology to transform cancer treatment by increasing the efficacy of radiotherapy. Its lead candidate, NBTXR3, is designed to enhance the overall efficacy of radiotherapy without resulting in additional side effects on the surrounding healthy tissues. NBTXR3 has been administered to more than 230 patients and is currently involved in a total of eight clinical trials worldwide. In April 2019, Nanobiotix completed the regulatory process for the CE mark of NBTXR3, allowing the product to be commercialized in the EU for the treatment of locally advanced soft tissue sarcoma under the brand name Hensify. Commercial launch is expected in the 2Q21. The Paris-based company was founded in 2003 and booked $3 million in revenue for the 12 months ended June 30, 2020. It plans to list on the Nasdaq under the symbol “NBTX.” Jefferies, Evercore ISI and UBS Investment Bank are the joint bookrunners on the deal. No pricing terms were disclosed.

Olema Pharmaceuticals Inc., a Phase 1 biotech developing a targeted oral therapy for ER+/HER2- breast cancer, raised $209 million by offering 11 million shares at $19, above the range of $16 to $18. The company offered one million more shares than anticipated. Olema is focused on the discovery, development and commercialization of next generation targeted therapies for women’s cancers. The company is studying the structure and function of the estrogen receptor (ER), a key driver of breast cancer in approximately 75% of patients, in order to develop more potent, oral therapies that completely inactivate this signaling pathway. The lead product candidate, OP-1250, has combined activity as both a complete ER antagonist and a selective ER degrader and was initiated in an ongoing Phase 1/2 trial in August of this year, with initial data expected in the second half of 2021. The San Francisco-based company was founded in 2007 and lists on the Nasdaq under the symbol “OLMA.” J.P. Morgan and Jefferies were lead managers on the deal. Shares exploded, closing the week up 257% to $48.90.

4D Molecular Therapeutics Inc., which is developing targeted gene therapies for multiple diseases, registered up to $75 million in an IPO. 4D is developing product candidates using its targeted and evolved AAV vectors, initially focusing on ophthalmology, cardiology, and pulmonology. Its lead candidates include 4D-125, which is currently in a Phase 1/2 trial for X-linked retinitis pigmentosa with initial data expected in 2021; 4D-110, which is currently in a Phase 1 trial for choroideremia with initial data expected in 2022; and 4D-310, which is currently in a Phase 1/2 trial for Fabry disease with initial data expected in 2021. It has two additional candidates that it expects to file INDs for in the 2H21. The Emeryville, CA-based company was founded in 2013 and booked $17 million in collaboration and license revenue for the 12 months ended September 30, 2020. It plans to list on the Nasdaq under the symbol “FDMT.” Goldman Sachs and BofA Securities are among the joint bookrunners on the deal. No pricing terms were disclosed.

And Scopus BioPharma Inc., a preclinical biotech developing immuno-oncology gene therapies for cancer, lowered the proposed deal size for its upcoming IPO. The New York, NY-based company now plans to raise $3 million by offering 0.5 million shares at a price range of $5.25 to $5.75. The company had previously filed to offer 1 million shares at the same range. At the revised deal size, Scopus BioPharma will raise -50% less in proceeds than previously anticipated.  The company’s lead candidate is a targeted immuno-oncology gene therapy for the treatment of multiple cancers being developed through a partnership with City of Hope. Preclinical testing at City of Hope showed reduced growth and metastasis, and the company plans to submit an IND for B-cell lymphoma in the 4Q20, with a Phase 1 trial anticipated in the 1Q21. Scopus was founded in 2017 and plans to list on the Nasdaq under the symbol “SCPS.” The Benchmark Company is the sole bookrunner on the deal.