Verily links with Janssen for at-home COVID-19 immune system study

 Verily is teaming up with Johnson & Johnson’s Janssen division to observe the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their own homes. 

The study will be launched through Verily’s Project Baseline testing program and aims to collect biological information and real-world data in the weeks immediately after a person tests positive for COVID-19.

The goal is to identify early warning biomarkers that could help guide healthcare providers through future treatments as well as potentially predict whether an individual’s case may turn severe.

“Acute respiratory distress syndrome caused by SARS-COV-2 and other viral and bacterial pathogens carries with it a high mortality rate, and more than 2.2 million people will suffer each year as a result,” said James Merson, Ph.D., Janssen’s global head of infectious disease R&D.

“Since immune response patterns observed in COVID-19 patients are similar to those caused by other respiratory pathogens, it is our hope to apply the findings from this study beyond COVID-19 to other illnesses that carry a high patient burden,” Merson said.

Verily, Google’s life-science-focused sibling company, and Janssen will also seek to tap into the data generated by people during their everyday lives to search for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

“COVID-19 is impacting millions of people worldwide from all different backgrounds and health statuses,” said Verily’s chief medical and scientific officer, Jessica Mega, M.D. “We anticipate learnings from the study will help to better inform future treatment and prevention practices as the world continues to fight this devastating pandemic.”

Separately, Verily recently opened up its coronavirus-focused Project Baseline efforts to children as young as four, while pediatric testing and COVID-19 research has previously been limited. The program will offer free COVID-19 testing to children through 450 screening sites across 16 states.

https://www.fiercebiotech.com/medtech/verily-links-janssen-for-at-home-covid-immune-system-study

Sanofi CEO: mRNA 'go-to' vaccine tech in pandemic, but 'bar is high' in other diseases

 In the global COVID-19 vaccine race, mRNA vaccines quickly got out to the front of the pack and two reached distribution in less than a year. But their quick success in COVID-19 doesn't guarantee mRNA will replace traditional vaccine technologies across the industry, Sanofi CEO Paul Hudson told Barron’s. 

During a single-antigen pandemic, where speed is a key consideration, "I think we have to accept that ... mRNA is probably the first go-to," Hudson told the publication. But in disease areas where there are established vaccines, mRNA candidates will have to “compete with the standard of care” shots that feature a “well-characterized safety profile.” There, the “bar is high,” he added. 

Sanofi is the world's top flu vaccine player, and it's among the top four Big Pharma companies in vaccines. Last year, the company's flu franchise generated €2.5 billion ($3 billion).

The drugmaker isn’t sitting on the sidelines in mRNA, either. The company has an mRNA vaccine partnership with Translate Bio in COVID-19, and last year the Big Pharma inked a $425 million partnership in infectious diseases with Translate. Hudson told Barron’s he’s “excited” about mRNA in disease areas that “have never been treated before.” 

Other vaccine experts agree that traditional vaccines aren't going anywhere—at least, for now. During a Fierce Pharma virtual panel discussion last month, execs said that while the industry is clearly enthusiastic about the mRNA platform, it’s not time to “walk away” from established technologies. 

The “beauty” of mRNA vaccines is that the production process is “universal,” CureVac’s chief technology officer, Mariola Fotin-Mleczek, said during the event. The company has a COVID-19 vaccine in development.

“If you invest in huge production capacity, you can produce different vaccines in the same plant," without needing to “start from scratch” or switch production processes, Fotin-Mleczek said.

Still, there's no guarantee mRNA will “immediately work against all pathogens," Takeda’s vaccine head Rajeev Venkayya said. Researchers have been working for decades on vaccines against “very hard targets," he added, and the industry can’t abandon its progress with proven platforms. 

In the global response to the COVID-19 pandemic, mRNA vaccines from Pfizer/BioNTech and Moderna beat all other technologies to emergency authorizations. Thanks to past research in the field, scientists were quickly able to enter testing after receiving the novel coronavirus’ viral sequence. The programs rapidly progressed through human studies and won emergency authorizations in the U.S. and elsewhere. 

Now, Pfizer and Moderna are set to earn big revenues from their coronavirus vaccines. Pfizer projects $15 billion in COVID-19 vaccine sales this year, while Moderna’s CEO has said his company could join the ranks of the top vaccine companies by revenue in 2021. 

Meanwhile, AstraZeneca’s adenovirus-based vaccine has been authorized in many countries around the world, and another adenovirus-based program from Johnson & Johnson is set for an FDA review this month. Another late-stage candidate from Novavax, a recombinant nanoparticle vaccine that uses an adjuvant, has started rolling reviews in various countries.

https://www.fiercepharma.com/pharma/sanofi-ceo-hudson-says-mrna-likely-go-to-vaccine-tech-for-a-pandemic-but-questions

Lonza $4.7B deal offloads specialty ingredients to Bain Capital, Cinven

 Switzerland's Lonza last month said it had whittled down a shortlist of bidders for its specialty ingredients business. Now, with buyers in place, the CDMO is in line for a big payout in the second half of the year. 

Lonza has agreed to sell off its specialty ingredients business to Bain Capital and Cinven for a whopping 4.2 billion CHF ($4.7 billion). The move should give the company some breathing room to focus on its Pharma Biotech & Nutrition segment, currently tapped in the manufacturing push for Moderna’s mRNA vaccine and AstraZeneca’s experimental COVID drug—plus, a hefty chunk of change to accelerate its own plans.

If the deal closes in the second half of the year as expected, Lonza will hand over the entirety of the business and operations, which comprises 17 global manufacturing sites and some 2,800 employees. The unit cranks out anti-dandruff ingredients, hygienic solutions for commercial launderers, disinfectants and more.

The business also performs custom development and production of specialty chemicals and composites for the electronics, aerospace, food and agrochemical fields. The parties say they expect the transition to be seamless for customers and employees alike.

“The sale of the Specialty Ingredients business will allow Lonza to focus on its position as a leading partner to the healthcare industry, and the free cash flows resulting from the sale will allow us to accelerate our strategic priorities,” Albert Baehny, chairman of Lonza, said in a statement.

The focus now, once Lonza is unshackled from its ingredients business, is to become a “pure-play partner to the healthcare industry,” the company said.

A specialty ingredients cast-off had been part of Lonza’s plans for some time, but it isn’t the CDMO’s first slim-down this year: The company in January said it would sell a pair of French and Scottish plants to British CDMO NextPharma. Both facilities produce lipid-oral dose drugs as liquid-filled hard capsules and softgels. With news of the upcoming divestment, Lonza added that it was exiting the pharma market for both products—though it said it would continue to make Licaps-branded lipid capsule products for nutritional and consumer health customers.

Meanwhile, the company last year established itself as one of the manufacturers to beat in the fight against COVID-19, courtesy of a 10-year production pact for Moderna’s mRNA-based vaccine. Lonza is also on deck to produce Humanigen’s COVID-19 antibody lenzilumab, as well as AstraZeneca’s experimental antibody AZD7442.

That work, coupled with Lonza’s non-COVID manufacturing duties, helped the company gin up 4.5 billion CHF ($5 billion) in 2020, marking a 12% jump over its 2019 revenue haul. Performance was largely buoyed by the company’s Pharma Biotech & Nutrition segment, which itself delivered 12.2% sales growth, the company said.  

https://www.fiercepharma.com/manufacturing/lonza-strikes-4-7b-deal-to-offload-specialty-ingredients-business-to-bain-capital

Medidata cloud platform to gather clinical trial data from sensors

 Medidata has launched Sensor Cloud, a platform to manage a range of sensor and digital health technology data during clinical trials.

The New York-based company, which is a wholly-owned subsidiary of French software firm Dassault Systemes, said the Sensor Cloud integrates Medidata’s existing clinical cloud technology while supporting data collection from the company’s own sources and third-party medical grade sensors.

In October last year, Medidata bought the digital biomarker firm MC10, adding more clinical analytics and biosensor technology to its cloud offering.

The technology could allow for remote monitoring in clinical trials of data including vital signs, movement and sleep patterns.

Using Sensor Cloud, researchers can access sensor data through a single Application Programming Interface (API), a software intermediary that allows two applications to talk to each other) and integrate new sensor technologies in weeks rather than months.

According to the company benefits include greater operational efficacy, making it easier for patients to share data and stay enrolled on trials.

Data uptake can be standardised through a common model, reducing the burden on patients and research sites by cutting down on-site visits.

It can also cut time and travel expenses through a more flexible engagement model.

Anthony Costello, president of Patient Cloud at Medidata, said: “As sensor usage in clinical trials is projected to surge up to 70% by 2025¹, it is imperative that we create the platform needed to rapidly standardize and integrate sensor data in order to harness the full power of these technologies for the benefit of patients.”

Costello added that while COVID-19 has forced many pharma companies to use remote technology during trials, the industry will continue to use it once the pandemic has receded.

“These advancements will continue to grow in popularity, outlasting the pandemic and, as trials continue to improve and become more patient-centric, communication and engagement become increasingly important. Patient involvement and the quality of the data generated by sensors will be integral to the overall evolution of this innovative research model.”

https://pharmaphorum.com/news/medidata-launches-cloud-platform-to-gather-clinical-trial-data-from-sensors/

Early Covid-19 'win' boosts Veru

 Veru yesterday heralded a win in a small trial of its oral microtubule inhibitor VERU-111 in hospitalised Covid-19 patients, and consequently enjoyed a 28% share price bump. But a close read of the data reveals that the findings are not quite as rosy as the company claims.

The central issue is that, to claim a hit by the thinnest of statistical margins, Veru had to exclude one crucial patient from its primary analysis. The group confirmed to Evaluate Vantage that this patient had suffered respiratory failure, a component of the study’s primary endpoint. There still might be a signal in the data, but on current showing it does not stand up to a rigorous statistical analysis.

Veru also reported an intriguing mortality benefit with VERU-111. But this was not a primary endpoint, and conversely to make this claim the company did use the full intent-to-treat population, which in this case slightly flattered the numbers.

VERU-111 warrants further investigation in Covid-19, but whether the company can repeat the effect in an upcoming pivotal trial is unclear.

Damn statistics

Veru said yesterday that its 39-patient phase II trial of VERU-111, in hospitalised Covid-19 patients at high risk for acute respiratory distress syndrome, had met its primary endpoint, the proportion of patients alive without respiratory failure.

According to the company, 94% of the VERU-111 group met this measure versus 70% of the placebo group; Veru said the difference only just met statistical significance, with p=0.05.

However, it revealed that this analysis was on a modified intent-to-treat population that excluded one of 19 patients given VERU-111. A spokesperson told Evaluate Vantage that this patient had only received one dose of VERU-111, but, crucially, had suffered respiratory failure and then recovered.

Thus, if this subject is included, the proportion of active cohort patients alive without respiratory failure becomes 17 of 19, or 89%. Given that the stated analysis was barely on the threshold of statistical significance, it seems clear that a difference of 89% versus 70% would have failed to clear the bar.

If the study was technically a bust, the admittedly interesting findings on mortality, a secondary endpoint, can only be considered exploratory. Veru yesterday pointed to an 82% relative reduction in deaths with VERU-111 versus placebo.

In this case the company did consider all 19 active cohort subjects.

Summary of Veru's study of VERU-111
	Active	Placebo
Intent-to-treat (ITT) population	19	20
Developed respiratory failure and died	1	6
Developed respiratory failure and recovered	1	0
Proportion alive without respiratory failure per ITT	89%	70%
P value for primary endpoint per ITT	Not given*
Relative reduction in death or respiratory failure per ITT	Not given**
Relative reduction in patient mortality per ITT	82%
*p=0.05 if using mITT population of 18 in active cohort; **81% relative reduction if using mITT population of 18 in active cohort.

Drugs that prevent deaths in hospitalised Covid-19 patients are desperately needed; the only product that has shown a clear benefit here is the steroid dexamethasone.

VERU-111 is thought to combat Covid-19 by disrupting the microtubules that make up the cytoskeleton of cells, thus perturbing the “highways” through which the coronavirus is transported.

Veru now plans a 200-patient pivotal trial, with a similar design to the phase II study and the same primary endpoint. This is set to start in April and finish by the fourth quarter, so it will not be too long until it becomes apparent whether VERU-111 could indeed become part of the anti-Covid-19 armamentarium.

If it fails, Veru can always fall back on its original plan of developing VERU-111 for prostate cancer.

https://www.evaluate.com/vantage/articles/news/trial-results/early-covid-19-win-boosts-veru

Supernus Resubmits Application for SPN-812 for ADHD Treatment

 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today announced it has resubmitted its New Drug Application (NDA) for SPN-812 for the treatment of ADHD in pediatric patients. The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the NDA in November 2020 indicating that the review cycle for the application was incomplete and that the application was not ready for approval in its present form. Supernus and the FDA held a Type A meeting in January 2021 to discuss the CRL and the requirements for the NDA resubmission.

The primary issue cited in the SPN-812 CRL relates to the Company’s in-house laboratory that conducts analytical testing, which recently moved to a new location. In the NDA resubmission Supernus removed reference to its in-house laboratory and addressed other contents of the CRL.

The FDA will classify the NDA resubmission as Class I or Class II upon acceptance of the resubmission. Generally, a Class I review constitutes a two-month review from the date of resubmission and a Class II review constitutes a six-month review from the date of resubmission.

In December 2020, Supernus announced positive results from a Phase III study for SPN-812 in adults with ADHD. The Company plans to submit a supplemental NDA to the FDA for SPN-812 in adults in the second half of 2021, assuming approval for pediatric patients.

https://www.globenewswire.com/news-release/2021/02/08/2171436/0/en/Supernus-Resubmits-New-Drug-Application-for-SPN-812-for-the-Treatment-of-ADHD-in-Pediatric-Patients.html

Merck expects interim data on COVID-19 drug in Q1

 U.S. drugmaker Merck & Co Inc said on Thursday it was expecting early data from a trial of the experimental antiviral drug it was developing with Ridgeback Bio as early as the first quarter.

The company has decided to focus on therapeutics after its two COVID-19 vaccines failed to generate desired immune responses, prompting it to abandon the program last week.

The antiviral is being currently tested in a Phase 2/3 trial that is set to be completed in May, but initial results could be reported by the first quarter, said Dean Li, head of the company’s research and development division.

Li also said the drug still needed to be tested to see if it was effective against the new variants of the novel coronavirus, but predicted that it would work on all types, based on its mechanism of action.

The most concerning new variants of the novel coronavirus currently are the so-called British, South African and Brazilian, all of which appear to spread more swiftly than others.

Merck expects to make 10 million courses of the antiviral, molnupiravir, by the end of 2021, Li said at a conference call to discuss the company’s quarterly earnings.

https://www.reuters.com/article/health-coronavirus-merck-co-idUSL4N2KA44U