Auris Medical pivots to RNA with Trasir Therapeutics buyout

It’s all-change at Auris Medical as it ditches its leaking pipeline, changes it name and its ticker as it picks up an RNA biotech to try to keep going, but in a new guise.

Auris Medical as we know it will become obsolete as it changes its name to Altamira Therapeutics and changes its trading symbol to "CYTO," the word root for cell in ancient Greek, killing off its former "EARS" ticker.

EARS was picked as the company had once been heavily focused on AM-111, a late-stage asset targeting sudden deafness, although this failed a phase 3 in 2017, with further failures in tinnitus also hitting the biotech.

This, along with the rest of its pipeline in eurotology, rhinology and allergology (which presumably includes its early nasal spray work for SARS-CoV-02) is being swept out and put up for sale, including its drugs for vertigo and allergens.

This has all been prompted by the buyout of Tampa, Florida-based Trasir Therapeutics, with the deal made up of 0.77 million common shares as well as the assumption of certain selling shareholders’ cash outlays and future share-based payment contingents.

Auris will tap the little-known, private biotech to fill up its now vacant pipeline with its RNA platform in extrahepatic oligonucleotide delivery. It’s all very early stage, but its so-called OligoPhore platform enables delivery to target tissues outside the liver, “creating the potential for developing RNA-based therapies for a range of indications with substantial unmet need,” the company said.

Preclinical work shows promise, the biotech said, for “several oncology indications” as well as rare diseases, rheumatoid and osteoarthritis and inflammatory pathologies such as atherosclerosis.

Work will first start on AM-401, a preclinical asset, in “oncology or rare disease indication,” with a trial start planned for next year.

It will also tap its new biotech for the delivery of siRNA, mRNA and gene editing constructs and will “seek to leverage the platform’s potential through strategic partnering.”

“Following a thorough review of various strategic options, we are very excited to enter with the Trasir acquisition the field of oligonucleotide delivery, which we believe will provide us with a new range of RNA-based therapeutics with disruptive potential,” said Thomas Meyer, Auris Medical’s founder, chairman and CEO.

“Although we continue to believe that our existing business holds great promise, we acknowledge investors’ preferences for highly focused company strategies. Therefore, we will prepare for its separation either through a divestiture or a spin-off to shareholders within the next 12-18 months. Through the strategic repositioning and transformation of the company, we aim to unlock and create significant shareholder value.”

Auris was up 10% premarket on the news, though it has a market cap of less than $50 million, trading for years at around the $8 a share price, a major slump from the days before 2016 and the first of its ear disorder flops.

https://www.fiercebiotech.com/biotech/after-flops-and-setbacks-auris-medical-pivots-to-rna-trasir-therapeutics-buyout

Valneva's not too late in COVID-19 as analysts eye $1.1B in 2022 vaccine sales

 Valneva has yet to successfully usher its inactivated COVID-19 vaccine across the regulatory finish line, but analysts still see reason to bet on the French vaccine specialist.

The biotech's shot could bring in roughly $1.1 billion in 2022 through supply deals and more than $500 million in 2023, Jefferies analysts wrote in a note to clients Tuesday. The vaccine, dubbed VLA2001, is currently in late-stage trials—tested against AstraZeneca's shot—with a readout expected in the third quarter this year. 

Unlike its pandemic competitors, Valneva is the only European-made inactivated, adjuvanted coronavirus candidate that has undergone clinical testing in the region. It will likely be the only, or at least the leading, shot of its type in Europe, the analysts said. 

The vaccine, which incorporates a CpG 1018 adjuvant from Dynavax, could have a safety edge since mRNA shots are still novel technologies and adenovirus vaccines from Johnson & Johnson and AstraZeneca have been plagued with concerns of rare but serious blood clots, the analysts wrote.

On top of that, Valneva’s shot won’t come with stringent cold-chain requirements and has a longer shelf life than current mRNA options. The Jefferies analysts believe that countries will likely stockpile COVID-19 vaccines “above and beyond what is necessary out of an abundance of caution," and VLA2001 could pose as a viable choice. 

Valneva, a relative unknown in the vaccine race so far, already has a supply agreement with the U.K. worth €1.4 billion ($1.63 billion) for a five-year supply of doses. The deal could eventually bring in 190 million doses by 2025. The company is preparing to deliver 60 million of those shots in the second half of 2021. 

In April, the company said it would no longer focus on striking a supply pact with the EU and would instead turn to individual countries. Germany, France and Sweden are likely candidates looking to buy more doses, Jefferies analysts wrote.

However, if the shot makes it to the market, the vaccine will have to face other well-established Big Pharmas that have accumulated vaccine sales far beyond Jefferies’ predictions for Valneva.

Moderna’s shot in the first quarter alone cashed in $1.73 billion and is expected to bring in $19.2 billion in sales by year-end. Meanwhile, Pfizer is expecting an even bigger payout. It’s vaccine, first to market in the U.S., reeled in $3.4 billion during the first quarter. Pfizer has laid out a $26 billion sales forecast for the full year. 

Those companies have raced to develop booster shots to target troublesome variants of the virus, a lucrative sign for investors who are eyeing pandemic revenue streams for years to come. 

For its part, Valneva is in “a good position to benefit” as a booster shot, the Jefferies analysts argue. The shot is part of a U.K.-government funded clinical trial examining seven different vaccines to determine how effective a booster of each shot is at protecting individuals from the virus, according to the company. Initial results are expected in September. 

https://www.fiercepharma.com/pharma/it-s-not-too-late-for-valneva-analysts-eye-1-1b-covid-19-vaccine-sales-next-year

Airborne Pathogen Standard Can Protect Us

 Healthcare providers have been at the forefront of many changes to our healthcare systems and society during the pandemic, as they treat COVID-19 patients and face a constant threat of exposure. Personal protective equipment (PPE) has become the norm, and it has undoubtedly kept healthcare workers safer. Now, as the pandemic may be nearing its end in the U.S., the question becomes whether these measures, adopted out of necessity, will persist. Personally, I am worried they will not. Once the pandemic recedes from memory and the impetus for wearing PPE isn't present on a daily basis, I fear these protections will no longer be universally provided to us.

So, how can we ensure that healthcare workers continue to have access to masks and other PPE? The Occupational Safety and Health Administration (OSHA) should create an airborne pathogen standard.

Throughout the pandemic, we worked to limit the toll this disease took on our country. This work came with significant risk: healthcare workers were three times more likely to be infected with COVID-19 compared to the general public. This high infectivity rate was likely exacerbated by PPE shortages during the initial phases of the pandemic, with many healthcare workers either having inadequate PPE or being forced to reuse their equipment well beyond its intended use. Early on, there were even alarming news stories of hospitals and other healthcare organizations that actually forbade their employees from wearing masks and other PPE.

However, it quickly became obvious that healthcare systems needed to implement new standards. With remarkable speed, wearing masks, eye protection, and gowns became standard practice. CDC developed COVID-19 recommendations for healthcare providers, which stated that providers "should wear well-fitting source control at all times while they are in the healthcare facility." Masks and other protective equipment, while initially in short supply, undoubtedly helped prevent scores of healthcare providers from becoming ill -- or worse -- due to a COVID-19 infection.

Adequate PPE is absolutely necessary to protect healthcare workers from airborne infection. An OSHA airborne pathogen standard is an important step to ensure the PPE shortages and restrictions on mask wearing we witnessed during this pandemic never happen to us again. The CDC issues recommendations, but an OSHA regulation can have real consequences, including loss of certification and significant fines. An OSHA airborne pathogen standard would not only codify best practices for prevention and protection, but it would also place the onus on hospitals and other healthcare employers to ensure they adequately protect their employees in the future.

This wouldn't be the first time OSHA produced workplace standards in response to a significant health event -- precedent already exists. In the early 1990s, OSHA created the Bloodborne Pathogens standard in response to the AIDS epidemic. This standard laid out requirements for employers "to protect workers who are occupationally exposed to blood or other potentially infectious organisms." Among other protections, it requires that gloves are provided to employees, disposable needles are used, and that post-exposure prophylaxis is made available to any employee who is potentially exposed to a bloodborne organism. These practices are now known as "universal precautions," meaning they are used for all patients. Most physicians cannot imagine practicing medicine without them. Yet, if it were not for this OSHA standard, these basic protective measures might not have become universal. OSHA's actions ensured that all employees would enjoy this level of protection.

It's time for OSHA to take action on the important issue we're facing today. At this point, it is not clear when or if COVID-19 is going to completely disappear. It's also unlikely that COVID-19 is the last, or even the worst, airborne pathogen we will face. The coronavirus pandemic, for all of the suffering it has caused, has also helped advance our understanding of best practices for airborne contagion prevention. It has shown us how vulnerable we are to these pathogens when basic, protective measures are lacking. OSHA, please give us this standard; we cannot let all that we have learned from this pandemic go to waste.

Gregory Jasani, MD, is an emergency medicine physician at the University of Maryland Medical Center in Baltimore.

https://www.medpagetoday.com/infectiousdisease/covid19/92899

Biotech Stocks With Huge Catalysts in June

 Biotech investors better buckle up for a wild ride this month. Some of the industry's biggest players are waiting for clinical trial results and decisions from the FDA that could send their stock prices rocketing higher overnight or stomp them into the dirt.

Here's why shares of Biogen (NASDAQ:BIIB), Vertex Pharmaceuticals (NASDAQ:VRTX), and Sage Therapeutics (NASDAQ:SAGE) will be extra jumpy this month. 

1. Biogen

This biotech is awaiting an extremely controversial decision the FDA has to make regarding its potential new Alzheimer's drug. Factions within the agency are eager to approve aducanumab for the treatment of early stage Alzheimer's disease despite a troubling lack of evidence it can keep patients from losing their minds.

It's been more than two years since the FDA's last commissioner, Scott Gottleib stepped down and the agency is still flying without an official pilot. The acting commissioner, Janet Woodcock is credited with pushing forward the FDA's most controversial new drug approval decision to date.

It's hard to tell which direction the FDA will take, but it's clear that Biogen needs a new growth driver. In the first quarter, product sales slid 24% year over year as the bottom fell out from under the company's aging multiple sclerosis franchise. Sales of spinal muscular atrophy (SMA) treatment, Spinraza also slid in response to recently approved alternatives like Zolgensma and Evrysdi.

Generic competition for Tecfidera that began last fall isn't going to subside and new treatment options for SMA aren't going away either. If the agency doesn't approve aducanumab, investors can expect Biogen's performance and stock price to deteriorate much further.

2. Vertex Pharmaceuticals

Before Vertex Pharmaceuticals launched its first cystic fibrosis (CF) drug in 2012, inheriting the disorder was considered a death sentence. Now it's a treatable condition for around 90% of people who inherit one of more than 1,700 known mutations to the CFTR gene that can slowly make it impossible to breathe.   

This company's oral treatments for cystic fibrosis (CF) have driven its topline and its stock price to astonishing heights, but there's a problem. Now that there aren't many CF patients left untreated, investors are worried that Vertex's bottom line won't continue growing at its usual pace. That's why all eyes are on expected trial data from VX-864, one of the company's most promising clinical-stage programs at the moment.

In the first quarter, Vertex Pharmaceuticals finished enrolling patients who are alpha-1 antitrypsin (AAT) deficient into a proof-of-concept study with VX-864. Investigators should have wrapped up the trial by now, so we can expect the results soon. 

Last October, Vertex's first attempt at correcting misfolded AAT proteins, VX-814 didn't work out as hoped. The company's hoping VX-864's slightly different structure will be able to get the job done. 

3. Sage Therapeutics

This company develops drugs for depression, but its first treatment to earn FDA approval, Zulresson hasn't made the splash investors had hoped for. Despite launching in 2019, sales of the difficult to administer infusion for women suffering from post-partum depression came in at just $1.6 million in the first quarter.

The pressure's on to launch a new drug, which is why there will be a lot of attention this month for trial results from zuranolone, the most promising clinical-stage program in the company's pipeline. In May, Sage Therapeutics told investors to expect results of a phase 3 trial with zuranolone and MDD patients before the end of June.

Before getting too excited about Sage Therapeutics, you should know that this stock lost half its value when zuranolone failed to meet its primary endpoint as a treatment for major depressive disorder (MDD) in 2019. 

Instead of throwing in the towel, Sage Therapeutics started testing a higher dosage and so far it seems to do the trick. In March, the company showed that 80% of MDD patients who received a higher zuranolone dose responded to the treatment.

Reading the tea leaves

Investors should know that Sage Therapeutics corporate operating officer left the day before the company announced the revival of its zuranolone program. That doesn't necessarily mean it's destined to disappoint, but you'll feel awfully silly about ignoring this warning if the same candidate fails again.

I'll be pleasantly surprised if Vertex Pharmaceuticals AAT program delivers positive data this month. In April the company told investors it was evaluating new AAT treatment candidates beyond VX-864 giving the impression the company expects to move on.

I think the evidence supporting Biogen's Alzheimer's disease drug warrants another phase 3 trial, not approval. That said, I wouldn't be surprised if the agency gives in to pressure from lobbying groups that have been clamoring for aducanumab's approval.

https://www.fool.com/investing/2021/06/02/3-biotech-stocks-with-huge-catalysts-in-june/

Citi picks Goldman partner for healthcare and consumer M&A 'super group'

 Citigroup has poached a Goldman Sachs partner to launch a new M&A advisory franchise in New York focusing on global healthcare, consumer and wellness as it seeks to increase its presence in key industries, according to a memo seen by Reuters.

Chuck Adams, a 16-year Goldman veteran, will take on a new role in December as vice chairman and global head of the new Citi unit, described in the memo as a "super group" which will absorb its existing operations in healthcare as well as consumer and retail.

The move was prompted by a growing convergence among sectors that are becoming "more integrated, consumer-centric, tech enabled and focused on sustainable wellbeing", the memo said.

Adams, who was responsible for Goldman's healthcare investment banking practice on the West Coast and also headed its West Region advisory business, will report to Tyler Dickson and Manolo Falco, global co-heads of Citi's banking, capital markets and advisory (BCMA) business.

"Healthcare, Consumer & Wellness will be one of our largest groups in BCMA and represents one of the greatest opportunities for growth," the pair said in the memo.

The healthcare sector, unlike other industries, has been largely immune to the disruptions caused by the pandemic and is likely to see a wave of consolidation led by cash-rich companies in coming years.

Before joining Goldman, Adams spent 10 years at Morgan Stanley, where he focused on healthcare investment banking. 

https://finance.yahoo.com/news/1-citi-picks-goldman-partner-174807248.html

CanSinoBIO's inhalation COVID-19 shot in mid-stage trial, says CEO

 An inhalation version of CanSino Biologics' COVID-19 vaccine is being tested in a Phase II clinical trial, Chief Executive Yu Xuefeng said late on Wednesday.

Yu made the comment at a panel of the Global Health Forum of the Boao Forum for Asia.

https://news.trust.org/item/20210602114934-bxfdh/

Israel finds a ‘probable link’ between Pfizer vaccine and myocarditis cases

 A small number of cases of myocarditis – a type of heart inflammation – may be linked to the second dose of Pfizer’s BioNTech-partnered COVID-19 vaccine, Israel’s Health Ministry announced this week.

The cases, observed mostly in young men who had received the Pfizer/BioNTech jab, have a ‘probable link’ to the second dose of the vaccine, according to Israel.

Israel’s Health Ministry reported that it was aware of 275 cases of myocarditis occurring between December 2020 to May 2021, with 148 of these cases happening within a short time frame after receiving the Pfizer/BioNTech vaccine.

The ministry added that the majority were seen among men under the age of 30 years and particularly in those aged between 16 and 19 years old.

Of the 5,401,150 who had been given the vaccine within this time period, 27 myocarditis cases were reported, including in 11 people with pre-existing conditions, shortly after their first Pfizer/BioNTech vaccine dose.

This increased after the second dose, however, with 121 cases, including 60 people with pre-existing conditions, developing myocarditis within 30 days out of a total 5,049,424 fully vaccinated individuals in Israel.

The ministry added that most of the cases were mild, with one death linked to myocarditis but not conclusively proven.

Pfizer said that it is ‘aware’ of the reports and findings in Israel of myocarditis in vaccinated people, although it added that no causal link has yet been established.

Last month, a US Centers for Disease Control and Prevention (CDC) advisory group said that further study of myocarditis in young adults who have received mRNA-based COVID-19 vaccines. Pfizer/BioNTech and Moderna’s are the only two mRNA-based vaccines approved for use in the US.

In a statement, the CDC’s Advisory Committee on Immunization Practices said that it had evaluated cases among a very small number of adolescents and young adults who developed myocarditis following COVID-19 vaccination.

The CDC added that it had not seen more cases than would typically be expected in the population, although advisory committee members said that healthcare providers should be wary of the reports of the “potential adverse event”.

https://www.pmlive.com/pharma_news/israel_finds_a_probable_link_between_pfizer_vaccine_and_myocarditis_cases_1371097