Vertex will not advance trial from Phase 2, picks alternate product

 - Treatment with VX-864 led to a statistically significant increase from baseline in plasma functional AAT levels as compared to placebo and wa

s generally well tolerated -

- Results provide proof-of-mechanism, although magnitude of treatment effect observed unlikely to translate into substantial clinical benefit; VX-864 will not advance into late-stage development -

- Vertex to advance additional novel small molecule(s) with potential for greater clinical efficacy into the clinic in 2022 -

- Vertex will host an investor conference call and webcast today, Thursday, June 10 at 4:30 p.m. EDT -

Investor Webcast

The conference call will be webcast live, and a link to the webcast can be accessed on the Vertex website at www.vrtx.com in the "Investors" section. To access the call via phone, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the companies’ website for approximately 30 days.

https://finance.yahoo.com/news/vertex-announces-primary-endpoint-achieved-200300353.html

2 'Strong Buy' Penny Stocks That Could Rally to $10 (or More)

 Let’s take a moment to talk about opportunity, share price, and risk/reward considerations. These are some of the factors investors must consider when moving into penny stocks – and we haven’t even touched on the fundamental soundness of the company or its business model.

Penny stocks – as their name suggests, they once traded for just a pennies per share, but these days are considered those equities trading at less than $5 – are a challenging market niche. The penny stock critics make valid points when defending their stance. Sure, the price tag may look like a steal, but the fact that shares are trading at such low levels could reflect overwhelming headwinds or weak fundamentals.

That being said, the fans offer up a solid argument as well. Not only does the low price mean you get more shares for your money, but hefty returns are also on the table. Even seemingly insignificant share price appreciation can result in colossal percentage gains that other more well-known or expensive names aren’t as likely to deliver.

The nature of these investments presents somewhat of a dilemma. How are investors supposed to separate the penny stocks that are ready to take off on an upward trajectory from those set to remain down in the dumps?

To help with the due diligence process, we used TipRanks’ database to zero in on only the penny stocks that have received bullish support from the analyst community. We found two that are backed by enough analysts to earn a “Strong Buy” consensus rating. Not to mention each offers up massive upside potential, as some analysts see them climbing to $10, or more.

Aptinyx, Inc. (APTX)

We’ll start with Aptinyx, a company in the biopharma industry. Aptinyx works on the treatments for brain and nervous system disorders, developing new synthetic small molecule medications for commercialization. The company has a proprietary NMDA receptor modulator discovery platform, which enables a novel approach to the targeted disorders.

Aptinyx’s research pipeline currently has three compounds in the clinical stages, all in Phase 2 trials. NYX-458 is a potential treatment for Parkinson’s disease and Lewy body dementia, two serious central nervous conditions of aging. Preclinical and Phase 1 studies showed robust activity in rodent models and favorable safety tolerance in human patients; the Phase 2 clinical trial will focus on patients with mild levels of cognitive impairment and dementia.

NYX-783, the second compound in the pipeline, is being studied for its efficacy in treating post-traumatic stress disorder. A Phase 2 exploratory study showed positive results in symptom reduction in just 4 weeks of treatment, and the FDA has granted this drug a Fast Track designation.

Finally, NYX-2925, the lead drug candidate in the pipeline, is under study as a treatment for two conditions: fibromyalgia, and diabetic peripheral neuropathy (DPN). These are both chronic, painful, conditions, and NYX-2925 has demonstrated reduced pain levels in patients during earlier stage testing. The DPN study, like NYX-783 above, has FDA Fast Track designation.

Based on the potential of the company's drug candidates, and its $2.84 share price, several members of the Street believe that now is the time to get in on the action.

Among the bulls is Leerink Analyst Mark Goodman who sees a string of catalysts ahead to boost the stock.

“The stock has been trending lower during the past several months mainly due to the lack of near-term catalysts, but we believe that investors will start to get much more focused on the name moving into 2H21 ahead of the multiple data points in 2022. We continue to have a positive view of Aptinyx's focus on NMDA receptor modulators and its pipeline opportunities… Aptinyx has a strong drug discovery platform plus a library of >1,000 identified compounds, which should enable continued pipeline expansion and sustain long-term growth,” Goodman explained.

To this end, Goodman rates APTX an Outperform (i.e. Buy) along with a $12 price target. Investors could be pocketing gains of 324%, should Goodman's thesis play out as expected. (To watch Goodman’s track record, click here)

It’s not often that the analysts all agree on a stock, so when it does happen, take note. APTX’s Strong Buy consensus rating is based on a unanimous 7 Buys. On top of this, the average price target is $10.67, suggesting robust growth of ~277% from current levels. (See APTX stock analysis on TipRanks)

Axcella Health (AXLA)

The next penny stock we’re looking at, Axcella, is another biotech company. Axcella is using endogenous metabolic modulators (EMMs) as a jump-off to approach new treatments for complex diseases, including non-alcoholic steatohepatitis (NASH, or fatty liver disease), along with overt hepatic encephalopathy (OHE). These are both serious liver conditions, and can have cascading consequences on the whole body.

For both tracks, Axcella has completed early clinical studies and is beginning Phase 2 trials. AXA1125, the drug candidate under development to treat NASH, started the EMMPACt Phase 2b clinical trial in May of this year. The study will enroll a total of 270 patients, and will stratify them by the presence or absence of type 2 diabetes, and important complicating factor.

Also in May, Axcella announced positive clinical data on AXA1665, the drug being studied as a treatment for OHE. The data showed that two different drug doses proved safe and well-tolerated, and showed positive results when compared to placebo. Both AXA1665 and AXA1125 have had their IND application cleared by the FDA.

AXLA's strong pipeline has scored it substantial praise from Chardan analyst Keay Nakae.

“We view Axcella's leading asset, AXA1665, as likely to exceed the benefits provided by the current standard of care therapies lactulose and rifaximin in HE, due to its clean safety/tolerability profile and ability to target more aspects of HE than simply blocking ammonia absorption in the gut. Beyond AXA1665, we also anticipate success for AXA1125 in NASH; the NASH market is crowded with potential therapeutics but is also a very large commercial opportunity that we anticipate will allow for the success of multiple market entrants. We expect AXA1125 to thrive as a base-level therapy due to its impressive safety/tolerability profile as a result of its mechanisms of action, with potential to move forward in pediatric NASH sooner than competitors due to this safety profile,” the 5-star analyst noted.

In line with his optimistic approach, Nakae gives AXLA shares a Buy rating and his $10 price target suggests ~186% potential upside for the coming year. (To watch Nakae’s track record, click here.)

Other analysts are on the same page. With 3 additional Buy ratings, the word on the Street is that AXLA is a Strong Buy. The shares are priced at $3.50, and the $11.25 average price target suggests it has ~222% upside ahead of it. 

https://finance.yahoo.com/news/2-strong-buy-penny-stocks-153512732.html

Wedding boom is on in the US as vendors scramble to keep up

 Couples in the U.S. are racing to the altar in a vaccination-era wedding boom that has venues and other vendors in high demand.

With restrictions on large gatherings loosening, wedding planners and others who make the magic happen said they’ve started pushing their bookings into late 2022 and early 2023.

Mannequins in wedding gowns appear in a window display at a bridal store in Nogales, Ariz., on March 15. (AP Photo/Suman Naishadham)

“We’ve run out of trucks for some dates this year and that hasn’t happened before,” said Ben Goldberg, co-founder and president of the New York Food Truck Association. “Our phones have been ringing off the hook with clients looking to have the weddings they had to put off during COVID.”

Also contributing to the rush are couples who went ahead and got hitched during stricter pandemic times with few or no guests and are now on their second go-arounds with larger groups. They’re competing for services with those who had always intended to marry this year.

“We’re seeing a lot of last-minute bookings with shorter planning windows,” said Anna Noriega, who owns the luxury Alorè Event Firm in Miami. “With vaccinations becoming more prevalent and on-site COVID testing available for events, we’ve seen an uprise in guest counts and a push for bookings.”

Namisha Balagopal, 27, in Emeryville, California, is among the double brides.

She and Suhaas Prasad, 33, met in 2014 and got engaged in May 2019. They planned a traditional South Asian Indian wedding last August in Utah, where Balagopal grew up, with 320 guests and events over five days. But they couldn’t make it happen under pandemic restrictions. They decided on a small sunset ceremony that month with fewer than 10 people in attendance on Muir Beach near San Francisco. It’s where they had their first date and where Prasad proposed.

Now, their big celebration is on for Aug. 15 outdoors at their original venue in Park City, Utah, with about 230 guests and events over several days, including seven clothing changes for bride and groom. Many of their closest loved ones in India aren’t permitted to travel to the U.S.

“It’s just a really big part of our culture,” Balagopal said of the extravaganza. “In the end, it was really important to our parents.”

She’s mostly beyond the frustration phase of being a pandemic bride.

“The wedding is going to be so much fun. It’s just delayed gratification at this point,” Balagopal smiled.

The boom is on in bridal and bridesmaids dresses, too.

The budget-friendly David’s Bridal chain, with 282 stores in the U.S. and more in the UK, Canada and Mexico, has 300,000 dresses in stock due in part to the wedding drought of 2020.

“Moving forward it’s going to be an unprecedented wedding season this year,” said Maggie Lord, a vice president at David’s whose online wedding planning guide, Rustic Wedding Chic, was acquired by the company. David’s has been tracking broad wedding data through the pandemic.

Anita Galafate checks wedding dresses in a shop in Rome on March 9.

“Couples are getting super creative and having Thursday night ceremonies or Friday afternoon ceremonies just because of the amount of people getting married this year,” Lord said. “We do know that 90 percent of brides this year are looking to have their weddings at outdoor venues,” where there are fewer restrictions.

Lord said the pandemic has helped normalize non-traditional aspects of weddings: an end to passed hors d’oeuvres and buffets, for instance, more livestreaming to accommodate travel restrictions, and more online planning and shopping.

Competition for vendors has some upping their prices. “They know they have customers who will pay it,” she said. “Wedding vendors are making up for a year of limited if no work at all.”

Anna Price Olson, associate editorial director for Brides magazine, said many vendors in the wedding industry are small businesses.

“They’re trying to meet the demand of new clients and clients who have postponed,” she said. “In order to do so, in many cases, they’re having to charge more. They’re having to hire additional resources, bring staff members back. Also the cost of goods is increasing. There are only so many linens, only so many rentals and only so many flowers that were planted this past season.”

One thing’s for sure, Lord said: Brides and grooms are “bringing back the big wedding, with guest lists that are a little bit more curated and maybe not 300 people on a crowded dance floor.”

Tirusha Dave is the owner and CEO of the upscale wedding planning company Bravura Brides used by Balagopal. She handled 10 weddings in 2019, with just three in 2020. Dave already has 11 weddings booked this year with 250 to 300 guests planned at each.

“I think everybody’s ready for things to bounce back, but just in a safe way,” she said.

Lord’s online planning site has far more vendors with bookings two years out, rather than the more traditional 12-month planning period as couples hold out for venues they want and seek to distance their special days from the pandemic altogether.

Justin Warshaw is the creative director and CEO of the global Justin Alexander Group, a bridal design and manufacturing house with five core brands and more under licensing and white-labeling arrangements.

He’s seen wedding dress bookings increase by 593% from April 1-May 15 2020 compared to the same period this year. Eighty-eight percent of his 2,200 retailers in 80 countries are now open and operating, with the U.S. his largest market.

Tom Li, and his fiancé, Leah Li, right, pose for photos taken by Ella Chang, left, at Gas Works Park in Seattle on May 10. (AP Photo/Ted S. Warren)

Comparing January through May 2019, before the pandemic began, with January through May this year, Warshaw experienced a 40% increase in sales of made-to-order gowns in the U.S.

“A lot of that has to to do with pent-up demand and also what we envisioned would happen: So many COVID couples turning into COVID engagements, turning into weddings,” said Warshaw, who’s among the newly engaged. “With the optimism from vaccinations, people want to celebrate with family and friends, and get on with their lives.”

Chappall Gage, who with his mother runs Susan Gage Caterers in Washington, D.C., has seen a boom in special events overall, with 30% to 40% of their weekend business in weddings.

“It’s the one big event that’s coming back quickly,” he said. “Right now we’re at this transition point where micro-weddings are ending and now people are starting to get comfortable doing larger weddings. When the mayor announced she was allowing dancing at weddings I could hear an audible cheer from the city.”

https://apnews.com/article/wedding-vendors-scramble-vaccination-era-6d386032347b37161a125e6ea5729b73

Germany starts rolling out a digital EU vaccination pass

 Germany on Thursday started rolling out a digital vaccination pass that can be used across Europe as the continent gets ready for the key summer travel season.

The country’s health minister said starting this week vaccination centers, doctors practices and pharmacies will gradually start giving out digital passes to fully vaccinated people. The CovPass will let users download proof of their coronavirus vaccination status onto a smartphone app, allowing them easy access to restaurants, museums or other venues that require proof of immunization.

The vaccination passport should be available to everyone in Germany who is fully vaccinated by the end of this month, Health Minister Jens Spahn said.

“The goal is that this certificate can also be used in Helsinki, Amsterdam or Mallorca,” Spahn told reporters in Berlin.

People who have been fully vaccinated will either get a letter with a QR-code they can scan with their phones or they can contact their doctors or pharmacies to retroactively get the digital pass.

“By doing so, we in the European Union are setting a cross-border standard that doesn’t exist elsewhere in the world yet,” Spahn said, adding that the digital vaccination pass is an important step for the revival of international tourism.

The country’s disease control agency, the Robert Koch Institute, reported Thursday that 47% of the population, or about 39.1 million people, have gotten one shot. Almost 24%, or 19.9 million people, are fully vaccinated.

On Wednesday, almost 1.3 million people received a vaccine jab, the second highest daily number ever.

Also Thursday, Germany’s committee on vaccinations, known as Stiko, recommended that children aged 12 to 15 only receive the shot if they have certain illnesses such as obesity, chronic lung or heart diseases, dpa reported. Healthy teenagers can, however, also be vaccinated if they, their parents and doctors decide to do so.

The German panel’s opinion differs from the European Medicines Agency, which last month recommended expanding the use of the Pfizer-BioNTech vaccine to children 12 to 15, a decision that offers younger and less at-risk populations across the continent access to a COVID-19 shot for the first time.

The head of Stiko, Thomas Mertens, said while youths are very well protected by the vaccine, only few children and teenagers had participated in medical studies and possible severe side effects could therefore not be excluded.

Mertens said the recommendation was also based on the fact that very few children fall severely ill with COVID-19 as opposed to older people.

https://apnews.com/article/europe-germany-coronavirus-pandemic-technology-government-and-politics-03043240f9780a2504dd5bb98d90cb5f

COVID-19 vaccine and cell and gene players are tackling the viral vector bottleneck

 Bristol Myers Squibb’s cancer cell therapy Breyanzi. Johnson & Johnson and AstraZeneca’s recombinant COVID-19 vaccines. Novartis’ gene therapy Zolgensma. All of these, plus many others, are united by a common element of production—viral vectors. That’s creating big problems for the industry.

Viral vectors are engineered viruses used to deliver gene therapies, gene-modified cell therapies—such as Novartis’ CAR-T Kymriah—and certain vaccines. And their shortage is already upon us, thanks to manufacturing approaches that simply haven’t kept pace with the advance of cell and gene therapies.

Add to that the role of viral vectors in AstraZeneca and Johnson & Johnson’s COVID-19 vaccines, plus a looming wave of new drug approvals, and the bottleneck is due to tighten even more. That's unless regulators, biopharmas and contract manufacturers step up in a big way, GlobalData said in a recent report.

Many CDMOs in the vector field have been sounding the alarm. When VIVEbiotech sketched out a 400% increase in vector production in Spain, CEO Gurutz Linazasoro cited a "significant increase" in demand.

And Byung Se So, CEO of fledgling CDMO Matica, put it even more bluntly in a recent statement: “The global demand for virus products continues to outpace the capacity for production.”

Even as cell and gene therapies advance rapidly through the clinic and reach the market, viral vector manufacturing is "maybe a generation or two behind something like monoclonal antibody manufacturing," Fiona Barry, associate editor at GlobalData’s PharmSource, told Fierce Pharma. The industry's monoclonal antibody production quickly became “more standardized” and “partially automated,” she said.

"That’s the kind of revolution that we need for viral vectors,” she added.

As it stands, strained capacity, inefficient manufacturing and the need for complex, specialized facilities have all played a role in the shortage of viral vectors, GlobalData said. 

All told, 14 gene therapies, gene-modified cell therapies and recombinant vector vaccines are already approved and sold in Europe, Japan, the United Kingdom and the United States, according to the report.

Over the next six years or so, 100 or more vector-hungry cell and gene therapies are expected to cross the regulatory finish line. Once those therapies launch at commercial scale, the viral vector bottleneck will really close in, Barry said.

Meanwhile, the “more immediate stress” on the viral vector supply chain is the demand stemming from COVID-19 vaccines—including Russia’s Sputnik V and Cansino’s Ad5-nCoV—GlobalData points out. While mRNA shots from Pfizer and Moderna were the first to reach the public in the U.S., many other countries are depending on vector-based shots, which are cheaper to make, easier to produce and simpler to transport than mRNA vaccines.

While initially turbulent vaccine rollouts have smoothed in places like the U.S. and the U.K., that certainly isn’t true for much of the world, Barry noted. The world still has "billions of doses to go," she said.

To crank out enough viral vectors to meet “near-future need,” the industry would need 3 to 6 billion liters of bioreactor capacity a year, GlobalData figures. It currently has less than 1% of that.

Addressing the shortage will take a two-pronged approach, Barry estimates. The first, and perhaps the most straightforward solution, is for companies simply invest and scale up, she said.

Major CDMOs—such as Catalent, Thermo Fisher and Lonza—as well as Big Pharmas Johnson & Johnson, AstraZeneca, Bristol Myers Squibb, Gilead Sciences and Novartis have all been making moves in this arena, whether through facility and equipment investments or M&A.

Take Thermo Fisher, for instance. The CDMO made its first gene therapy foray—but certainly not its last—back in 2019, when it snapped up viral vector producer Brammer Bio for $1.7 billion. Then in January, Thermo Fisher laid out $878.2 million in cash for Novasep’s viral vector manufacturing division, Henogen. 

Novasep, for its part, inked a multiyear pact with AstraZeneca in November to chip in on large-scale production of adenovirus vector for its COVID-19 vaccine.

Major drugmakers such as Gilead and Sanofi have gotten in on the action, too. In July 2019, Gilead’s Kite Pharma blueprinted a 67,000-square-foot facility in California to churn out viral vectors for use in CAR-T therapies like its own Yescarta. Until then, the company had relied on contractors.

Later that same year, Sanofi, lagging its peers in the gene therapy arena, said it would retrofit a vaccine plant in France to work on vector-based gene therapies.

Still, it's not "enough to just build out, build out, build out,” Barry explained. The industry also needs to introduce serious improvements to manufacturing efficiency and getting there will take expertise and innovation. Regulators getting behind standardized viral platforms could be another boon, she added.

Biopharmas and contract manufacturers are already hard at work on upgrades to the viral vector manufacturing process, GlobalData noted. Change is needed both upstream and downstream, Barry said, with the upstream phase—the stage at which cells are grown—particularly lacking. Changes there could come in the form of better cell lines, suspension cultures and upsized scale.

Meanwhile, the FDA’s Peter Marks, who directs its Center for Biologics Evaluation and Research, has previously voiced support for standardized viral vector platforms, Barry pointed out. What many experts want to see is the introduction of standardized platforms that biopharma developers and CMOs could deploy. That could in turn shorten R&D time and make it easier for contractors to switch between products, she said.

https://www.fiercepharma.com/manufacturing/vexed-by-vectors-how-covid-19-vaccine-and-cell-and-gene-players-are-meeting-viral

Moderna, BioNTech Slip on Reports of Heart Inflammation Cases

 Shares of Moderna (NASDAQ:MRNA) and BioNTech (NASDAQ:BNTX) slipped in Thursday’s trade after the CDC said the number of cases of a heart inflammation condition in 16-to-24-year-olds was higher than expected after they received their second dose of Pfizer Inc (NYSE:PFE) or Moderna’s Covid-19 vaccines.

Moderna was down 2.5% while BioNTech fell 1.5%. Pfizer was still holding up around its gain of 1% after slipping below it for some time.

BioNTech and Pfizer have a tie-up to make and market the vaccine, built on the former’s mRNA technology.

The U.S. Centers for Disease Control and Prevention cited preliminary data from its vaccine safety monitor system while disclosing the findings.

There have been 275 reported cases of myocarditis or pericarditis, which are inflammation conditions of the heart, in people ages 16 to 24 as of May 31, CNBC reported. This was according to a CDC presentation prepared for a U.S. Food and Drug Administration advisory panel meeting Thursday.

https://finance.yahoo.com/news/moderna-biontech-slip-reports-heart-111106812.html

FDA Extends Shelf Life of Johnson & Johnson COVID-19 Vaccine

 Johnson & Johnson said on Thursday the U.S. Food and Drug Administration has extended the shelf life of its single-shot COVID-19 vaccine from three months to four-and-a-half months.

The health regulator's decision was based on data from ongoing studies, which demonstrated that the vaccine is stable at 4.5 months when refrigerated at temperatures of 36 to 46 degrees Fahrenheit (2 to 8 degrees Celsius), the drugmaker said.

JNJ said vaccine providers should visit its website to check expiration dates of vaccines currently available in the country.

https://www.usnews.com/news/top-news/articles/2021-06-10/fda-extends-shelf-life-of-johnson-johnson-covid-19-vaccine