Can Sarepta's 'Remarkable' Gene Therapy Pull Shares Out Of The Dumps?

 An experimental gene therapy from Sarepta Therapeutics (SRPT) showed "remarkable" stability at two years in muscular dystrophy patients, an analyst said Friday as SRPT stock edged higher.

The biotech company tested a low dose of its gene therapy, dubbed SRP-9003, in three patients with limb-girdle muscular dystrophy. This form of muscular dystrophy is caused by deficient levels of beta-sarcoglycan, a protein tied to muscle function.

Patients showed an increase in beta-sarcoglycan levels after two years. On average, the protein was expressed in 54% of tissues after two years. That increased from 36% at 60 days after receiving the gene therapy. On a scale measuring muscle function, patients showed an average improvement of 5.7 points at 18 months. That score was still intact at the two-year mark.

Needham analyst Chad Messer said the results showed "remarkable stability."

"The three patients continue to demonstrate improvements from baseline in all functional measures including time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test," Messer said in a report to clients. He maintained his buy rating and 166 price target on SRPT stock.

SRPT Stock Rises On Gene Therapy Test

In morning trading on today's stock market, SRPT stock edged up a fraction near 83.20. Earlier, shares popped 3.6%.

The long-term functional data "bolsters confidence" in Sarepta's gene therapies, SVB Leerink analyst Joseph Schwartz said in his note to clients.

Sarepta also unveiled results for a group of patients who received a higher dose of the gene therapy a year ago. All three patients showed increases in beta-sarcoglycan and functional improvements. At one year, they showed an average 4-point improvement from baseline on a key scale.

"We believe these new data are incrementally positive, and support the hypothesis that gene therapy can lead to function improvements in patients with muscular diseases," Mizuho Securities analyst Difei Yang said in a note. She has a buy rating and 160 price target on SRPT stock.

Gene Therapies Share A Component

The news helps SRPT stock, which tumbled to a two-year low in January after its experimental gene therapy for Duchenne muscular dystrophy produced mixed results in a test called Study 102. This form of gene therapy treats a condition due to lacking dystrophin protein. The gene therapy known as SRP-9001 produced large amounts of dystrophin, but patients didn't show functional improvements.

But the two gene therapies share a key component, Schwartz said.

"We see incrementally positive read-though from (Thursday's) update not only to Sarepta's broader limb-girdle muscular dystrophy portfolio, but also to Sarepta's SRP-9001, which has been knocked off its pedestal recently following disappointing Phase 2 Study 102 results in January," he said.

Schwartz kept his outperform rating on SRPT stock.

https://www.investors.com/news/technology/srpt-stock-jumps-remarkable-2-year-data-muscular-dystrophy-gene-therapy/

On first day of trading, Finch Therapeutics raises $128M in upsized IPO

 Finch Therapeutics, the Somerville, Mass. startup founded in 2014 to address a wide variety of diseases with microbiome treatments, has raised $128 million in its first day of trading on the Nasdaq.

In mid-day trading, shares of the company (Nasdaq: FNCH) were trading over $19, up from the offering price of $17. The company sold 7.5 million shares in the offering — 1.3 million more shares than it had anticipated.

Finch had initially filed to raise $100 million, with BofA Securities, Jefferies and Evercore ISI backing the listing.

Finch is testing microbiome therapies to treat to recurrent C. difficile, Autism spectrum disorder, ulcerative colitis and Crohn’s disease. The company's primary method involves repurposing donated fecal samples from a nonprofit stool bank its CEO, Mark Smith, co-founded, then growing specific microbes through fermentation. The resulting bacteria are then dried, ground into a powder and placed into a pill capsule to be swallowed — the method by which Finch's lead drug candidate, CP101, is meant to treat C. difficile.

Phase 2 trials for CP101, which will ultimately include about 70 participants, are expected to conclude next month. Finch plans to enter Phase 3 trials for the drug in mid-2021, fueled by a $90 million Series D round it closed in September.

Finch is the fourth Massachusetts biotech to go public this year. Decibel Therapeutics, Vor Biopharma and Cullinan Oncology all made their stock market debuts in January and February. Cullinan Oncology, which was the first of those to go public, was trading at $42 in mid-day trading on Friday, twice what shares were initially priced at in its Jan. 12 IPO. Cullinan has a market cap of $1.814 billion.

Vor has also doubled the price of each share since it began trading on Feb. 9; Decibel shares, by contrast, have declined by 16 percent since the company's Feb. 12 IPO.

https://www.bizjournals.com/boston/news/2021/03/19/finch-therapeutics-raises-128m-in-upsized-ipo.html

Calling robot doctors to ER to reduce COVID spread

 The digi-doctor is in.

Dr. Spot, a dog-like mobile robot programmed to triage hospital patients, is the newest front-line health-care worker to join the fight against COVID-19. 

And while at first glance this four-legged creature might look like something out of your worst nightmare, people are actually doggone happy about it. 

“People are very positive and accepting of robotic systems in health-care settings, particularly during the pandemic,” MIT assistant professor of mechanical engineering Giovanni Traverso told The Post.

In a new study out this month, Traverso and colleagues Peter Chai and Henwei Huang found that patients are widely receptive to receiving medical attention from robots designed to evaluate symptoms in a contact-free way. 

They’re even willing to let robo-docs like Spot — made of aluminum, plastic and circuit boards — perform minor procedures on them, such as assessing their vital signs, taking a nasal swab or placing an intravenous catheter. 

“Early on in the pandemic we wanted to help protect the health-care workforce from the virus by limiting their exposure to potentially COVID-infected patients,” Traverso said.

“We wondered if we could do that by incorporating robotic systems in health-care environments, and if patients would be willing to engage with robots during their evaluations,” he said. 

Dr. Spot, a four-legged mobile robot, helps reduce the spread of COVID-19 by providing health-care services to sick patients in hospitals.

YouTube

To test their theory, researchers first conducted a nationwide survey of 1,000 people to analyze their thoughts on receiving medical services from robots. 

“We found that folks across the nation were willing to engage with robots, particularly systems that facilitate telehealth and systems that facilitate measuring vital signs like heart rate, respiratory rate and oxygen levels,” Traverso said. 

The researchers then teamed up with engineering and robotics design firm Boston Dynamics to create Dr. Spot. 

“It takes a few months to build a robot,” Marco da Silva, principal roboticist, told The Post. 

The company’s offerings start around $74,500, with the option of additional customizations that can cost over $20,000 each, according to its Web site.

“It took another six weeks to make the payload for Dr. Spot,” da Silva said of the specialized software and modifications needed to give the bot the abilities to perform health-care assessments on patients. 

Dr. Spot is equipped with a number of sophisticated cameras and computer systems that can identify a person’s face even if they’re wearing a mask. It can also assess body temperature, breathing and pulse rates, and uses special lens filters to determine blood oxygen saturation. 

Researchers then introduced the robot to 40 patients in the emergency department at Brigham and Women’s Hospital in Boston. 

For this study, the clinicians mounted an iPad to Spot’s base. The tablet displayed a real-time person-to-person video that allowed doctors and nurses to conduct telehealth interviews with patients while they operated the robot with a remote controller from a distance. 

“Overall, patients in the emergency department responded very favorably to Dr. Spot, especially because it reduces the risk of person-to-person exposure to COVID,” Traverso said. 

“Most people reported that interacting with a health-care provider via a robotic system was similar to in-person interaction with a physician.”

More than 90 percent of the patients reported that their interaction with the robot was satisfactory, and said they would be willing to interact with more robotic systems in the future, according to the study.

“Robotics, to some extent, are used in hospital settings already,” Traverso noted. 

“But in the setting of COVID-19, we’re seeing that robotic systems really could play a significant role in health care based on people’s high acceptance.”

https://nypost.com/2021/03/19/mit-robot-doctors-see-patients-fight-spread-of-covid-19/

Cause of Rare Blood Clotting Linked to AstraZeneca Vaccine Reported Found

 Scientists in Europe said they had identified a mechanism that could lead the AstraZeneca PLC vaccine to cause potentially deadly blood clots in rare instances as well as a possible treatment for it.

Two teams of medical researchers in Norway and Germany have independently found that the vaccine could trigger an autoimmune reaction causing blood to clot in the brain, which would offer an explanation for isolated incidents across Europe in recent weeks.

Several European countries briefly halted their rollouts of the vaccine this week after more than 30 recipients were diagnosed with the condition known as cerebral venous sinus thrombosis or CVST. Most of the people affected were women under the age of 55.

The issue affected a tiny portion of those who had received the shot however, and after investigating, the European drugs regulator ruled that the benefits outweighed the potential risks of the vaccine, and recommended vaccinations resume.

Some countries, such as Germany, France and Italy, resumed vaccination with AstraZeneca's shot on Friday, with an added warning that it could be linked to blood clotting. The French healthcare authority, which recorded three cases of CVST connected to the vaccine, advised the government on Friday to only administer the shot to people older than 55.

Others, including Norway, Sweden and Denmark, said they needed more research before restarting their rollouts. Norway registered three cases of CVST, one of them fatal. The country vaccinated around 120,000 people with the shot. Finland suspended the use of AstraZeneca on Friday, after recording two cases of what the authorities called unusual blood clotting.

Pål André Holme, a professor of hematology and chief physician of the Oslo University Hospital who headed an investigation into the Norwegian cases, said his team had identified an antibody created by the vaccine that was triggering the adverse reaction.

"Nothing but the vaccine can explain why these individuals had this immune response," Prof. Holme said.

Norway's health authority cited the findings when announcing that it would not resume the vaccination.

A team of German researchers around Andreas Greinacher, professor of transfusion medicine at the Greifswald University Clinic, said they had independently came to the same conclusion as Prof. Holme in a statement and a press conference on Friday.

In Germany, 13 cases of CVST were detected among around 1.6 million people who received the AstraZeneca vaccine. Twelve patients were women and three died.

The German researchers, who coordinated with colleagues in Austria, Ireland and Britain, said in a statement that patients who show symptoms four days after vaccination, such as headaches, dizziness or impaired vision, could be quickly diagnosed with a blood test. Prof. Greinacher said the news meant that people should not fear the vaccine.

"Very, very few people will develop this complication," Prof. Greinacher said in a press conference Friday. "But if it happens we now know how to treat the patients."

He said that, after a swift diagnosis, the condition could be treated in any mid-sized hospital.

The German government said it was examining the findings, but stuck to its decision to resume use of the AstraZeneca vaccine.

AstraZeneca didn't immediately react to a request for comment. Neither did the medicines regulators in Britain, Germany, Austria and The Netherlands, where vaccinations using the AstraZeneca vaccine have either resumed or weren't suspended this week.

The European Medicines Agency, or EMA, which regulates medicines for most European countries, said that it had assessed the cases from Germany and Norway and discussed them with the relevant national authorities.

A spokesperson for EMA said that the vaccine may be associated with very rare cases of blood clots, including CVST, but that the benefits of the vaccine outweigh that risk.

"A causal link with the vaccine is not proven, but is possible and deserves further analysis," the spokesperson said in a statement.

Neither the German nor the Norwegian findings were published or peer reviewed. Prof. Greinacher said he had submitted his findings for publication to the British medical journal The Lancet.

The German Society for Thrombosis and Hemostasis Research reviewed Prof. Greinacher's work and issued a statement Friday advising physicians how to diagnose and treat the condition should it arise in vaccine recipients.

Dr. Robert Klamroth, deputy-chairman of the Society for Thrombosis and Hemostasis Research, said the rare autoimmune reaction occurred more frequently in Germany because the country initially only authorized the vaccine for people younger than 64. Britain, which had fewer incidents but vaccinated many more people, was predominantly giving the shot to older recipients.

Once diagnosed, the condition should be treated with blood thinning medication and immunoglobulin, which targets the antibody that causes the problem.

"We believe the most likely hypothesis is that this particular vaccine is causing a rare autoimmune reaction that triggers antibodies, which then interact with the platelets, but we don't know why this is happening," Dr. Klamroth said.

https://www.marketscreener.com/quote/stock/ASTRAZENECA-PLC-4000930/news/AstraZeneca-nbsp-Scientists-Say-They-Found-Cause-of-Rare-Blood-Clotting-Linked-to-AstraZeneca-Vacc-32741181/

SPAC Healthcare Merger II files for a $300 million IPO

 Healthcare Merger II, a blank check company led by Steven Shulman and William Sanger targeting the healthcare industry, filed on Thursday with the SEC to raise up to $300 million in an initial public offering.

The New York, NY-based company plans to raise $300 million by offering 30 million units at $10. Each unit consists of one share of common stock and one-third of a warrant, exercisable at $11.50. At the proposed deal size, Healthcare Merger II would command a market value of $384 million.

The company is led by Co-CEO and Director Steven Shulman, the Managing Partner of Shulman Family Ventures and former CEO of Magellan, and Co-CEO and Director William Sanger, the Co-Owner and Chairman of EA Health Solutions, Managing Partner of Sanger Holdings, and co-founder and Managing Partner of BIDON Companies.

Shulman's previous SPAC, Healthcare Merger Corp., completed its acquisition of SOC Telemed (TLMD; -28% from $10 offer price) this past October.

Healthcare Merger II was founded in 2020 and plans to list on the Nasdaq under the symbol HCXXU. The company filed confidentially on September 15, 2020. Cantor Fitzgerald is the sole bookrunner on the deal.

Relevant Profile: HCXXU

https://www.renaissancecapital.com/IPO-Center/News/79622/Healthcare-SPAC-Healthcare-Merger-II-files-for-a-$300-million-IPO

Why Idera Is Crashing Today

 Shares of Idera Pharmaceuticals (NASDAQ:IDRA) were crashing 63.5% at 10:49 a.m. EDT on Friday. The plunge came after the company announced disappointing results Thursday from a late-stage study of tilsotolimod in combination with Bristol Myers Squibb's Yervoy in treating advanced melanoma. 

Idera had hoped that the tilsotolimod-Yervoy combo would sail through its pivotal study. That didn't happen.

The company reported an objective response rate (the percentage of patients with tumor-size reduction of a predefined amount) for patients taking the combo of only 8.8%. Patients receiving Yervoy by itself had an objective response rate of 8.6%.

Vincent Milano, Idera Pharmaceuticals CEO, said that he and his team "are surprised and disappointed" by the results from the late-stage study. What wasn't a surprise was the reaction from Wall Street. Multiple analysts quickly downgraded the biotech stock.

What's next for Idera? The company stated that it's evaluating whether or not to keep the late-stage study going to see if the tilsotolimod-Yervoy combo can meet its overall survival endpoint. There's still at least a sliver of hope that this could happen and set the stage for a path to regulatory approval. Idera will also move forward with its phase 2 study of tilsotolimod and Yervoy in treating microsatellite stable colorectal cancer.

https://www.fool.com/investing/2021/03/19/why-idera-pharmaceuticals-stock-is-crashing-today/

Cancer biotech Instil Bio raises deal size by 25% ahead of $312 million IPO

 Instil Bio, an early stage biotech developing immune cell therapies for cancer, raised the proposed deal size for its upcoming IPO on Thursday.

The Dallas, TX-based company now plans to raise $312 million by offering 16 million shares at a price range of $19 to $20. The company had previously filed to offer 13.9 million shares at a range of $17 to $19. At the midpoint of the revised range, Instil Bio will raise 25% more in proceeds than previously anticipated.

Instil Bio is developing a cell therapy pipeline of autologous tumor infiltrating lymphocyte (TIL) therapies for the treatment of cancer. Its lead TIL candidate, ITIL-168, is being developed for the treatment of advanced melanoma. The company plans to submit an IND and initiate a Phase 2 trial in the 2H21, which the company believes could support a BLA submission in 2023. Instil plans to initiate Phase 1 trials of ITIL-168 in additional indications with unmet medical need in the 1H22.

Instil Bio was founded in 2018 and plans to list on the Nasdaq under the symbol TIL. Morgan Stanley, Jefferies and Cowen are the joint bookrunners on the deal. It is expected to price during the week of March 15, 2021.

Relevant Profile: TIL

https://www.renaissancecapital.com/IPO-Center/News/79608/Cancer-biotech-Instil-Bio-raises-deal-size-by-25-ahead-of-$312-million-IPO