Santhera scores in Duchenne

 Santhera has long claimed that, in vamorolone, it has a safer alternative to steroids for the treatment of Duchenne muscular dystrophy. This was borne out today by topline data from the pivotal Vision-DMD study, which compared vamorolone versus placebo and the steroid prednisone. The trial met its primary endpoint, time to stand, with vamorolone 6mg statistically superior to placebo at 24 weeks; a 2mg dose also hit on this measure. Also importantly, vamorolone was linked with fewer adverse events than prednisone, while the 6mg dose was similar on efficacy, Santhera said. Steroids are a mainstay of DMD therapy but can cause side effects including stunted growth and weight gain. Santhera plans to file vamorolone in the US in the first quarter of 2022. The Swiss group reckons vamorolone could bring in over $500m at peak in DMD, putting it among the top-selling therapies for the disorder, according to Evaluate Pharma. The company intends to market the drug alone in the US and EU; this could prove trickier than gaining approval, considering Santhera only has SFr26.4m ($29.4m) of available cash, including up to SFr18m that it can draw down from a Highbridge loan. The group’s stock shot up 50% this morning – still, it is only worth SFr46m

https://www.evaluate.com/vantage/articles/news/snippets/santhera-scores-duchenne.

Gene editing biotech Verve indicates swift swerve onto Wall Street, eyes $100M IPO

 Verve Therapeutics, on the march toward the clinic with a next-gen heart drug, wants a piece of the ever-growing biotech IPO pie.

Verve debuted just over two years ago with $58.5 million and a goal to bring one-and-done gene editing treatments to heart disease.  

Then, in January this year, the company began its march toward the clinic with a treatment for a genetic form of high cholesterol, raising an extra $94 million for its series B and work on the asset.

The treatment, VERVE-101, is a base editor, meaning it doesn’t cut DNA like CRISPR gene editing systems do. Instead, it changes one base, or letter, in the genome to a different one without affecting the letters around it. Its first target? An inherited form of high cholesterol called heterozygous familial hypercholesterolemia, or HeFH.

People with HeFH have a gene mutation in the liver that causes very high cholesterol levels and leads to heart attacks or strokes relatively early in life. VERVE-101 is designed to cut those cholesterol levels by blocking the PCSK9 gene.

With a target and a clinical plan, the young biotech is now seeking a quick turn onto the public markets, gunning for the standard $100 million IPO, though this could swell in the summer heat of biotech IPOs as many have before it.

Verve will use the cash toward its ongoing IND-enabling studies for VERVE-101, with plans to submit an IND next year, according to its SEC-1 filing.

The treatment is a lipid nanoparticle comprising a guide RNA to find the target letter on the PCSK9 and an mRNA that changes an A base in the gene to a G, thus inactivating the gene and lowering cholesterol.

Verve reported data last June showing the approach worked in monkeys. Two weeks after injection, the study found the treatment switched off the PCSK9 gene in 67% of liver cells, causing an 89% drop in PCSK9 protein in the monkeys’ blood and a 59% dip in LDL, or “bad,” cholesterol in their blood. Now, six months down the line, those changes have held.

Verve is starting with HeFH because it’s a serious disease with a clear genetic cause that’s hard to treat. After this first indication, though, Verve plans to branch out into bigger patient populations, first into “the more garden variety” atherosclerotic heart disease and then into the preventive setting.

Verve hopes a one-time gene editing injection could eliminate the hurdles facing cholesterol-lowering statins and other medicines. These include poor adherence, high costs, limited access and lack of insurance, Kathiresan said. And though approval and commercialization are still far out, Verve is already learning how it might roll out its treatments.

As Verve moves VERVE-101 toward the clinic, it will keep plugging along at its preclinical pipeline. Besides PCSK9, it has identified seven other genes, such as ANGPTL3, where loss-of-function mutations—mutations that “break” the gene—protect people from heart attacks. The company is evaluating whether base editing, CRISPR-based editing or an entirely different approach would work best for each target, the biotech told Fierce Biotech at the J.P Morgan annual healthcare conference earlier this year.

It plans to list on the Nasdaq under the ticker "VERV."

https://www.fiercebiotech.com/biotech/gene-editing-biotech-verve-indicates-a-swift-swerve-onto-wall-street-asking-for-100m-ipo

Is pharma ready to talk cure in cancer? Maybe with better access, screening, ASCO preview panel says

 As the American Society of Clinical Oncology conference preps to open this week, AstraZeneca asked cancer experts what they think about mentioning the C-word—in this case, cure—in talking about cancer treatment. 

The panel of cancer experts, oncologists, researchers and patient advocate leaders offered many perspectives and personal examples, but not surprisingly, didn’t come to a simple consensus.

Still, the hypothetical exercise shines a light on drug development advances—and the fact that some cancer cures are moving within reach. What will happen when drugs adopt, or at least consider using, the word “cure” in talking about cancer therapies?

“In one week’s time, we’ll have the opportunity to learn about some of the latest and most important advances in the oncology space and within that context, on the eve of the latest practice-changing (advances), it’s perfect that we’re having this conversation,” said David Fredrickson, AstraZeneca executive VP of its oncology business unit.

Christian Massacesi, senior VP and head of AstraZeneca's late-stage oncology development, related the story of his own uncle, who was diagnosed with late-stage cancer, facing a life expectancy of only a few months. But thanks to advanced therapies, his uncle is not only still alive six years later, but also cancer-free.

“Can we say he is cured? Probably we cannot. The science and the current data don’t tell us that for sure,” Massacesi said, adding that as a researcher at AstraZeneca, his mission now is developing treatments, like those for his uncle, that will “provide meaningful time to patients" but also ones "ultimately that can lead to cure.”

While the not-yet-cure words in use now by drugmakers, physicians and even patients may vary—remission, cancer-free, stable disease or even chronic condition—AstraZeneca’s panelists did agree on other issues. Especially the industry changes necessary to begin using the word cure legitimately.

Screening—earlier, more comprehensive and more of it—topped the list for many. Another important change is the need for ubiquitous and fair access across the full scope of cancer care.

“Do you really cure a disease if you have people who are left behind with it? ... The thing that keeps me up at night is does everyone benefit from the clinical trials we’re doing. Will everybody have access?” said Craig Emmitt Cole, M.D., who heads clinical research in hematology/oncology and multiple myeloma at the Michigan State University Breslin Cancer Center.

Elizabeth Franklin, president of the Cancer Support Community, agreed, adding, “There is actually no such thing as cure without equity. Period. … I don’t want to see a system of haves and have-nots, and I worry that when we see this rapid innovation, it’s going to benefit a certain group in our society. I want to make sure that when we talk about cure we are absolutely talking about cure for all.”

Patient perspectives, shown in a short video of opinions displayed onscreen, varied from those who said the word cure offers “false hope” and has “no place in clinical” discussions to another patient who said yes to the word because it “gives hope.”

Surgeon scientist Jonathan Spicer, M.D., medical director of the McGill Thoracic Oncology program, said that for many of his patients, cure is at the top of their agenda, although as he acknowledges “obviously it’s more complicated than that.”

He talked about a recent lung cancer surgical patient who was in her 30s. He successfully removed her tumor and offered the additional positive news that her tumor had a genetic indication for which there are targeted treatments.

“It was so important for me to tell her that she was cured, and it’s a very difficult conversation to have,” he said. “I don’t know whether she’ll have a recurrence or something down the road … There’s a lot on the line.”

As the panel moderator, Ron Winslow, a medical and science reporter formerly with The Wall Street Journal, chimed in—and perhaps the crux of the dilemma: “There is this sort of ‘you’re cured, but.’ There’s always that ‘but’ hanging in the air."

And that is the reality of the use of the word cure in cancer—at least for now.

https://www.fiercepharma.com/marketing/pharma-ready-to-talk-cure-cancer-astrazeneca-gathers-expert-panel-ahead-asco-to-debate

Nuwellis, Premier Enable Nationwide Access to Ultrafiltration Therapy

 Nuwellis, Inc. (Nasdaq: NUWE), formerly CHF Solutions, Inc., has been awarded a three-year national contract with Premier, Inc., effective today. The new Aquapheresis category agreement allows Premier members, at their discretion, to take advantage of special pricing and terms pre-negotiated by Premier for Aquadex SmartFlow®, the gentle fluid management technology developed by Nuwellis.

Aquadex® is the first and only therapy currently available in this newly-established Aquapheresis product category. The collaboration provides Nuwellis with the opportunity to expand into many of the thousands of hospitals with which Premier holds relationships.

“Premier is a trusted healthcare leader, and working with them to expand access to ultrafiltration with Aquadex is a significant milestone for the Company and the customers we serve,” said Nestor Jaramillo, Jr., President and CEO of Nuwellis. “The immense value of this collaboration will be felt by hospitals dedicated to offering its patients with the most innovative technology. We’re ultimately grateful for Premier’s dedication to deliver transformative solutions that power real results.”

Nuwellis is dedicated to transforming the lives of patients suffering from fluid overload – a condition associated with multiple disease states that can cause a significant burden on vital organs if left untreated. The Company prioritizes collaboration and innovation to provide unsurpassed care to pediatric, critical care and heart failure patients. Aquadex is built to gently remove excess fluid with customizable rates to improve patient outcomes.

Premier, Inc. is a leading healthcare improvement company, uniting an alliance of more than 4,100 U.S. hospitals and health systems and approximately 200,000 other providers and organizations to transform healthcare. With integrated data and analytics, collaboratives, supply chain solutions, and consulting and other services, Premier enables better care and outcomes at a lower cost.

https://finance.yahoo.com/news/nationwide-access-ultrafiltration-therapy-possible-120000488.html

CytoDyn Receives First Purchase Order from Chiral Pharma for Leronlimab

 CytoDyn Inc. (OTC.QB: CYDY), (“CytoDyn” or the “Company"), a late-stage biotechnology company developing Vyrologix™ (leronlimab-Pro 140), a CCR5 antagonist with potential multiple therapeutic indications, announced today that Chiral Pharma Corporation in the Philippines placed its first purchase order for leronlimab under a Compassionate Special Permit (“CSP”) to treat critically ill COVID-19 patients in the Philippines. The Company expects to recognize revenues related to this purchase order upon fulfillment of the purchase order and the terms of the agreement.

Chris Recknor, M.D., Chief Operating Officer and Head of Clinical Development of CytoDyn, said, "Current Philippine CSP data is significant for 21% mortality rate which is below that observed in CD12.  One of the patient successes was recently reported last week on One News Philippines TV station. This patient had severe COVID-19 with deteriorating lung function despite treatment with antivirals and steroids. Testing was significant for high inflammation in the blood with Ferritin 17x elevated. Oxygen saturation on maximum oxygen flow was only 82% (normal >95%). Twenty hours after receiving 700 mg of leronlimab subcutaneously at the patient's home, the patient's heart rate decreased from 120s to low 90s. Shortly thereafter the patient regained appetite, improved lung function, and had reduced inflammation with Ferritin down to 4x elevated.  At week two, the patient successfully went off oxygen shortly after second dose also given at home.  At week three, the patient continued to improve in stamina and returned to routine physical activities. Inflammation markers were still elevated with Ferritin still two times elevated, so a third dose was given.  At week four, the patient is currently back to normal life activities."

Dr. Recknor further stated, “Although the CSP data is not randomized double-blinded control, the numerous case reports from the Philippine CSP are consistent with CD12 prespecified major endpoint metrics showing Vyrologix when combined with standard of care is better than standard of care alone (e.g. antivirals/dexamethasone) and may improve clinical outcome status four times better than standard of care at day 14 (p< 0.021) and reduce hospital stay by as much as 6 days (p<0.005) with a higher likelihood of returning to activities of daily living without limitations vs. placebo control in critical COVID patients."

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, stated, “After we provided leronlimab, free of charge, for the first 100 patients in the Philippines for critically ill COVID-19 patients, we are now on a clear path to achieving revenue with our first purchase order today under CSP from Chiral Pharma. We are so proud to be able to provide leronlimab in the capital region where the use of intensive care unit (ICU) capacity is above 70% for COVID-19 patients with hospital beds in great shortage. We are equally excited about our EUA package (CMC, non-clinical and clinical sections) being prepared for submission to the Philippine FDA.“

About Vyrologix™ (Leronlimab) The U.S. Food and Drug Administration (FDA) granted CytoDyn Fast Track designation to explore two potential indications using leronlimab to treat Human Immunodeficiency Virus (HIV) and metastatic cancer. The first indication is combination therapy with HAART for HIV-infected patients, and the second is for metastatic triple-negative breast cancer (mTNBC). Leronlimab is an investigational humanized IgG4 mAb that binds to CCR5, a cellular receptor important in HIV infection, tumor metastases, and other diseases, including nonalcoholic steatohepatitis (NASH). Leronlimab has been studied in 16 clinical trials involving more than 1,200 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab combined with HIV standard care in patients with multi-drug resistance to current available classes of HIV drugs).

https://tylerpaper.com/ap/business/cytodyn-receives-first-purchase-order-from-chiral-pharma-corporation-for-use-of-leronlimab/article_9dec947f-7ba2-557a-b5ba-5a09de9909a4.html

Abbott cuts 2021 profit forecast on lower COVID-19 testing demand

 Abbott Laboratories on Tuesday cut its full-year 2021 profit forecast due to a projected drop in COVID-19 diagnostic testing demand, sending its shares down 4.1% before the bell.

"This has been driven by several factors, including significant reductions in cases in the U.S. and other major developed countries, accelerated rollout of COVID-19 vaccines globally and, most recently, U.S. health authority guidance on testing for fully vaccinated individuals," the drugmaker said.

Abbott generated billions in sales for its COVID-19 tests last year, but analysts have cautioned that demand is likely to fall this year.

The company now expects full-year adjusted profit from continuing operations of $4.30 to $4.50 per share. It had forecast at least $5 per share in January. Analysts expect $5.04 per share, according to Refinitiv data.

Abbott sees second-quarter adjusted profit from continuing operations of at least $1 per share, compared with analysts' estimates of $1.23 per share.

https://finance.yahoo.com/news/abbott-cuts-2021-profit-forecast-125827138.html

Rezolute: ‘Strong Buy’ Stock with 100% Upside Potential

 Every stock investor wants a strong return; that’s axiomatic, it’s why people get into the stock market to begin with. But the markets are inherently risky, and finding the sweet spot – the right combination of risk and reward – seems as much an art as a science. You can use science, however, to minimize the risk.

We’re talking about statistical science, the study of numbers, their patterns, and the relationships between them. This can give investors an objective view of the broader market or specific stocks, and can even be used to measure the success of those artists of the stock market, the professional traders and analysts.

We’ve used the tools on the TipRanks platform to sort through the publicly traded stocks and find three that are showing a solid combination of risk and reward. Specifically, we’ve looked for Strong Buy stocks that have recently received a thumbs up from an analyst – along with a price target suggesting 100% or better upside potential. Doubling your money sounds like a good return, so let’s find out what else these stocks have going for them.

Rezolute (RZLT)

We’ll start in the biopharmaceutical industry, where Rezolute specializes in developing drug therapies – new medications – for patients with difficult-to-treat metabolic conditions. These are frequently considered orphan diseases, illnesses that have very few patients and therefore a limited market.

Rezolute is currently working on two pipeline projects, both for conditions similar to or related to diabetes. The company’s leading drug candidate, RZ358, in currently undergoing a Phase 2b open-label study as a treatment for congenital hyperinsulinism (CHI), a rare pediatric disorder in which the pancreas produces too much insulin, causing extremely low blood sugar, with cascading effects on the whole body. RZ402, the second drug candidate, is in Phase 1 clinical trials. It is an orally dosed treatment for diabetic macular edema, one of the causes of diabetic-related blindness.

In its recent financial report for fiscal Q3 2021, Rezolute included development updates on both leading drug candidates. For RZ358, the company noted that the Phase 2b RIZE study is still enrolling patients and that top line data is expected to become available in 2H21. For the Phase 1 study of RZ402, Resolute announced that the trial is complete and that the initial results demonstrated that once-daily oral dosing is feasible. The company will initiate a Phase 1b trial in 3Q21, as a step toward Phase 2 studies.

In financial results, Rezolute reported having on hand $32 million available in cash and equivalents, enough to fund operations into the third calendar quarter of 2022.

H.C. Wainwright’s five-star analyst Douglas Tsao initiated his coverage of RZLT with an upbeat outlook, writing, “Rezolute is ready to enter the spotlight with two assets featuring novel mechanisms… Despite assets with promising data and differentiated mechanisms, Rezolute has largely been overlooked by the investment community, which we largely attribute to its entry into the public markets via a reverse merger and an OTC listing. However, with key catalysts upcoming and a recent up listing on the NASDAQ, we think it’s time for investors to pay attention to this story.”

Tsao gives the stock a Buy rating and $21 price target that implies an upside of 103% for the coming year. (To watch Tsao’s track record, click here.)

The Strong Buy consensus rating on RZLT shares is based on 3 recent reviews – and they are all positive, making the consensus unanimous. The shares are priced at $10.33, with a $25.33 average price target, making the one-year upside potential a robust 145%.

https://finance.yahoo.com/news/3-strong-buy-stocks-100-141738180.html