Dyne upped to Strong Buy by Raymond James

 

• Raymond James has upgraded Dyne Therapeutics Inc 
  DYN+12.8%+ Free Alerts
   from Outperform to Strong Buy and raised the price target to $27 from $16.
• The analyst notes proximity to myotonic dystrophy type 1 (DM1) data for DYNE-101 (2H23), for which it has high conviction, will be positive.
• The ACHIEVE Phase 1/2 trial evaluating DYNE-101 for DM1 is underway. It includes a 24-week multiple ascending dose (MAD), a 24-week open-label extension, and a 96-week long-term extension. 
• The ACHIEVE trial, designed to be registrational, is expected to enroll approximately 64 adult patients. Data from the MAD placebo-controlled portion of the ACHIEVE trial on safety, tolerability, and splicing are anticipated in the second half of 2023.
• Raymond James also sees a substantial valuation gap with Avidity Biosciences Inc 
  RNA+1.27%+ Free Alerts
  , which should serve as a tailwind for DYN in 2023 between now and data (especially because the analyst thinks RNA will move higher also).
• DYNE-101 will be a valuable therapeutic if initial low doses simply recapitulate the MARINA interim data profile without the SAE. 
• Raymond James literature analysis drives conservative expectations on spliceopathy.
• DM1 is, at its peak, a ~$9 billion revenue opportunity writes Raymond James analyst, which Dyne and Avidity will compete.

https://www.benzinga.com/analyst-ratings/analyst-color/23/02/31074180/analyst-says-this-small-cap-biotech-firm-has-very-valuable-therapeutic-upgrades-sto

< 10% of Eligible Type 2 Diabetes Patients Get New but Pricey Drugs

 Fewer than 10% of American adults with type 2 diabetes who qualified for treatment with newer agents — such as an SGLT2 inhibitor or GLP-1 agonist — actually received treatment with at least one drug from drug class in 2017-2020, based on a new analysis of just over a thousand adults who participated in a representative, biannual survey and self-reported a diabetes diagnosis.

The cost of these agents, and their uncertain cost-effectiveness at current prices, is likely a key driver of the low usage rate, say the authors of a brief report published February 27 in Annals of Internal Medicine.

"Clinical studies have shown that both GLP-1 [glucagon-like peptide-1] receptor agonists and SGLT2 [sodium-glucose cotransporter 2] inhibitors yield additional clinical benefits compared with older treatments in reducing body weight and progression of cardiovascular disease and chronic kidney disease," write Shichao Tang, PhD, from the US Centers for Disease Control and Prevention (CDC), Atlanta, Georgia, and colleagues.

"However, these medications come at a substantially higher cost," they stress.

Tang explained in an interview that the new study "points to prior studies about the high cost of these medications as a potential barrier to use, but more research is needed to understand cost-effectiveness and any potential barriers to use, including cost."

The work "did not include research into cost-effectiveness or why the percentage of people already using these medications was low," he emphasized.

Most Qualify, but Drug Cost-Effectiveness May Be Lacking at Current Prices

Tang and colleagues used data collected by the US National Health and Nutrition Examination Survey (NHANES) during two 2-year cycles between 2017 and 2020 that included 1417 people who self-identified a diagnosis of diabetes.

Excluding those who likely had type 1 diabetes and those with incomplete data left 1330 survey participants, including 1133 (85%) who fit criteria for the treatment of type 2 diabetes with an agent from one of the two studied classes, as recommended in 2022 by a panel representing the American Diabetes Association and the European Association for the Study of Diabetes.

Among these 1133 people — who represent more than 22 million American adults with type 2 diabetes who fit the 2022 criteria — a scant 3.7% were actually taking a GLP-1 agonist and 5.3% were taking an SGLT2 inhibitor.

"While it's important to note that our data predate the 2022 recommendations, these drugs were offered as second-line therapy for patients with certain diabetes-related complications in 2017-2020," and hence, provide potentially useful insights, noted Tang, a health economist with the CDC National Center for Chronic Disease Prevention and Health Promotion.

Based on retail prices listed on a US-based website, a 30-day supply of an oral SGLT2 inhibitor can cost about $550-$600/month, while common subcutaneously injected GLP-1 receptor agonists can run from a few hundred dollars for a daily injection or close to $1000 for a formulation administered weekly.

"Cost-effectiveness was not formally considered in the current guideline, but an assessment of cost-effectiveness may assist better targeting of interventions to achieve the greatest effect at a sustainable cost," the researchers conclude.

The study received no commercial funding. None of the authors had relevant financial relationships.

Ann Int Med. Published online February 27, 2023. Abstract

https://www.medscape.com/viewarticle/988819

Epinephrine Nasal Sprays, Sublingual Film Making Headway

 Nearly half a century since the epinephrine autoinjector hit the market in 1987, several companies are working on needle-free versions of this medicine, which is vital for treating anaphylaxis yet dangerously underused.

For the second year in a row, data from these investigational epinephrine products — two intranasal and one sublingual — drew attention at the American Academy of Allergy, Asthma and Immunology (AAAAI) 2023 Annual Meeting. The nasal sprays are further along, with ARS Pharmaceuticals' neffy under review as Bryn Pharma prepares to submit a New Drug Application for Utuly. Later this year, Aquestive Therapeutics plans to start a pivotal trial of its postage stamp–sized film (AQST-109), which delivers an epinephrine prodrug when placed under the tongue.

"Needle-free epinephrine — that is easier to carry and easier to administer than currently available forms of epinephrine — is a huge unmet need in food allergy and anaphylaxis management," said Brian Schroer, MD, a pediatric allergist at Cleveland Clinic in Ohio.

At last year's AAAAI meeting, ARS Pharmaceuticals reported that its neffy 1 mg spray reached maximal epinephrine plasma concentration in healthy adult volunteers in 30 minutes — slower than EpiPen 0.3 mg (20 minutes) and faster than manual intramuscular (IM) 0.3 mg injection (45 minutes). Since then, the company has completed similar pharmacokinetic (PK) and pharmacodynamic (PD) comparisons of the two existing epinephrine options against neffy 2.0 mg — single and double dosing (two doses given 10 minutes apart). 

As reported on a February 24 poster at this year's AAAAI meeting in San Antonio, neffy 2.0 mg had a PK profile comparable to approved epinephrine injection products in the single-dose analysis and looked even better in the double-dose comparison. PD effects on blood pressure and heart rate looked similar for all epinephrine delivery modes, as did safety profiles.

In Bryn Pharma's PK/PD analyses, Utuly 13.2 mg, administered as two consecutive 6.6-mg doses, produced higher and more sustained epinephrine plasma levels than did EpiPen 0.3 mg and manual IM 0.5 mg.

"We deliver the drug as fast as an autoinjector but maintain the level a bit longer," CEO David Dworaczyk, PhD, told Medscape Medical News. Safety and PD profiles looked comparable for all epinephrine products.

One difference between the two intranasal formulations is the delivery approach. ARS Pharmaceuticals uses an absorption-enhancing technology (Intravail) that is used in nasal spray medications approved for treating seizures (Valtoco) and migraines (Tosymra), according to CEO Richard Lowenthal. Bryn Pharma's absorption enhancer is a generally recognized as safe (GRAS) compound that has been used in oral and topical drug delivery systems but never before in a nasal spray, said Dworaczyk.

For its part, Aquestive Therapeutics compared PK/PD profiles of epinephrine delivered by its sublingually absorbed film (AQST-109 12 mg) vs EpiPen 0.3 mg or manual IM 0.3 mg. The sublingual product produced peak plasma levels faster (12 minutes) than did the EpiPen (23 minutes) and manual IM (45 minutes) but resulted in a lower overall plasma concentration than did these traditional routes. Company researchers also showed that AQST-109 effects were sustained in real-world conditions, such as peanut butter lingering on the tongue after consumption of a sandwich.

Several aspects of the AQST-109 data raised questions. One issue was PD: Compared with the injected forms of epinephrine, the sublingual product led to larger increases in both systolic and diastolic blood pressure. "That's a little bit of a concern," especially given epinephrine's known effects on the heart, Schroer said.

Another issue was PK: After a fast rise to maximum levels, serum epinephrine also dropped quickly. Because epinephrine works in part by blocking continued release of histamine and other chemicals that trigger anaphylaxis, the impact of the sublingual product may not last as long as an epinephrine autoinjector, said Schroer. "One way to overcome that would be to administer it again — which, frankly, if you're sticking something under your tongue, is probably not that hard to do."

Based on available data, the intranasal products seem to do a better job "mirroring what happens when you use the self-injectable epinephrine," Schroer said.

Dworaczyk is CEO of Bryn Pharma. Lowenthal is CEO of ARS Pharmaceuticals. Schroer reports no relevant financial relationships.
American Academy of Allergy, Asthma, & Immunology (AAAAI) 2023 Annual Meeting. Presented February 24, 2023. Poster 014

American Academy of Allergy, Asthma, & Immunology (AAAAI) 2023 Annual Meeting. Presented February 24, 2023. Poster L01

American Academy of Allergy, Asthma, & Immunology (AAAAI) 2023 Annual Meeting. Presented February 24, 2023. Poster L13

American Academy of Allergy, Asthma, & Immunology (AAAAI) 2023 Annual Meeting: Poster 007

https://www.medscape.com/viewarticle/988817

AdaptHealth cuts 2023 guidance

 Guidance Updated for Fiscal Year 2023

The Company is updating its initial financial guidance for fiscal year 2023, as follows:

• Net revenue of $3.160 billion to $3.240 billion (previously $3.210 billion to $3.290 billion);

• Adjusted EBITDA of $650 million to $710 million (previously $690 million to $750 million);

• Total capital expenditures representing 10-12% of net revenue (previously 9-11% of net revenue).

Guidance for fiscal year 2023 does not include any contributions from acquisitions that have not yet closed.

Conference Call

Management will host a conference call at 8:30 am ET tomorrow to discuss the results and business activities. Interested parties may participate in the call by dialing:

• (800) 245-3047 (Domestic) or

• (203) 518-9765 (International)

When prompted, reference Conference ID: AHCO4Q22

Webcast registration: Click Here

Following the live call, a replay will be available for six months on the Company's website,www.adapthealth.com under "Investor Relations."

https://finance.yahoo.com/news/adapthealth-corp-announces-fourth-quarter-222800572.html

'Jill Biden talks safe sex, condoms with Kenya’s young adults'

 It was a Saturday of learning for U.S. first lady Jill Biden in Kenya.

She praised young adults for learning about safe sex and dating practices, attended a meeting of women who created their own banking system and chatted with local entrepreneurs who have been helped by a program that connects tractor owners and farmers.

All three programs aim to help women and young people take control of their lives so they can support themselves and their families. Biden has been highlighting U.S.-backed efforts to empower these groups during a five-day, two-country visit to Africa this week.

“These are issues that really all people need to talk about and yet, somehow, they don’t, and the consequences of not talking about it are so dire,” Biden told dozens of young people after talking with them about safe sex, condom use and birth control at the Shujaaz Konnect Festival, a local youth empowerment event. “So I love seeing the young people here.”

At a tent where young people were having networking-like conversations, they showed her a questionnaire they use to spur discussion. The first question: “What would you say if I told you I had a condom in my pocket right now?”

Biden laughed. “And this is the first time they’re meeting?” she asked.

A Shujaaz representative said such blunt propositions help teenagers and young adults overcome shyness, saying that it’s sometimes easier to ask strangers these types of questions.

“I’m surprised you don’t start with like, ‘What’s your biggest achievement?’ rather than, ‘I have a condom in my pocket,’” the first lady said.

The festival is a collaboration with MTV Staying Alive Foundation, which works with the U.S. Agency for International Development and the President’s Emergency Plan for AIDS Relief to help teach young Kenyans how to avoid becoming infected with HIV, which causes AIDS.

Biden, who is on the fourth day of her five-day trip to Namibia and Kenya, has spent the week promoting HIV/AIDS education programs and initiatives that teach woman and young people skills they need to find jobs or start businesses.

Her visit is part of a commitment by President Joe Biden to deepen U.S. engagement with the nations of Africa, many of which feel overlooked by the United States. Part of that effort is also about countering China’s influence on the continent that Beijing has achieved through increased trade and spending on roads and other public works projects.

Biden was scheduled to cap her visit by traveling on Sunday to an area near Kenya’s border with Tanzania to raise awareness about a severe drought that is endangering lives and livelihoods.

Earlier Saturday, the first lady went to a government community center in Kibera, an informal settlement in Nairobi, to attend a meeting of women small-business owners who participate in the Joyful Women program. Founded in 2009 by Rachel Ruto, Kenya’s first lady, the program promotes women’s economic empowerment and financial inclusion.

Participants create “table banking” groups, pooling their resources so they can lend each other money they cannot get from traditional banks. Some of the women have used the loans to start businesses. One woman said she opened a day care center.

“It’s pretty ingenious that women found a way to support other women, to lift them up and to increase economic prosperity for families, right?” said Biden, who visited a different empowerment program on a 2010 stop at Kibera.

“I’ve always taught my own daughter and my granddaughter the importance of being financially independent and, so now, here, you’ve found a way to do your own banking system, which is pretty incredible,” Biden said. Her granddaughter, Naomi, 29, sat nearby.

Before taking her seat at the table, Biden was wrapped from the waist down in an apron-like cloth known as a leso or kanga that women wear in the home.

At a separate event, Biden chatted with local entrepreneurs, small farmers and others who have been helped by Hello Tractor, which connects tractor owners and farmers who need the machinery.

The first lady also laid a wreath at August 7th Memorial Park to honor those who were killed the 1998 bombings of the U.S. embassies in Kenya and in Dar es Salaam, Tanzania. More than 200 people were killed, including 12 Americans. More than 4,500 people were wounded.

https://apnews.com/article/biden-united-states-government-kenya-jill-africa-a2d1df44f86a36950c6844afdcdb7447

Feds seek to limit telehealth prescriptions for some drugs

 The Biden administration moved Friday to require patients see a doctor in person before getting attention deficit disorder medication or addictive painkillers, toughening access to the drugs against the backdrop of a deepening opioid crisis.

The proposal could overhaul the way millions of Americans get some prescriptions after three years of relying on telehealth for doctor’s appointments by computer or phone during the pandemic.

The Drug Enforcement Administration said late Friday it plans to reinstate once longstanding federal requirements for powerful drugs that were waived once COVID-19 hit, enabling doctors to write millions of prescriptions for drugs such as OxyContin or Adderall without ever meeting patients in person.

Patients will need to see a doctor in person at least once to get an initial prescription for drugs that the federal government says have the the most potential to be abused — Vicodin, OxyContin, Adderall and Ritalin, for example. Refills could be prescribed over telehealth appointments.

The agency will also clamp down on how doctors can prescribe other, less addictive drugs to patients they’ve never physically met. Substances like codeine, taken to alleviate pain or coughing, Xanax, used to treat anxiety, Ambien, a sleep aid, and buprenorphine, a narcotic used to treat opioid addiction, can be prescribed over telehealth for an initial 30-day dose. Patients would need to see a doctor at least once in person to get a refill.

Patients will still be able to get common prescriptions like antibiotics, skin creams, birth control and insulin prescribed through telehealth visits.

The new rule seeks to keep expanded access to telehealth that’s important for patients like those in rural areas while also balancing safety, an approach DEA Administrator Anne Milgram referred to as “expansion of telemedicine with guardrails.”

The ease with each Americans have accessed certain medications during the pandemic has helped many get needed treatment, but concerns have also mounted that some companies may take advantage of the lax rules and be overprescribing medications to people who don’t need them, said David Herzberg, a historian of drugs at the University of Buffalo.

“Both sides of this tension have really good points,” said Herzberg. “You don’t want barriers in the way of getting people prescriptions they need. But anytime you remove those barriers it’s also an opportunity for profit seekers to exploit the lax rules and sell the medicines to people who may not need them.”

U.S. overdose deaths hit a record in 2021, about three-quarters of those from opioids during a crisis that was first spun into the making by drug makers, pharmacies and doctors that pushed the drugs to patients decades ago. But the grim toll from synthetic opioids like fentanyl far outstripped deaths related to prescription drugs that year, according to Centers for Disease Control Data. Fentanyl is increasingly appearing on the illicit market, pressed into fake prescription pills or mixed into other drugs.

The proposed rules deliver a major blow to a booming telehealth industry, with tech startups launching in recent years to treat and prescribe medications for mental health or attention deficit disorders. The industry has largely benefitted from the reprieve on in-person visits for drugs brought on by the pandemic, although some national retailers stopped filling drug orders generated by some telehealth apps over the last year.

The DEA has grown increasingly concerned over the last two years that some of those startup telehealth companies are improperly prescribing addictive substances like opioids or attention deficit disorder medication, putting patients in danger, a DEA official told The Associated Press on Friday.

The official said the agency plans to have the new rule in place before the COVID-19 public health emergency expires on May 11, which will effectively end the loosened rules. That could mean people who may seeking treatment from a doctor who is hundreds of miles away need to start developing plans for in-person visits with their doctors now, pointed out Boston-based attorney Jeremy Sherer, who represents telehealth companies. Patients will have six months to visit their doctor in person when the regulation is enacted.

“Providers and their patients need to know what that treatment is going to look like moving forward and whether, once the public health emergency ends in May, if they’re going to need to figure out a way to have a visit in person before continuing treatment, and that can be a real challenge,” he said.

Many states have already moved to restore limitations for telehealth care across state lines. By October, nearly 40 states and Washington, D.C., had ended emergency declarations that made it easier for doctors to see patients in other states.

https://apnews.com/article/prescription-drugs-medication-opioids-health-1f23131435341fd192f41f9db027255f