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Monday, June 13, 2022

Why Forma Therapeutics' Current Price Level Is 'Attractive Entry Point'

 

  • During the European Hematology Association (EHA) conference, Forma Therapeutics Holdings Inc  presented updated results from the 12-week open-label extension (OLE) of etavopivat's Phase 1 trial in sickle cell disease.
  • Treatment with etavopivat 400mg QD elicited a >1g/dL hemoglobin (Hb) increase in 73% of the 15 patients and an average maximal Hb increase of 1.5 g/dL across the entire cohort. 
  • Considering OLE data and management's guidance on the first interim analysis of etavopivat efficacy in sickle cell disease in the Phase 2/3 trial, the analysts view current share price levels as an attractive entry point for investors. 
  • They have reiterated the Buy rating with a price target of $53. 
  • HC Wainwright notes that RBC analysis shows that treated patients' RBCs also exhibited a statistically significant improvement in deformability and markers of inflammation, hypercoagulability, and tissue hypoxia. 
  • "Given these changes, we find it unsurprising that treatment with etavopivat was associated with a 68% decrease in the annualized vasoocclusive crisis (VOC) rate relative to the patient's historical annualized VOC rate (0.30 vs. 0.93, respectively)," HC Wainwright wrote.
  • The analysts acknowledge the small sample size but believe these results demonstrate etavopivat's ability to improve Hb levels and reduce VOCs.
  • It writes that there is no FDA-approved treatment for sickle cell disease that improves Hb levels and reduces the VOC rate. 

Soleno Long Data Show Improvements in Prader-Willi

 Soleno Therapeutics, Inc. (Soleno or the Company) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today presented new positive clinical data from its ongoing late-stage DCCR development program for the treatment of Prader-Willi syndrome (PWS) at ENDO 2022, which is being held June 11-14, 2022, in Atlanta, Georgia, USA.

An oral presentation titled, “Hormonal and Cardiometabolic Changes Associated with Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome,” presented by Dr. Eric Felner, Emory University School of Medicine, summarized data demonstrating positive hormonal, cardiometabolic and body composition changes in PWS patients following one year of treatment with DCCR. The patients were treated in C601, a Phase 3 randomized, double-blind, placebo-controlled study and C602, its ongoing open-label extension.

Patients (n=82) experienced improvements in body composition, as evidenced by statistically significant changes in lean body mass (p<0.0001) and the ratio of lean body mass to fat mass (p=0.0005), based on DXA scanning. These changes were accompanied by reduced levels of leptin (p<0.0001), fasting insulin (p=0.0004) and an improvement in insulin sensitivity (HOMA IR p=0.0033), likely related to a combination of reduced fat mass and improved leptin resistance. A statistically significant increase of adiponectin (p<0.0001), a cardioprotective marker, was also observed. Similar effects were observed in obese participants (n=40) enrolled in these studies.

https://finance.yahoo.com/news/soleno-therapeutics-presents-long-term-200500320.html

FDA Approves First Systemic Treatment for Alopecia Areata to Eli Lilly's (LLY) Olumiant

 U.S. Food and Drug Administration approved Olumiant (baricitinib) oral tablets to treat adult patients with severe alopecia areata, a disorder that often appears as patchy baldness and affects more than 300,000 people in the U.S. each year. Today’s action marks the first FDA approval of a systemic treatment (i.e. treats the entire body rather than a specific location) for alopecia areata.

“Access to safe and effective treatment options is crucial for the significant number of Americans affected by severe alopecia,” said Kendall Marcus, M.D., director of the Division of Dermatology and Dentistry in the FDA’s Center for Drug Evaluation and Research. “Today’s approval will help fulfill a significant unmet need for patients with severe alopecia areata.”

Alopecia areata, commonly referred to as just alopecia, is an autoimmune disorder in which the body attacks its own hair follicles, causing hair to fall out, often in clumps. Olumiant is a Janus kinase (JAK) inhibitor which blocks the activity of one or more of a specific family of enzymes, interfering with the pathway that leads to inflammation.

The efficacy and safety of Olumiant in alopecia areata was studied in two randomized, double-blind, placebo-controlled trials (Trial AA-1 and Trial AA-2) with patients who had at least 50% scalp hair loss as measured by the Severity of Alopecia Tool for more than six months. Patients in these trials received either a placebo, 2 milligrams of Olumiant, or 4 milligrams of Olumiant every day. The primary measurement of efficacy for both trials was the proportion of patients who achieved at least 80% scalp hair coverage at week 36.

https://www.streetinsider.com/Corporate+News/FDA+Approves+First+Systemic+Treatment+for+Alopecia+Areata+to+Eli+Lillys+%28LLY%29+Olumiant/20207076.html

Mustang: Phase 1/2 shows high overall, complete response rates, CAR T persistence in follicular lymphoma

 Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that updated interim data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T cell therapy, show a favorable safety profile, high overall response (“ORR”) and complete response (“CR”) rates, and CAR T persistence in patients with follicular lymphoma (“FL”). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center (“Fred Hutch”) to treat patients with relapsed or refractory B-cell non-Hodgkin lymphomas (“B-NHLs”) and chronic lymphocytic leukemia (“CLL”).

The updated results presented during an on-site oral presentation at the European Hematology Association 2022 Hybrid Congress (“EHA2022”) by Mazyar Shadman, M.D., M.P.H., Associate Professor and physician at Fred Hutch and University of Washington, included interim safety and efficacy data from the cell manufacturing process that was modified to combine the culture of CD4+ and CD8+ cells. CAR-T cells were administered at one of 5 dose levels: 1x105, 3.3x105, 1x106, 3.3x106 and 1x107 CAR T cells/kg. Treatment for all patients was infused in the outpatient setting except for the first patient of each dose cohort, each of which was kept for overnight observation.

BioMarin resumed at Neutral by Wedbush

 Target to $70 from $146\

https://finviz.com/quote.ashx?t=BMRN

Belite Bio, Inc’s (NASDAQ:BLTE) Quiet Period Expired on June 8th

 Belite Bio’s (NASDAQ:BLTE  Get Rating) quiet period is set to expire on Wednesday, June 8th. Belite Bio had issued 6,000,000 shares in its public offering on April 29th. The total size of the offering was $36,000,000 based on an initial share price of $6.00. During Belite Bio’s quiet period, insiders and underwriters that worked on the IPO are restricted from issuing any earnings forecasts or research reports for the company because of regulations issued by the Securities and Exchange Commission. Following the end of the company’s quiet period, it’s expected that the brokerages that served as underwriters on the stock will initiate research coverage on the company.

BLTE traded down $0.17 during trading on Wednesday, hitting $14.95. 14,347 shares of the company traded hands, compared to its average volume of 607,820. Belite Bio has a one year low of $8.80 and a one year high of $17.50.

Belite Bio, Inc, a clinical-stage biopharmaceutical drug development company, engages in the research and development of novel therapeutics targeting atrophic age-related macular degeneration and autosomal recessive stargardt diseases. Its lead product candidate is LBS-008, an oral once-a-day treatment that can reduce and maintain the delivery of vitamin A to the eye to reduce the accumulation of toxic vitamin A by-products in ocular tissue that is in phase 3 clinical trial.

https://www.defenseworld.net/2022/06/01/belite-bio-incs-nasdaqblte-quiet-period-to-expire-on-june-8th.html

AMA Debates Policy on Informal Milk Sharing Amid Formula Shortage

 A draft policy calling for the American Medical Association (AMA) to "discourage" informal milk sharing faced an onslaught of opposition from nearly a dozen physicians, most of them parents, during the AMA's House of Delegates meeting on Sunday.

So many delegates opposed the policy that one of them, Ajanta Patel, MD, who introduced herself as a breastfeeding mother, suggested flipping the policy entirely to "promote informal milk sharing. Since it sounds like that's what the House actually wants to do."

The debate began when the Medical Student Society introduced a draft policy calling for the AMA to "discourage the practice of informal milk sharing," when the practice doenot meet the same health and safety standards as milk banks -- for example when it does not involve screening donors or pasteurization.

The proposed policy also called for the AMA to encourage breastfeeding mothers to donate to regulated human milk banks and urged more research into the "status of milk donation" in the U.S. and ways to increase donation rates.

Breast milk protects infants from a range of diseases and conditions, such as bacteremia, urinary tract infections, lower respiratory tract infections, necrotizing enterocolitis, and sudden infant death syndrome, noted the draft resolution.

"Donor human milk provides nutrients comparable to a mother's own milk, yielding positive effects on neurodevelopment and tolerance of feedings," the authors of the resolution wrote.

Raymond Tu, MD, a delegate from Washington, D.C., applauded the authors of the resolution for tackling a timely issue.

Given the current formula shortage, Tu said, Medicaid enrollees are extremely confused about how to feed their babies, asking, "Can I use cow's milk? ... Can I add Gatorade to my milk?"

But "informally shared milk, especially that's sold or given by a stranger does have risks due to lack of medical screening of the donor and safe storage practices," noted Zarah Iqbal, MD, MPH, who supported the resolution on behalf of the American Academy of Pediatrics (AAP). She also noted that while some breastfeeding moms donate their milk, others have begun selling their milk on Facebook and Craigslist.

Melissa Garretson, MD, another AAP delegate, also spoke in support of the resolution, and cited the Academy of Breastfeeding Medicine's position statement on the issue. "Internet-based breast milk sharing is not recommended under any circumstances," she noted.

Garretson also favored an amendment put forward by the Women Physicians Section calling on the AMA to encourage human milk sharing "along with the development of standards that promote both safe and equitable access."

Speaking on behalf of her delegation, Carolynn Francavilla Brown, MD, an alternate delegate from Colorado and a "lactating parent," argued that informal milk sharing has been painted in a bad light.

"There's nothing nefarious going on here," she said.

The milk that is being shared is "clean, well-stored," and "without medication or supplements," said Francavilla Brown. "It is milk that is good enough for their own baby. There is no reason it wouldn't be good enough for someone else's baby."

She said "we need to examine why we have an 'ick' factor around this," arguing that the real problem is a form of "cultural bias."

In response to the criticism the proposal was receiving, Anna Laucis, MD, MPhil, of the Women Physicians Section, which supports the resolution, offered an amendment calling for the AMA to encourage human milk sharing and highlight the importance of developing standards that "promote both safe and equitable access."

While much of the concern around informal milk sharing centered around strangers and internet-based sharing, Garretson also stressed the importance of medical screening and safe storage, no matter who the donor is.

A woman may think, "'my best friend's milk is fine for me,'" Garretson said, but "your best friend may not want to tell you that they've been taking antidepressants for a number of years, and they shouldn't have to tell you that."

Kavita Arora, MD, MBE, delegate for the Young Physicians Section (YPS), who opposed the resolution but supported the Women Physicians Section amendment, noted that "any amount of breast milk given safely ... is still better than formula."

She also argued that milk from many who are on antidepressants is safe.

"We can thread this needle," she said. Arora called for creating a set of questions to ask potential donors during the informal milk-sharing process, such as "What is your HIV status?" and "What is your hepatitis C status?"

"These informal guides are much better than regulating and commercializing milk sharing," Arora said.

Alicia Reyes, MD, speaking on behalf of the YPS added that "we as members of the YPS as well as our progeny ... have benefited from informal milk sharing" and therefore her section opposed the resolution as written.

Finally, Siri Holton, MD, a delegate from the American College of Obstetricians and Gynecologists, and also a lactating parent, shared the story of a medical school colleague who lost her life to postpartum depression, leaving behind a newborn.

Because the deceased mother badly wanted her child to be breastfed, around 20 mothers stepped up and formed a "milk train" providing time-stamped bags of breast milk to be frozen and thawed for the woman's infant son, as the Star Tribune noted in a story last December.

For Holton's 3-month-old baby girl, "I am fortunate to be able to provide her with my own breast milk," she told MedPage Today in a follow-up phone call. "And if something were to happen to me or if I was no longer able to provide breast milk for my infant, I would certainly seek out safe peer-sharing of breast milk from my trusted friends or colleagues."

https://www.medpagetoday.com/meetingcoverage/ama/99203