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Wednesday, June 29, 2022

Trevi: Positive Results from the Ph2b/3 Trial

 Haduvio (oral nalbuphine ER) demonstrated statistically significant results on the primary efficacy endpoint as measured by a 4-point reduction in the Worst Itch – Numerical Rating Scale (WI-NRS) (p=0.0157)

The trial also met key secondary endpoints, with a safety profile consistent with prior studies

Topline data will be presented by management and study investigator, Jennifer L Parish, MD, on a conference call and webcast on June 29, 2022, at 8:30 am EDT

To participate in the live conference call by phone, please dial (888) 317 6003 (domestic) or (412) 317 6061 (international) and provide access code 8601030. A live audio webcast and presentation will be accessible from the 'Investors & News' section on the Company's website at www.trevitherapeutics.com. An archived replay of the webcast and the presentation will also be available for 60 days on the Company's website following the event.

https://finance.yahoo.com/news/trevi-therapeutics-reports-positive-results-113000703.html

GBT Initiates Phase 2/3 Trial in Sickle Cell Disease

 Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that it initiated the Phase 2 portion of a planned Phase 2/3 trial of GBT021601 (GBT601), the company’s investigational next-generation sickle hemoglobin (HbS) polymerization inhibitor. The study (NCT05431088) is a randomized, multicenter Phase 2/3 clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of GBT601.

“Based on compelling preclinical and clinical data, we believe GBT601 has the potential to be a best-in-class therapy for patients with sickle cell disease,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development of GBT. “Specifically, it has the potential to improve on the clinical results achieved with Oxbryta® at a lower daily dose. The initiation of our Phase 2/3 trial is an important milestone in our efforts to bring GBT601 to patients. We anticipate that initial data from this trial as well as data from the continuation of our GBT601 Phase 1 study will be available before the end of the year.”

The study is planned to include sites in Africa, Europe, the Middle East, South America, and the United States, including several sites that are expected to begin enrolling patients in the near term. The Phase 2 portion of the study will evaluate the safety, tolerability, and efficacy of GBT601 and enroll up to 60 patients with SCD who are 18 to 65 years of age. Patients with hemoglobin (Hb) levels between 5.5 g/dL and 10.5 g/dL and 10 or fewer vaso-occlusive crises in the prior year are eligible for enrollment. Patients will be randomized 1:1:1 to a daily maintenance dose of GBT601 of 100, 150 or up to 200 mg. The primary outcome measure is the number of participants with a change from baseline in Hb through Week 12. Secondary outcomes measures include assessments of PK and PD, as well as an assessment of the relationship between GBT601 and measures of anemia and hemolysis.


Palisade Bio Initiates Phase 3 Study

Completion of enrollment for Phase 3 study targeted within 18-24 months

In multiple clinical studies, LB1148 has demonstrated statistically significant acceleration in the time to the return of normal GI function, and has the potential to prevent damage to GI tissues and shorten the duration of costly post-surgery hospital stays

Phase 3 study design utilizes same dosing protocol and same primary endpoint as positive Phase 2 study that demonstrated 1.1-day improvement in return of bowel function

https://finance.yahoo.com/news/palisade-bio-initiates-phase-3-123000294.html

Lilly will supply additional 150,000 doses of Covid treatment to U.S.

 Eli Lilly and Company (NYSE: LLY) today announced a modified purchase agreement with the U.S. government to supply an additional 150,000 doses of bebtelovimab for approximately $275 million. The existing U.S. government supply of bebtelovimab, including the new purchase, is expected to meet present demand through late August 2022. Bebtelovimab continues to maintain neutralization activity against the most common, and fastest growing, Omicron variants (BA.2.12.1 and BA.4/BA.5) in the United States, in addition to all known variants of interest and concern.

"Lilly and its collaborators have partnered closely with the federal government throughout the pandemic to ensure broad and equitable access to our monoclonal antibodies," said David A. Ricks, Lilly's chair and CEO. "While Congress works toward additional COVID-19 funding, Lilly and the U.S. government will continue to work together to support the availability of bebtelovimab to maximize equity and accessibility in the U.S. market."

Delivery of doses will begin immediately and complete no later than August 5, 2022. The 2022 estimated financial impact of this agreement is approximately $275 million of revenue and approximately $0.08 of EPS. An option for an additional 350,000 doses to be exercised no later than September 14, 2022 will remain in the agreement.

This purchase has been supported in whole or in part with federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract number W58P0522C0012.

https://finance.yahoo.com/news/lilly-supply-additional-150-000-104500602.html

Facebook: ‘incorrect enforcement’ removed posts on abortion pill access

 Posts about access to abortion pills through mail were blocked on Facebook due to an “incorrect enforcement” of the platform’s policies, a Meta spokesperson said. 

Spokesperson Andy Stone tweeted that the Meta, Facebook’s parent company “discovered some instances” in which its policy banning attempts to buy, sell, trade, gift, request or donate pharmaceuticals was incorrectly enforced and added that the company is correcting them.

Stone’s tweet was in response to a Motherboard report published Monday about the platform removing posts from users who mentioned that abortion pills can be mailed.

A reporter for the outlet tested out the block by attempting to post the phrase “abortion pills can be mailed” on Facebook with a burner account. The post was flagged within seconds for violating the platform’s community standards. 

The Associated Press reported that a test post from a reporter offering to mail abortion pills was also removed, but when “abortion pills” was swapped out for “a gun” or “weed” it remained on the platform. The AP reported that posts about abortion pills were also removed on Instagram, which is also owned by Meta.

Although Facebook’s policies restrict content that attempts to buy, sell or trade pharmaceutical drugs, it does not limit users from posting about abortion pills or their accessibility. 

That same policy is meant to apply to marijuana and guns, as well.

The Federal Drug Administration in December expanded access to abortion pills by allowing patients to obtain them by mail. 

Access to abortion pills by mail will likely be even more crucial for some people seeking abortions after the Supreme Court struck down Roe v. Wade, which established federal protections for abortion, last week.

Republican-led states have already been making moves to limit or ban access to the pills.

https://thehill.com/policy/technology/3540043-facebook-says-incorrect-enforcement-removed-posts-on-abortion-pill-access/

White House details monkeypox vaccination strategy

 The White House on Tuesday said it plans to send out tens of thousands of vaccine doses immediately as the nation tries to head off a growing monkeypox outbreak, amid fears that the country is undercounting cases because of insufficient testing.  

Administration officials ​said the vaccine strategy will focus on protecting those most at-risk of monkeypox, and will use a tiered distribution system to prioritize vaccines for areas with the highest numbers of cases.

The Centers for Disease Control and Prevention (CDC) has confirmed about 300 cases of monkeypox in the U.S., though the actual number could be higher.  

The administration plans to allocate 296,000 doses of the Jynneos vaccine over the coming weeks, which is the only Food and Drug Administration-approved vaccine specifically for monkeypox.

Of those, 56,000 doses that are in the Strategic National Stockpile will be allocated immediately. Over the coming months a combined 1.6 million additional doses will become available, the White House said.

The CDC is recommending that the new vaccine doses be provided to individuals with confirmed monkeypox exposures and presumed exposures, rather than only those who have a confirmed case.

This includes those who had close physical contact with someone diagnosed with monkeypox, those who know their sexual partner was diagnosed with monkeypox, and men who have sex with men who have recently had multiple sex partners in a venue where there was confirmed monkeypox, or in an area where monkeypox is spreading.

Health officials said close contact, and sexual contact in particular, appear to be the primary drivers for this outbreak. 

“As case counts and the number of jurisdictions with cases increases, the need for medical countermeasures, including vaccines, continues to rise,” CDC Director Rochelle Walensky told reporters during a media briefing Tuesday.

“The administration’s current strategy is focused on containing the outbreak by providing vaccines to those most in need to prevent further spread of monkeypox in the communities most impacted, and as additional supply becomes available, we will further expand our efforts making vaccines available to a wider population,” Walensky said. 

The virus has been disproportionately spread among gay and bisexual men, though health authorities and LGBTQ advocacy groups have repeatedly stressed that monkeypox does not differentiate between those who are gay or straight and that everyone should stay aware and informed on the risks of the virus.

When monkeypox cases began cropping up around the U.S., federal health authorities said there were about 72,000 doses of Jynneos in the Strategic National Stockpile, a paltry amount compared to the more than 100 million doses of ACAM2000, an older smallpox vaccine.

ocal and state governments have so far expressed a preference for the newer Jynneos smallpox vaccine due to concerns over the possible side effects associated with ACAM2000. 

According to the White House, the Jynneos vaccine will be allocated using a four-tier distribution strategy that prioritizes jurisdictions with the highest case rates of monkeypox.Within each tier, doses of Jynneos will be allocated based on the number of individuals at risk for monkeypox who also have a contraindication to ACAM2000, such as those with HIV.

https://thehill.com/policy/healthcare/3540508-white-house-details-monkeypox-vaccination-strategy/

Takeda, Arrowhead Publish Results from Phase 2 Study of Fazirsiran

 Takeda (NYSE: TAK) (“Takeda”) and Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that results from a Phase 2 clinical study (AROAAT-2002) of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD) were recently published in the New England Journal of Medicine (NEJM) and presented in an oral presentation at The International Liver Congress™ 2022 - The Annual Meeting of the European Association for the Study of the Liver (EASL). The NEJM article was published online ahead of print and is titled, “Fazirsiran for Liver Disease Associated with Alpha-1 Antitrypsin Deficiency.” The EASL presentation was titled, “Reduction of Intra-hepatic Z-AAT Synthesis by Fazirsiran Decreases Globule Burden and Improves Histological Measures of Liver Disease in Adults with Alpha-1 Antitrypsin Deficiency.”

https://www.streetinsider.com/Corporate+News/Takeda+Pharmaceutical+%28TAK%29+and+Arrowhead+%28ARWR%29+Announce+Publication+of+Results+from+Phase+2+Study+of+Fazirsiran/20256655.html