The ongoing Phase III clinical trial is a randomized, observer-blind, active control study assessing the safety, reactogenicity, and immunogenicity of mRNA-1083 in two separate age groups, each consisting of about 4,000 adults. One group includes adults aged 65 and older, comparing mRNA-1083 with co-administered Fluzone HD®, a high-dose influenza vaccine, and Spikevax®, Moderna’s approved COVID-19 vaccine. The other group, comprised of adults aged 50 to 64, compares mRNA-1083 with co-administered Fluarix®, a standard-dose influenza vaccine, and Spikevax.
mRNA-1083 demonstrated a tolerable and safe profile. Most reported adverse reactions were mild to moderate (grade 1 or 2) and similar to those seen with approved vaccines used in the trial. The most frequent adverse reactions included injection site pain, fatigue, muscle aches, and headache.
Moderna intends to present the Phase III clinical trial data for mRNA-1083 at an upcoming medical conference and submit it for publication. The company will also discuss the next steps with regulatory authorities.
Almirall’s Klisyri Receives FDA Approval for Treating Actinic Keratosis on Expanded Facial or Scalp Areas, Now up to 100 cm²
Almirall revealed that the FDA has approved its recent supplemental New Drug Application (sNDA) to broaden the usage area for Klisyri to up to 100 cm². Klisyri, an ointment that acts as a microtubule inhibitor, is now approved in a 350 mg package size and serves as a 5-day topical field treatment for actinic keratosis on the face or scalp.
“The FDA’s authorization for Klisyri’s application in treating actinic keratosis across a broader area of the face or scalp marks a notable advancement benefiting both patients and dermatologists. As patients grapple with actinic keratosis spreading over larger regions, dermatologists seek methods to address the entire affected area to impede lesion progression,” states Karl Ziegelbauer, Almirall’s Chief Scientific Officer.
This latest approval expands the previous dosing guidelines for Klisyri (tirbanibulin) in treating surface areas, increasing it from a maximum of 25 cm2 to a maximum of 100 cm2. This adjustment enables healthcare professionals to address a larger region of the face or balding scalp. The supplemental New Drug Application (sNDA) received support from an additional Phase III clinical safety study involving over 100 patients across multiple centers in the US. The primary focus of this study was to assess the safety and tolerability of applying tirbanibulin to an area of about 100 cm2 on the face or balding scalp of adult patients with actinic keratosis. Results from this study were consistent with the original pivotal trials conducted on a 25 cm2 area, demonstrating similar outcomes regarding both local skin reactions and treatment-related adverse events (AEs).
Researchers also investigated the efficacy of tirbanibulin in treating a broader area, revealing a similar percentage decrease in AK lesion count as observed in the initial crucial studies.
“With the latest FDA authorization, medical practitioners can now extend treatment coverage to four times the previous surface area, enhancing their ability to address actinic keratoses. This expands treatment options, ensuring effective outcomes while maintaining a favorable safety and tolerance record for a broader range of patients,” stated Neal Bhatia, MD, based in San Diego, CA, who led the pivotal study focused on larger treatment areas.”
Klisyri will come in two different sizes: a 250 mg package (NDC 16110-391-05) designed for treating areas of up to 25 cm2, and a 350 mg package (NDC 16110-391-55) intended for treating areas of up to 100 cm2.
Lilly’s Tirzepatide Outperforms Placebo in Resolving MASH, with Over 50% of Patients Showing Fibrosis Improvement within 52 Weeks
Eli Lilly and Company released detailed findings from SYNERGY-NASH, a phase II trial involving 190 patients, with or without type 2 diabetes. The study aimed to assess the potential use of tirzepatide in adults diagnosed with metabolic dysfunction-associated steatohepatitis (MASH) along with stage 2 or 3 fibrosis, confirmed by biopsy. The efficacy results indicated that 51.8%, 62.8%, and 73.3% of participants administered with 5 mg, 10 mg, and 15 mg of tirzepatide respectively, achieved the absence of MASH without worsening fibrosis on liver histology after 52 weeks of treatment. This was notably higher compared to the 13.2% of participants on placebo, meeting the primary goal of the study. These findings were presented at the European Association for the Study of the Liver (EASL) Congress 2024 and published simultaneously in The New England Journal of Medicine (NEJM).
In a secondary measure, the effectiveness assessment displayed that 59.1%, 53.3%, and 54.2% of individuals receiving 5 mg, 10 mg, and 15 mg doses respectively, achieved a 1-stage or greater reduction in fibrosis without worsening of MASH, in comparison to 32.8% of those on placebo. Analysis of other secondary measures revealed that tirzepatide led to enhancements in body weight, blood indicators of liver damage, and markers of liver fat, inflammation, and fibrosis. Although the phase II trial wasn’t specifically aimed at demonstrating tirzepatide’s impact on fibrosis improvement, the findings indicated a potential for a clinically significant treatment effect across all dosage levels.
Dr. Rohit Loomba, Chief of Gastroenterology and Hepatology at the University of California San Diego School of Medicine, emphasized the pressing demand for innovative treatments as he noted that MASH ranks as the second leading cause of liver transplants in the United States. He underscored the significance of the study, emphasizing the critical need for therapies that can effectively slow down disease progression and lessen the risk of severe health complications.
The safety record of tirzepatide in the SYNERGY-NASH study mirrored what was seen in earlier SURMOUNT and SURPASS trials. In SYNERGY-NASH, the most frequently noted side effects were related to the gastrointestinal system, such as nausea, diarrhea, reduced appetite, constipation, and weight loss. These effects were mostly mild to moderate. Lilly is currently discussing with regulatory bodies the future actions regarding tirzepatide for addressing MASH.
Cycle Pharmaceuticals Proposes All-Cash Acquisition of Vanda Pharmaceuticals at $8.00 Per Share
Cycle Pharmaceuticals Ltd, a swiftly expanding pharmaceutical firm dedicated to developing treatments for rare diseases, has officially disclosed its proposal to acquire all of Vanda Pharmaceuticals, Inc.’s shares from the Board of Directors. The offer, made on May 24, 2024, suggests purchasing each Vanda share at $8.00 in cash, totaling $466 million in cash consideration.
Cycle’s proposition entails:
- Immediate and guaranteed monetary benefit for every Vanda shareholder.
- A substantial increase of 98% over Vanda’s closing share price on April 16, 2024, the day preceding the public announcement of Future Pak LLC’s initial bid of $4.05 per share.
- A significant rise of 63% over Vanda’s closing share price on May 23, 2024, the day before Cycle presented its proposal to the Vanda Board.
- An all-cash initial offer surpassing the cash component of Future Pak’s revised bid for Vanda on May 7, 2024.
- A notable escalation of 58% over Vanda’s closing share price on June 5, 2024.
Cycle possesses a significant amount of available cash, and both of its financial advisors express strong assurance that, with minimal due diligence, they will secure the necessary funding for the transaction. Before finalizing a merger agreement, Cycle will secure fully underwritten, obligatory commitment letters for all debt financing, with no clauses dependent on securing financing.
Ipsen’s Iqirvo Receives FDA Accelerated Approval for Treatment of Primary Biliary Cholangitis
The FDA has granted accelerated approval for Genfit’s Iqirvo (elafibranor) 80 mg tablets, in collaboration with Ipsen. This marks a pioneering treatment for PBC, to be used alongside ursodeoxycholic acid (UDCA) in adults who haven’t responded sufficiently to UDCA, or as a standalone therapy for those who can’t tolerate UDCA.
Elafibranor, under the trademark Iqirvo by Ipsen, will be introduced to the market and made available for prescription in the U.S. for eligible patients right away. Its approval is granted through accelerated approval, primarily based on its ability to reduce alkaline phosphatase (ALP) levels. However, there is no evidence yet demonstrating improvement in survival or the prevention of liver decompensation events. Further confirmation of its clinical benefits through additional trials may be necessary for continued approval of this indication. It’s advised that Iqirvo not be used for individuals with or at risk of decompensated cirrhosis (e.g., ascites, variceal bleeding, hepatic encephalopathy).
Pascal Prigent, the CEO of GENFIT, expressed his satisfaction, stating, “We are immensely proud of this approval, which reflects the hard work of all GENFIT employees. From the initial stages of drug discovery to the completion of Phase III trials, we have diligently shepherded elafibranor (Iqirvo) to this point. Through our collaboration with Ipsen, this medication will soon be accessible to healthcare providers in the US, offering patients a valuable treatment option. This upcoming launch marks a significant milestone and marks the beginning of a new phase. We anticipate that the revenues generated from the commercialization of elafibranor will fund the development of an exciting new range of programs focused on ACLF.
In 2024, as anticipated earlier, GENFIT foresees receiving around €89 million in total milestone payments from Ipsen. This includes €48.7 million upon the initial commercialization of Iqirvo in the U.S. Additionally, a €13.3 million milestone payment, invoiced in December 2023, was received in February 2024.
These incoming revenues open up new financial avenues for GENFIT and will support our strategic shift toward addressing Acute on-chronic Liver Failure and other liver-related illnesses.
