Following success in a phase III trial, the biotech company revealed last month that it has also submitted Bylvay for approval in the EU and US to treat Alagille syndrome (ALGS), a different rare disorder that causes obstructed bile flow from the liver to the small intestine and is also characterized by severe pruritus. Bylvay is also in late-stage development for biliary atresia (BA), a rare pediatric liver condition; enrollment in the phase III BOLD study just finished; data are anticipated by the end of 2024.
If the FDA approves this drug for this indication before the end of 2027, that will trigger the additional USD 10 CVR payment. If Bylvay is approved for all three of its primary indications, according to Albireo, which was previously based in Sweden and split off from AstraZeneca in 2008, it could become a USD 1 billion-plus product. Ipsen will also acquire the rights to A3907, a new oral systemic sodium-dependent bile acid transporter (ASBT) inhibitor being developed for adult cholestatic liver diseases such as primary sclerosing cholangitis if the merger goes through.
Italy’s Chiesi Farmaceutici to Buy Amryt Pharma in a USD 1.48 Billion Deal
Chiesi Farmaceutici S.p.A., a biopharmaceutical and healthcare company, will purchase Amryt Pharma Plc., which was founded in 2015, in an all-cash deal for USD 14.50 per American Depositary Share (ADS). 5 Amryt common shares are represented by a single ADS. The total transaction is valued up to USD 1.48 billion, with the upfront consideration of USD 1.25 billion representing a 107% premium to Amryt ADSs’ closing price of USD 7.00 on January 6. The transaction is supported by voting agreements from leading Amryt shareholders and directors and has received the unanimous approval and recommendation of the boards of both companies.
The deal also includes contingent value rights (CVRs) that can be worth up to an additional USD 2.50 per ADS and represent an approximate additional USD 225 million in potential consideration based on achieving certain milestones with Amryt’s Filsuvez, an Italian company’s plan to diversify its portfolio of medicines for rare diseases. Epidermolysis bullosa (EB), a hereditary skin condition that renders the skin extremely brittle and results in severe blistering and scarring, is treated with filsuvez in adults and children who are at least 6 months old.
According to Marco Vecchia, chief executive officer of the Chiesi Group, “This acquisition of the Amryt portfolio, as well as their experience, will help us in our path to provide medications to patients, no matter how rare their illness may be.” “Amryt has steadily expanded the markets for novel products, and by adding them to the Chiesi portfolio, we intend to make them accessible to even more patients who may need them,” he further stated.
Takeda Reports Positive Phase III Results of TAK-755 for Congenital Thrombotic Thrombocytopenic purpura (cTTP)
Takeda’s investigational treatment TAK-755 for the extremely rare condition congenital thrombotic thrombocytopenic purpura (cTTP) has produced encouraging findings from a phase III trial, paving the way for regulatory submissions. A lack of the ADAMTS13 protease enzyme results in cTTP, a subtype of thrombotic thrombocytopenic purpura (TTP), a rare and potentially fatal condition in which blood clots form in the body’s small blood capillaries. Less than 1,000 people in the US are thought to have the illness.
The missing enzyme was administered as an intravenous infusion in the pivotal study, and TAK-755 is a recombinant form of the ADAMTS13 protein created to fill the gap. According to Takeda, the medication is the only recombinant form of the ADAMTS13 protein under research and might be a game-changing new therapy for patients with cTTP who do not yet have any medications specifically licensed to treat their illness.
Currently, the only effective treatment is the infusion of fresh plasma to treat the symptoms of cTTP, such as hemolytic anemia, low platelet counts (thrombocytopenia), and neurological impairment, as well as to replenish ADAMTS13 levels in the body indirectly. TAK-755 was substantially more tolerated than plasma-based therapy in the phase III trial, where it reduced the incidence of thrombocytopenia events by 60%. Less than 9% of patients taking Takeda’s medication reported adverse effects, as opposed to nearly half (48%) of those receiving standard therapy.
ACELYRIN Acquires ValenzaBio, Strengthens Immunology Position
On Jan. 5, 2023, ACELYRIN, INC., announced the acquisition of ValenzaBio, a privately held company dedicated to the development of therapies for autoimmune and inflammatory diseases, in an all-stock transaction. ACELYRIN, INC. is a Los Angeles area-based late-stage clinical biopharmaceutical company dedicated to providing patients with life-changing and transformative treatment options by identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. The acquisition of ValenzaBio is anticipated to add multiple clinical and pre-clinical development programs to ACELYRIN’s robust immunology pipeline, which is led by izokibep, a small therapeutic protein inhibitor of interleukin-17A (IL-17A) designed to overcome the limitations of monoclonal antibodies.
Shao-Lee Lin, MD, Ph.D., founder and CEO of ACELYRIN has said, “the previously shared Phase 2 psoriatic arthritis data support our hypothesis that the characteristics of izokibep can lead to differentiated patient outcomes relative to current treatment options, and the results reported separately today for hidradenitis suppurativa now offer data in a second indication that further support that hypothesis. The acquisition of ValenzaBio adds additional programs to our portfolio, such as lonigutamab, which we believe similarly have the potential to offer clinically meaningful, differentiated benefits relative to current treatment options.”
As per the update from ACELYRIN, in conjunction with the acquisition of ValenzaBio, Dr. Doux will join the ACELYRIN Board of Directors. Dr. Doux is an analyst at Palo Alto Investors and a board-certified dermatologist. The acquisition of ValenzaBio is anticipated to boost the ACELYRIN portfolio with new assets, which include Lonigutamab (VB-421). Lonigutamab (VB-421) is a subcutaneously delivered anti-IGF-1R currently being evaluated in a Phase 1 study for Thyroid Eye Disease, which is expected to have an estimated market potential of $4 billion. Similarly, VB-517 is another product added to ACELYRIN’s portfolio. VB-517 is a pre-IND anti-c-KIT being studied as a treatment option in Chronic Urticaria, with more than $2.5 billion estimated market opportunity.
FDA Approves LEQEMBI Under the Accelerated Approval Pathway for Alzheimer’s Disease
On Jan. 6, 2023, Eisai Co. and Biogen Inc. announced that the US FDA, under the Accelerated Approval Pathway, had approved lecanemab-irmb (Brand Name in the U.S.: LEQEMBI™) 100 mg/mL injection for intravenous use, a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (“protofibril”)* and insoluble forms of amyloid beta (Aβ) for the treatment of Alzheimer’s disease (AD).
The FDA approval is based on Phase 2 data that showed that LEQEMBI reduced the accumulation of Aβ plaque in the brain, a defining feature of Alzheimer’s disease. Using the recently published data from the large global confirmatory Phase 3 clinical trial, Clarity AD, Eisai will work quickly to file a Supplemental Biologics License Application (sBLA) to the FDA for approval under the traditional pathway. LEQEMBI is the result of a strategic research alliance between Eisai and BioArctic. Eisai has initiated the submission of data for BLA to the National Medical Products Administration (NMPA) of China in December 2022. Eisai is further planning to file for marketing authorization applications of lecanemab in Japan and Europe by the end of Eisai’s FY2022.
Haruo Naito, Chief Executive Officer at Eisai Co., Ltd., has said, “the FDA’s approval of LEQEMBI under the Accelerated Approval pathway is an important milestone in Eisai’s four decades of research in Alzheimer’s disease and reflects our continued commitment to alleviating the burden of Alzheimer’s disease for patients and their families. Eisai has made great efforts to understand the reality of the challenges and concerns facing patients and their families who are living in the various stages of Alzheimer’s disease, and we are incredibly pleased to offer LEQEMBI as a new treatment option to help with the tremendous unmet needs of this community,”.
As per DelveInsight, about 5.4 million Americans age 54 and older are living with Alzheimer’s disease, and nearly seventy-three percent of the affected people are age 74 or older. The number of cases is anticipated to rise in the coming years. Alzheimer’s disease can lead to several medical implications for patients and their families. With the growing number of new and existing cases of Alzheimer’s, the demand for better and more effective treatment is expected to rise significantly in the coming years. The approval of LEQEMBI provides new hope for better outcomes for patients with Alzheimer’s disease.
FDA Grants Lantern Pharma Orphan Drug Designation for Drug Candidate LP-284 in Mantle Cell Lymphoma
In a major regulatory update, on January 05, 2023, Lantern Pharma announced that the US Food and Drug Administration (FDA) had granted LP-284 Orphan Drug Designation (ODD) for the treatment of mantle cell lymphoma (MCL). Lantern Pharma is a clinical-stage biopharmaceutical company using its proprietary RADR® artificial intelligence (“A.I.”) and machine learning (“M.L.”) Platform to transform the cost, pace, and timeline of oncology drug discovery and development.
Lantern Pharma’s LP-284 is a novel small molecule agent that preferentially damages DNA in cancer cells harboring mutations in DNA damage repair pathways. Lantern is developing LP-284 for several aggressive B-cell NHLs, including MCL and double-hit lymphoma (DHL), where LP-284 has shown potent antitumor activity in preclinical models. Lantern Pharma has been able to advance LP-284 from initial RADR® A.I. insights regarding anti-cancer activity and potential mechanisms of action in hematological cancers to the selection of specific subtypes of lymphomas with a superior response, to late-stage IND enabling studies and initial design of first-in-human clinical trials in less than two years.
On achieving the key landmark, Panna Sharma, President & CEO of Lantern Pharma, has stated that “receiving Orphan Drug Designation is an important milestone for our latest drug candidate, LP-284, and further validates our data-driven approach to oncology drug discovery and development. At ASH, we recently reported positive preclinical data demonstrating LP-284’s potent antitumor activity in new MCL tumors and also against tumors that had grown resistant to the MCL standard-of-care agents Ibrutinib and Bortezomib. These findings are critically pertinent due to the high relapse rate and poor prognosis of the majority of MCL patients”.
Mantle cell lymphoma (MCL) is a rare and aggressive subtype of B-cell non-Hodgkin lymphoma (NHL) with an annual incidence of one case per 200 000 people. Mantle cell lymphoma comprises around 5% of all non-Hodgkin’s lymphomas. MCL is typically diagnosed at advanced stages in elderly patients. Moreover, there is an unmet clinical need for new and effective therapies to treat Mantle cell lymphoma. Globally, several key companies are active in the Mantle cell lymphoma therapeutics market, with their drug candidate in various stages of clinical development. The further launch and approval of emerging therapies like LP-284 for Mantle Cell Lymphoma are anticipated to overcome the existing medical needs significantly.
