Paul Hudson, Chief Executive Officer, Sanofi
“Teva is entering a new era, and our robust, innovative pipeline is critical to our Pivot to Growth strategy.” This collaboration further supports Teva’s strong science with our internally created anti-TL1A. We are thrilled to collaborate with Sanofi to combine their proven strengths, leadership, and success in immunology and gastroenterology with our capabilities to optimize development and worldwide launches.”
Richard Francis, President and Chief Executive Officer, Teva
Teva will get an upfront payment of €469 million ($500 million) and up to €940 million ($1 billion) in development and launch milestones under the provisions of the new collaboration agreement. Each business will split development costs and net profits and losses in large markets equally, with minor areas subject to a royalty structure, and Sanofi will lead the Phase III program development. Teva will be in charge of product commercialization in Europe, Israel, and a few other countries, while Sanofi will be in charge of commercialization in North America, Japan, other parts of Asia, and the rest of the world. After normal closing conditions are met, the transaction will become effective. The first outcomes of the program are expected around 2024.
Amgen Completes Acquisition of Horizon Therapeutics PLC
Amgen announced the completion of its $116.50 per share cash acquisition of Horizon Therapeutics plc, reflecting a transaction equity value of approximately $27.8 billion.
“Today marks an exciting milestone as we welcome Horizon employees to Amgen and begin working together to serve even more patients around the world suffering from serious illnesses,” stated Robert A. Bradway, chairman and CEO of Amgen. “We have strong momentum in our core business and the addition of Horizon will further position Amgen as a leader across a broader range of diseases.”
The acquisition’s strong strategic and financial rationale includes:
- Inclusion in Amgen’s core strategy, focuses on groundbreaking medications that significantly improve the lives of patients afflicted with severe illnesses.
- Enhancement of Amgen’s leadership in treating inflammatory conditions by introducing pioneering, early-stage drugs like TEPEZZA (teprotumumab-trbw), KRYSTEXXA (pegloticase), and UPLIZNA (inebilizumab-cdon) for rare inflammatory diseases.
- Utilization of Amgen’s top-tier expertise in biologics research, development, and manufacturing, combined with a global presence spanning over 100 countries.
- Creation of a substantial cash flow stream to bolster capital allocation strategies, including continuous investments in innovation while maintaining a strong credit rating.
- Acceleration of revenue growth, with anticipated non-GAAP earnings per share accretion commencing in 2024.
Amgen expects to release an updated FY 2023 forecast on its third-quarter earnings call.
FDA Grants Orphan Drug Designation to GC Biopharma’s Drug Candidate for Thrombotic Thrombocytopenic Purpura
On October 5, 2023, GC Biopharma Corp. announced that its drug candidate, GC1126A, intended for the treatment of Thrombotic Thrombocytopenic Purpura (TTP), was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) on September 27, 2023.
GC1126A stands as a groundbreaking ADAMTS13 mutein meticulously engineered to circumvent autoantibodies, featuring an extended half-life. Presented at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in June, non-clinical findings showcased the drug candidate’s effectiveness in disease models while sustaining elevated activity levels in comparison to conventional treatments or wild-type ADAMTS13.
In response to this accomplishment, GC Biopharma expressed their dedication, stating, “We are dedicated to collecting data to develop a Best-in-Class treatment for such rare disorders. We remain committed to our mission of providing patients with new treatment options by continuously developing innovative drugs.”
Thrombotic Thrombocytopenic Purpura is a rare blood disorder. There are two main types of TTP, inherited/ congenital (cTTP) and acquired (aTTP). Inherited TTP is passed from parents to children through genes and mainly affects newborns and children, while acquired TTP is the more common type, primarily affecting adults. TTP impacts approximately 3 to 11 individuals per 1 million in the population. This life-threatening condition is characterized by the formation of small blood clots throughout the body, which obstruct the flow of blood to vital organs such as the brain and heart. If left untreated, TTP can lead to a mortality rate as high as 90%.
Plasma therapy (also called plasmapheresis) is the foundation of front-line and refractory Thrombotic Thrombocytopenic Purpura management. It involves two methods: fresh frozen plasma for people who have inherited Thrombotic Thrombocytopenic Purpura and plasma exchange for people who have acquired TTP. The proposed mechanism of therapeutic plasma exchange (TPE) supplies adequate levels of ADAMTS13 while removing circulating anti-ADAMTS13 autoantibodies. Any delay in therapy can lead to early mortality, which is preventable with prompt initiation of TPE. To further improve the Thrombotic Thrombocytopenic Purpura treatment scenario, globally, several major pharma and biotech companies are actively working in the Thrombotic Thrombocytopenic Purpura therapeutics market.
Anaptys Announces Positive Top-Line Phase 3 Clinical Trial Results of Imsidolimab (IL-36R) in Generalized Pustular Psoriasis (GPP)
On October 9, 2023, AnaptysBio, Inc. (Nasdaq: ANAB) unveiled encouraging top-line findings from its global Phase 3 GEMINI-1 trial, which assessed the safety and effectiveness of imsidolimab (IL-36R mAb) in patients experiencing flares of Generalized Pustular Psoriasis (GPP). The investigational imsidolimab successfully met its primary endpoint within the study population, achieving rapid clearance of pustules, erythema, and scaling within four weeks after a single 750mg IV imsidolimab dose. Additionally, the top-line data showcased a favorable safety and tolerability profile.
“The success of the GEMINI-1 trial highlights Anaptys’ commitment to patients and our ability to internally discover and develop differentiated antibodies,” said Daniel Faga, president and chief executive officer of Anaptys. “Moving forward, we intend to out-license imsidolimab with this compelling and competitive dataset to bring this therapy to patients living with this highly morbid condition and reallocate the potential proceeds of a transaction to further invest in the broad development of our best-in-class immune cell modulators, including our checkpoint agonists, in autoimmune and inflammatory diseases.”
“GPP is an unpredictable and potentially life-threatening skin disease with systemic symptoms,” said Professor Hervé Bachelez, M.D., Ph.D., Hôpital Saint-Louis, Paris, one of the world’s leading experts on GPP. “Achieving positive top-line results utilizing the GPPPGA composite endpoint in this well conducted, randomized controlled, global trial, along with a compelling safety profile, represents the potential for a single dose of imsidolimab to predictably provide relief for patients living with this burdensome disease.”
Imsidolimab is an IgG4 antibody designed to inhibit the activity of the interleukin-36 receptor (IL-36R), a critical component of the immune system’s signaling pathway that has been implicated in the development of inflammatory diseases, including GPP.
The GEMINI-1 trial, which aimed to support the drug’s registration for Generalized Pustular Psoriasis treatment, enrolled a cohort of 45 patients. It represents a significant milestone as the first randomized, double-blind, placebo-controlled trial to employ a composite endpoint for assessment. This composite endpoint, known as the GPP Physician Global Assessment (GPPPGA) in Week 4, offers a comprehensive evaluation of disease severity. To meet the GPPPGA criteria, patients needed to achieve an overall clinical response score of 0/1, indicating either clear or nearly clear status, across all aspects of GPP, encompassing pustulation, erythema, and scaling.
Imsidolimab Was Well Tolerated Through the End of the Study
- All AEs reported in imsidolimab-treated patients were mild or moderate and balanced across imsidolimab-treated vs. placebo patients
- No SAEs or severe AEs reported in imsidolimab-treated patients
- No cases reported of Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) or Guillain-Barré syndrome (GBS)
- Low incidence and no elevation of infections vs. placebo
- No infusion reactions reported
- One of 30 (3.3%) imsidolimab-treated patients had detectable anti-drug antibodies, which were non-neutralizing
Anaptys has intentions to unveil extensive findings from GEMINI-1 and the summary of GEMINI-2 outcomes during a medical conference scheduled for the second half of 2024. Additionally, the company has set its sights on submitting a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the third quarter of 2024.
“We are excited that these top-line results from the Phase 3 GEMINI-1 trial support that a single infusion of imsidolimab is efficacious and well tolerated,” said Paul Lizzul, M.D., Ph.D., chief medical officer of Anaptys. “We would like to thank the patients, investigators, and study personnel for their participation in this trial. We look forward to engaging with FDA and plan to submit a BLA by Q3 2024.”
Generalized Pustular Psoriasis is a rare and persistent systemic autoinflammatory ailment that can pose a serious threat to life when not appropriately addressed. GPP flares bring about the abrupt emergence of distressing pustules, often spanning extensive regions of the skin. It’s crucial to promptly diagnose and treat Generalized Pustular Psoriasis to manage its potentially severe consequences on both skin health and overall well-being. Pharma and biotech giants such as Anaptys, among others, are making significant strides in overcoming Generalized Pustular Psoriasis treatment challenges through innovative approaches and advancements in medical research. Companies are advancing Generalized Pustular Psoriasis treatment options by harnessing the power of biologics, conducting clinical trials, exploring combination therapies, supporting patients, raising awareness, investing in research, and obtaining regulatory approvals. These efforts collectively contribute to improved outcomes and a better quality of life for individuals living with GPP.
Boehringer Moves Obesity Drug into Phase III Clinical Trials
Boehringer Ingelheim is on the verge of making a notable entrance into the profitable obesity treatment market. The pharmaceutical company recently disclosed progress in advancing a potential treatment into three Phase III trials following promising data. This candidate, Survodutide, is a dual agonist of glucagon and glucagon-like peptide 1 (GLP-1), jointly developed with Zealand Pharma. During a Phase II dose escalation trial focused on patients with obesity but without type 2 diabetes (T2D), Survodutide showcased a significant weight loss of up to 19% after a 46-week treatment period.
In an official statement outlining their plans, Boehringer Ingelheim mentioned that insights gained from previous studies would guide the design of the upcoming Phase III trials, aiming to assess the effectiveness and safety of Survodutide. Patient enrollment for these trials is slated to commence before the conclusion of 2023. Further details about the trials will be disclosed closer to their initiation.
Survodutide had previously received fast-track designation from the US Food and Drug Administration (FDA) for its potential use in treating adults, both with and without T2D, who have liver fibrosis and non-alcoholic steatohepatitis (NASH). The drug is now set to compete with other GLP-1 agonists in the realm of weight loss treatments. Novo Nordisk’s Wegovy, a subcutaneous formulation of semaglutide, has been a dominant player in the market, while Eli Lilly’s retatrutide, with a triple-agonist mechanism of action, is emerging as a strong competitor, boasting impressive weight loss data.
Novo Nordisk’s stronghold on the weight loss market has been challenged due to supply constraints, opening doors for drugs in late-stage trials to enter the market. GlobalData predicts the market to reach $37.1 billion by 2031, and while Wegovy is expected to maintain its position as a top-selling drug, there’s room for promising candidates from late-stage trials.
What sets Boehringer Ingelheim’s candidate apart is its targeting of the hormone glucagon in addition to GLP-1. The efficacy of this dual agonist approach, compared to GLP-1 receptor mono-agonists, will be determined through the planned Phase III trials.
FDA Places Partial Clinical Hold on IND for Lacutamab in CTCL/PTCL
The FDA has imposed a partial clinical hold on the investigational new drug application for lacutamab (IPH4102), resulting in a temporary halt in the enrollment of new patients for two clinical trials: the phase 2 TELLOMAK trial (NCT03902184) and a phase 1b trial (NCT05321147). These trials assessed the effectiveness of lacutamab in patients with advanced cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL), respectively.
This decision was prompted by an unexpected and severe adverse event (AE) occurrence. Specifically, one patient experienced a fatal case of hemophagocytic lymphohistiocytosis. However, patients who were already receiving treatment and benefiting from it in either trial can continue their treatment after providing renewed consent.
Lacutamab is an innovative, anti-KIR3DL2 humanized cytotoxicity-inducing antibody under investigation for the treatment of CTCL and PTCL patients.
The TELLOMAK trial is a global, open-label, multi-cohort, multicenter study assessing lacutamab in patients with relapsed/refractory stage IVA/B CTCL who have previously received at least 2 prior systemic therapies, including mogamulizumab-kpkc (Poteligeo). The trial includes various cohorts, each focusing on different subsets of patients with CTCL.
All patients in these cohorts receive lacutamab treatment following a specific dosing regimen. The primary endpoint of the trial is the objective response rate, with secondary endpoints including safety, quality of life, progression-free survival, overall survival, and pharmacokinetics. TELLOMAK has completed enrollment, and final data are anticipated in the fourth quarter of 2023.
The phase 1b trial is evaluating the use of single-agent lacutamab in patients with relapsed/refractory PTCL expressing KIR3DL2 who have received at least 2 cycles of a prior line of treatment. Key inclusion criteria include specific levels of KIR3DL2 expression and other clinical indicators. Like the TELLOMAK trial, patients in this phase 1b study follow a particular dosing schedule. The primary endpoint for this trial is the frequency of adverse events, and preliminary data are expected in the fourth quarter of 2023, pending a futility interim analysis.
