Next Breakthrough: Innovative Therapies Approach a Trillion-Dollar Market—Can They Break the Lifelong Medication Curse?

Jan 21, 2024 07:20 CST Updated 07:20

Kyverna

Developer of Immunocyte Therapy

Cartesian Therapeutics

Pharmaceutical R&D Developer

Nektar Therapeutics

Pharmaceutical R&D Developer

January 21, 2024

eMedClub News

Autoimmune diseases affect approximately 5%-8% of the global population. Not only are there numerous types of these diseases, but once diagnosed, patients often require lifelong medication. In fact, among the global pharmaceutical sales contributions, autoimmune disease drugs rank just behind those for oncology and infectious diseases. According to Frost & Sullivan data,The Global Autoimmune Drug Market Size Was Nearly One Trillion RMB in 2022It is estimated that by 2030, the global market size will reach 1.3 trillion RMB.

However, the development of biologic innovative drugs in the autoimmune field is still in a stage of rapid growth. Traditional autoimmune therapies have limited efficacy, significant side effects, and relatively concentrated indications, leaving ample room for targeted drugs to make an impact. The fact that Argenx generated $400 million in revenue in the first year and saw its stock price increase 30-fold with just Efgartigimod (the world’s first FcRn inhibitor) offers a glimpse into the enormous demand for innovative drugs in the autoimmune market.

In the past two days, Kyverna Therapeutics, a novel T-cell company, filed for an IPO, and Tr1X Bio, a rising star in Treg therapy, completed a $75 million Series A financing round, both revealing the prominence of novel cell therapies.Focus on AutoimmunityThe trend is gaining momentum, and the speed competition among major companies is about to unfold.

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CAR-T: Moving Forward Together, Blooming in Multiple Areas

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The first to enter the battlefield is undoubtedly CAR-T therapy. Its tremendous success in the field of B-cell hematological malignanciesThe impressive efficacy has driven this therapy to continue expanding its indications, and B-cell-driven autoimmune diseases have naturally become the next promising choice after cancer. Following the encouraging results achieved by the world's first systemic lupus erythematosus patient treated with CAR-T cell therapy in 2021, CAR-T officially opened the door to autoimmune diseases.

From the table, we can easily see that currently, CAR-T therapies based on B-cell targets CD19 and BCMA are the main force in expanding into autoimmune diseases. Among them, there are already marketed products, which are expected to gain an advantage in regulatory approval again due to their technical maturity. Besides,The development progress of companies in the autoimmune field is evenly matched both in China and abroad., most entered clinical trials in 2023.

▲ CAR-T Therapy Autoimmune Indication Pipeline (Source: MedMerck整理)）

Erythematosus Lupus (SLE), acknowledged by Carl H. June, the father of CAR-T, is naturally the preferred target for companies navigating autoimmune diseases. However, more companies are opting for a preemptive, differentiated layout of indications across a variety of disease types, aiming to establish a first-mover advantage and benefit more patients. For latecomers in the CAR-T field, this also presents an excellent opportunity to overtake competitors on the curve.

In August 2022, the BCMA CAR-T product Equecabtagene Autoleucel (CT103A) Injection, co-developed by Innovent and IASO Biotherapeutics, had not yet received marketing approval for the indication of relapsed/refractory multiple myeloma. However, its IND for neuromyelitis optica spectrum disorder (NMOSD) was approved in China, marking the first such approval in China.The world's first CAR-T cell therapy for neuromyelitis optica spectrum disorder (NMOSD).

New Approach in Cell Therapy: Late Bloomer, Promising Future

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Relying on the prominent regulatory capabilities of cell therapy for B cells and T cells, in addition to enabling the production of CAR-T targeted antibodies targeting CD19 or (and) BCMA, researchers have also developed a variety of innovative approaches, among which...Chimeric Autoantibody Receptor CAAR-T Targets Autoantibodies, CAR-Treg Targets Regulatory T Cells Most Mature。Whether it is controlling autoreactive cells or strategically modifying regulatory cells to control autoimmune destruction, preliminary data suggests that novel cell therapies hold promise for achieving long-term drug-free remission in autoimmune diseases.

▲ Three CAR-T Strategies for Treating Autoimmune Diseases (Source:DOI:10.1007/s00011-021-01470-1）

Recently, Cartesian Therapeutics announced its first-in-class BCMA-targetedmRNA CAR-TTherapy Descartes-0812-Month Follow-Up Data from Phase 2a Clinical Trial in Patients with Myasthenia Gravis (MG). Patients did not require lymphodepleting chemotherapy and achieved significant and sustained clinical improvements across four validated MG disease scoring systems.

Also entering Phase II is Treg star company Nektar Therapeutics, with the 1b phase data blunder impacting the treatment of atopic dermatitis (AD).Treg-Induced IL-2 Therapy RezpegThe highly anticipated. New data presented at the 2023 EADV Congress showed that, at the highest study dose, the proportion of DLQI (Dermatology Life Quality Index) responders was 75% at week 12, with dose-dependent improvement sustained beyond 36 weeks post-treatment. No anti-REZPEG antibodies were detected, indicating REZPEG's potential to target immune system imbalance for durable disease improvement in patients.

More notably,The World's First Clinical Trial Data of CAR-NK Therapy for Lupus Erythematosus Born in China. Originated from Enrui Kainuo's internationally leading new generationUniversal Off-the-Shelf CAR-NK Cell Therapy KN5501Four severe patients achieved deep remission after receiving this cellular therapy, transitioning from "severe" to "mild," and demonstrated corresponding durability. Two of the severe patients reached deep remission after 15 days of treatment, showing very positive therapeutic effects. SNC109, the allogeneic CD19 CAR-NK product developed by Senova Biopharma, has also filed an IND application for moderate-to-severe refractory systemic lupus erythematosus as its fourth extended indication.

▲ Novel Cell TherapyAutoimmune Indications Pipeline (FigureSource:Compiled by MedMickey）

In novel cell therapies,Pioneers' Steps Gradually Enter Key Clinical TrialsThe feasibility of the therapy continues to be validated and advanced, even surpassing CAR-T in progress; while the newcomers, although still in the early stages of development, are mostly venturing into the unknown based on existing experience.Continuously Innovate Drug Development Approaches, with multi-dimensional innovations in versatility, targeting, and drug delivery methods to address the current pain points of cell therapy. In the future, it will certainly carve out a differentiated blue ocean market through exploration.

With the increasing depth of mechanistic and clinical research in the field of novel cell therapies for various autoimmune diseases such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), multiple sclerosis (MS), and type 1 diabetes (T1D) in recent years, the targeting mechanisms have become clearer, the sites more precise, and the approaches more diverse. Cell therapy treatments are emerging like mushrooms after rain, ready to break through. It is believed that with continuous advancements and breakthroughs in technological thinking, novel cell therapies will once again make significant progress in autoimmune diseases, overcome existing treatment limitations, meet more unmet clinical needs, and have great potential in the next trillion-dollar market.

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