Acoramidis Joins Bayer’s Robust Lineup, Boosting Cardiology Solutions

Bayer has obtained the exclusive rights to market acoramidis in Europe from Eidos Therapeutics Inc., BridgeBio International GmbH, and BridgeBio Europe B.V. Acoramidis, a highly potent and selective small molecule given orally, functions as a stabilizer for transthyretin (TTR). This medication is designed for the treatment of patients with ATTR CM, a progressive and ultimately fatal condition characterized by infiltrative, restrictive cardiomyopathy leading to heart failure. In a Phase III study of ATTR CM patients, acoramidis successfully achieved all clinical goals, notably reducing the burden of hospitalizations, enhancing survival rates, and maintaining the functional abilities and quality of life for those in need.

The request for approval to market acoramidis with the European Medicines Agency (EMA) was submitted in January 2024, following the announcement of acoramidis meeting the main goal in the crucial Phase III trial. This confirmed its significant effectiveness and favorable tolerability among the participants. Additionally, a New Drug Application (NDA) for acoramidis has been presented to the FDA and has been acknowledged, with the Prescription Drug User Fee Act (PDUFA) decision expected by November 29, 2024.

Juergen Eckhardt, M.D., a member of Bayer’s Pharmaceuticals Division Executive Committee and in charge of Business Development, Licensing & Open Innovation, stated that Bayer aims to revolutionize cardiovascular care for patients. He added that acoramidis is a valuable addition to their specialty cardiology portfolio. Being a key player in cardiovascular disease management, Bayer is committed to swiftly offering this new treatment to patients once it receives approval from European authorities.

According to the agreement, BridgeBio and its associates are set to receive a total of up to $310 million in initial and soon-to-be-achieved milestone payments. They also have the potential to earn further, unspecified sales milestone payments and tiered royalties starting at a percentage in the low-thirties.

EU Validates Two Datopotamab Deruxtecan Uses for Advanced Non-Squamous NSCLC & HR+/HER2- Breast Cancer

The European Medicines Agency (EMA) has given its approval to two requests for marketing authorization for datopotamab deruxtecan (Dato-DXd), a medication developed by AstraZeneca and Daiichi Sankyo, for use in treating two specific types of cancer. One application is for the treatment of adults with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) who need systemic treatment after prior therapy. The second application is for adults with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have experienced progression despite endocrine therapy and have undergone at least one other systemic treatment.

The validations serve to verify the finalization of the applications and mark the beginning of the scientific assessment carried out by the EMA’s Committee for Medicinal Products for Human Use. These submissions are founded on the data from the significant Phase III trials, TROPION-Lung01 and TROPION-Breast01, showcased during two Presidential Symposia at the 2023 European Society for Medical Oncology Congress.

Susan Galbraith, the Executive Vice President of Oncology Research and Development at AstraZeneca, expressed the aspiration for datopotamab deruxtecan to enhance and supplant traditional chemotherapy in addressing various forms of cancer. The recent confirmation of our submissions for lung and breast cancers signifies a significant advancement for this potential medication, moving it closer to reshaping the treatment landscape for patients with two prevalent types of cancer in Europe.

Further regulatory applications for datopotamab deruxtecan in both lung cancer and breast cancer are currently in progress within the United States and internationally.

AbbVie Joins Forces with OSE Immunotherapeutics for Innovative Monoclonal Antibody Targeting Chronic Inflammation

AbbVie Inc. and OSE Immunotherapeutics SA have revealed a significant collaboration aimed at advancing OSE-230, a monoclonal antibody meant to address persistent and serious inflammation. This antibody, still in its early pre-clinical phase, is innovative as it activates ChemR23, a G-Protein Coupled Receptor (GPCR) target. This activation of ChemR23 could provide a fresh approach to resolving long-term inflammation, influencing the activities of macrophages and neutrophils.

Jonathon Sedgwick, Ph.D., the senior vice president and global head of discovery research at AbbVie, emphasized the company’s dedication to broadening its immunology lineup with the primary aim of enhancing the treatment standards for individuals worldwide grappling with inflammatory conditions. He stated, “Through this partnership, we aim to propel the development of OSE-230, which introduces an innovative approach to addressing chronic inflammation, capitalizing on our proficiency in immunology drug advancements.”

“We are delighted to partner with AbbVie, a renowned worldwide expert in creating and bringing groundbreaking medicines to market, in advancing our OSE-230 program,” stated Nicolas Poirier, CEO of OSE Immunotherapeutics. “This collaboration marks a significant achievement for our organization and underscores the worth of our pioneering research and development strengths. I extend my appreciation to all our staff whose dedication and diligence have led us to this important milestone.”

According to the agreement, AbbVie will gain the sole worldwide rights to develop, produce, and market OSE-230. OSE Immunotherapeutics will be given an initial payment of $48 million and could potentially earn up to $665 million more through various milestones in clinical development, regulatory processes, and commercial achievements. Additionally, OSE Immunotherapeutics stands to receive incremental royalties based on the global sales of OSE-230. The completion of this transaction depends on meeting standard closing requirements, including the necessary waiting period as per the Hart-Scott-Rodino Antitrust Improvements Act.

vTv Therapeutics Makes Major Move: Cadisegliatin Heads for FDA’s Phase 3 Study in Type 1 Diabetes

On March 04, 2024, vTv Therapeutics Inc. (Nasdaq: VTVT), a pioneering clinical-stage biopharmaceutical firm dedicated to advancing innovative treatments, took a significant step forward in its mission to revolutionize diabetes care. The company unveiled its latest milestone by submitting the study protocol to the FDA for its inaugural Phase 3 trial. This trial aims to rigorously assess the safety and efficacy of cadisegliatin (TTP399), the company’s flagship candidate, when used alongside insulin therapy in adults grappling with the challenges of type 1 diabetes (T1D). Cadisegliatin (TTP399) is a liver-selective glucokinase activator currently under investigation. Its efficacy and safety have been examined in both healthy volunteers and individuals diagnosed with type 1 and type 2 diabetes. Cadisegliatin holds promise as an adjunctive therapy to insulin, potentially offering individuals with T1D enhanced control over their blood glucose levels and improved quality of life. 

The submission of the study protocol signifies a pivotal moment in the journey toward regulatory approval. Upon FDA approval and subsequent initiation of the Phase 3 trial, vTv Therapeutics will embark on an exhaustive evaluation of cadisegliatin’s performance in real-world settings. This trial will enlist adults diagnosed with T1D, underscoring the company’s commitment to addressing the pressing healthcare challenges faced by this demographic. By advancing cadisegliatin into Phase 3 clinical trials, vTv Therapeutics reaffirms its dedication to translating scientific innovation into tangible improvements in patient care. The company’s steadfast pursuit of excellence in biomedical research heralds hope for a future where individuals living with T1D can experience greater autonomy and well-being.

Anticipated to commence in the second quarter of 2024, this Phase 3 trial is meticulously designed as a randomized, double-blind, placebo-controlled study. It aims to recruit around 150 participants across a network of up to 20 sites situated throughout the United States. The trial targets patients currently managing their condition with multiple daily insulin injections and continuous subcutaneous insulin infusion, and who utilize a continuous glucose monitor (CGM).

In this trial, subjects will receive either two different doses of orally administered cadisegliatin or a placebo. The primary objective of this study is to compare the frequency of Level 2 or Level 3 hypoglycemic events between individuals treated with cadisegliatin and those administered with the placebo. This critical endpoint serves as a benchmark to evaluate the efficacy of cadisegliatin as an adjunctive therapy alongside existing insulin regimens for individuals grappling with type 1 diabetes.

“vTv’s primary focus is on expeditiously confirming the safety and efficacy of cadisegliatin, and this initial Phase 3 trial will help provide a more robust body of clinical evidence on the drug’s profile in a relatively short timeframe. The ongoing support from institutional investors who participated in our recent private placement reflects the urgent need for treatments that improve glycemic control and have a positive impact on the quality of life of patients with T1D,” said Thomas Strack, MD, PhD, Chief Medical Officer, vTv Therapeutics.

This trial marks a significant extension of vTv’s exploration into the potential of cadisegliatin as a complementary treatment to insulin. This endeavor includes an upcoming Phase 2 trial targeting patients with type 2 diabetes, set to commence in Middle Eastern countries in 2024. This initiative is a collaborative effort between vTv and its partner, G42 Healthcare Research Technology Projects LLC, along with the clinical research organization IROS, a prominent health technology group based in the United Arab Emirates. Cadisegliatin has not received licensure or approval for use anywhere worldwide, and its safety and efficacy for any medical application have not been confirmed.

Currently, insulin therapy remains the cornerstone of treatment for Type 1 Diabetes. However, many individuals still struggle to achieve optimal glycemic control, leading to complications and reduced quality of life. Cadisegliatin offers the promise of enhancing blood glucose management when used alongside insulin, potentially reducing the frequency of hypoglycemic events and improving overall glycemic stability.

If approved, Cadisegliatin could provide healthcare providers with a valuable additional tool in their arsenal for managing Type 1 Diabetes. This could lead to a shift in treatment paradigms, with Cadisegliatin becoming a standard adjunctive therapy alongside insulin. Furthermore, its introduction could stimulate competition and innovation in the diabetes treatment market, potentially leading to improved outcomes and increased options for patients.

Overall, the submission of the Phase 3 study for Cadisegliatin represents a hopeful step forward in addressing the unmet needs of individuals living with Type 1 Diabetes. It has the potential to significantly impact the treatment landscape and market outlook in the upcoming years, offering new hope and possibilities for improved management of this chronic condition.

A2 Bio Scores FDA Orphan Drug Designation for Groundbreaking Cell Therapy Program A2B530 in Colorectal Cancer

A2 Biotherapeutics, Inc. (A2 Bio) made a significant announcement on March 4, 2024, revealing that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its therapy, A2B530. This designation applies to its potential application in treating a specific subset of patients suffering from colorectal cancer. Specifically, it targets germline heterozygous HLA-A02(+) individuals whose cancer cells express carcinoembryonic antigen (CEA) but have experienced loss of HLA-A02 expression. The Orphan Drug Designation underscores the innovative potential of A2B530 in addressing the unique needs of this patient population, representing a milestone in the quest for more effective treatments in oncology. 

As per the updates, enrollment continues in EVEREST-1, an integrated Phase 1/2 clinical trial designed to assess the safety and efficacy of A2B530 across a spectrum of cancers including colorectal, pancreatic, and non-small cell lung cancers.

A2B530 marks a groundbreaking advancement as the inaugural autologous logic-gated cell therapy arising from A2 Bio’s pioneering Tmod™ platform. This innovative platform is engineered with a dual-receptor architecture comprising an activator, which homes in on tumor cells, and a blocker, designed to shield healthy cells. The distinctive feature of this platform addresses a core obstacle in the realm of solid tumor cancer treatments: the imperative to discriminate between malignant and healthy cells effectively. A2B530, leveraging this platform, features an activator meticulously tailored to target carcinoembryonic antigen (CEA) and a blocker adept at pinpointing HLA-A*02. This strategic combination holds immense promise in revolutionizing cancer therapeutics by delivering targeted therapy while minimizing collateral damage to healthy tissues. Such precision-engineered treatments offer a glimpse into the future of oncology, where therapeutic interventions can be tailored with unprecedented specificity, maximizing efficacy and minimizing adverse effects. 

“The FDA granting Orphan Drug Designation validates the tremendous unmet need for improved therapies for patients with colorectal cancer,” said William Go, M.D., Ph.D, Chief Medical Officer of A2 Bio. “This designation supports our commitment to use our novel technology platform to develop new treatment options for patients with difficult-to-treat cancers.”

According to DelveInsight, the total number of new cases of Colorectal Cancer in 2022 stood at approximately 546,000 cases across the 7 Major Markets (7MM), with expectations of a potential increase by 2032. Among these markets, the United States reported the highest incidence of Colorectal Cancer, with approximately 151,000 cases documented in 2022. Additionally, the total number of new cases of metastatic Colorectal Cancer (mCRC) in the 7MM reached around 237,000 cases in 2022. The Orphan Drug Designation granted to A2B530 represents a significant breakthrough in the field of colorectal cancer therapeutics, as it offers a tailored approach that addresses an unmet medical need. This designation not only highlights the therapeutic potential of A2B530, but also streamlines its development process, providing incentives and support for further research and clinical trials. As a result, A2B530 has the potential to introduce a new dimension to the colorectal cancer therapeutics market, offering hope to patients and healthcare providers alike by overcoming existing treatment challenges and paving the way for more effective and personalized treatment options.

Artiva Biotherapeutics Rockets Forward with FDA Fast Track Designation for AlloNK® in Lupus Nephritis

Artiva Biotherapeutics made a significant announcement on February 22, 2024, revealing that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its flagship program, AlloNK® (also referred to as AB-101). This designation is specifically for the treatment of Lupus Nephritis (LN) when used alongside rituximab or obinutuzumab. This development underscores the growing recognition of AlloNK’s potential in addressing autoimmune diseases.

Prior to this, Artiva had already received FDA clearance for an Investigational New Drug (IND) application for AlloNK in combination with rituximab to tackle LN. Notably, this marked a milestone as it represented the first instance of an allogeneic, off-the-shelf NK cell therapy receiving IND clearance for use in autoimmune diseases. 

AlloNK® (or AB-101) is an allogeneic NK cell therapy aimed at enhancing the antibody-dependent cellular cytotoxicity (ADCC) effect of monoclonal antibodies or NK cell engagers. It’s a cryopreserved, off-the-shelf therapy with potential for community-based administration. Being non-genetically modified and manufactured without integrating vectors, AlloNK eliminates the need for extensive patient follow-up and avoids potential secondary malignancy risks linked with approved autologous CAR-T therapies. Leveraging Artiva’s cell therapy manufacturing platform, thousands of doses of infusion-ready AlloNK cells can be generated from a single umbilical cord blood unit, maintaining consistent CD16 expression and activating NK receptor levels.

AlloNK is currently undergoing investigation in a multi-center, open-label clinical trial aimed at evaluating both its safety profile and clinical effectiveness when administered alongside anti-CD20 antibodies. This trial is specifically targeting patients diagnosed with Lupus Nephritis (LN) who either experienced relapse or showed inadequate response to previous standard treatment regimens. The trial is registered under the identifier NCT06265220 on

Furthermore, the safety and efficacy of AlloNK, particularly in combination with rituximab, have been established through a Phase 1/2 multicenter clinical trial. 

“The FDA Fast Track designation gives us an opportunity to accelerate our efforts to bring our AlloNK cell therapy to autoimmunity patients,” said Fred Aslan, M.D., Chief Executive Officer of Artiva. “We are encouraged by clinical data from our Phase 1/2 multicenter clinical trial in non-Hodgkin lymphoma, where we observed that AlloNK in combination with rituximab can drive deep B-cell depletion in patients with late-line B-cell cancers. Our therapy has a mechanism very similar to the B-cell targeted autologous CAR-T therapies, but with the benefits of being an off-the-shelf therapy with a better safety profile, and that we believe will not be subject to the secondary malignancy risk associated with genetically engineered cell therapies.”

According to DelveInsight, the total number of diagnosed prevalent cases of Systemic Lupus Erythematosus (SLE) in the 7 Major Markets (7MM) was estimated to be around 657,000 cases in 2022, with projections indicating a rise in the coming years. Specifically, in the United States, the total diagnosed cases of Lupus Nephritis reached nearly 130,500 in 2022. Within the European Union (EU4) and the United Kingdom, the UK registered the highest number of Lupus Nephritis cases, followed by Germany, while France reported the fewest cases in 2022. The FDA’s Fast Track Designation for AlloNK® in Lupus Nephritis heralds a promising new chapter in the treatment landscape for this challenging condition.

By expediting the regulatory review process, this designation accelerates the potential availability of AlloNK® alongside standard therapies like rituximab or obinutuzumab. This swift pathway not only offers hope to patients grappling with Lupus Nephritis but also injects fresh optimism into the overall market outlook. AlloNK®’s unique mechanism of action, coupled with its off-the-shelf nature and safety profile, holds the potential to alleviate the burden on patients and healthcare systems alike. With this designation, there’s a tangible shift towards a more efficient and effective treatment paradigm for Lupus Nephritis, promising improved outcomes and quality of life for those affected.