The M&A market in the biomedical sector has attracted considerable attention in 2022, and a number of technological innovations have entered the project output stage. According to the incomplete statistics of the People's Daily health client, the global biomedical financing exceeded 7.9 billion US dollars in January, and cell therapy is still the most popular "cutting-edge technology", with a total of nearly 20 financing events; gene therapy/gene editing therapy is the third most popular cutting-edge technology in January, with about 10 financing and IPO events.
In this environment, which biotech companies are high-profile acquisition targets in 2022? The People's Daily Health Client combed through the relevant article published by Fiere Biotech on February 15 and found that the following 10 biotechnology companies have attracted much attention, of which up to 7 companies focus on innovative treatment models such as cell therapy, gene therapy, and gene editing therapy.
Adaptimmune
Adaptimmune is committed to developing T cell receptor (TCR) cell therapies for the treatment of a variety of solid tumors. The company's T-cell therapy, which targets the MAGE-A4 antigen, has yielded positive results in a critical Phase 2 clinical trial for the treatment of advanced synovial sarcoma or mucoid/round cell liposarcoma (MRCLS), supporting the submission of a biologics licensing application (BLA) to the U.S. FDA this year. It could be the first TCR cell therapy approved to treat solid tumors.
The company's next-generation TCR cell therapy not only expresses TCR that targets tumor antigens on the surface of T cells, but also adds CD8α receptors that enhance T cell activity. A new generation of TCR cell therapies developed based on this strategy that target MAGE-A4 have shown better anti-cancer activity in Phase 1 clinical trials. The company also struck a $3 billion research and development deal with Roche's Genentech Corp. last year to develop allogeneic TCR cell therapies based on induced pluripotent stem cells (iPSCs) to treat a variety of cancers.
Alnylam
Alnylam Pharmaceuticals already has three FDA-approved RNAi therapies in its pipeline. Its subcutaneous injection of RNAi therapy vutrisiran is under review by the US FDA and is expected to be approved in the first half of this year. Currently, the company is working on developing delivery technologies that target organs outside the liver, and has made breakthroughs in delivery targeting the central nervous system. At last year's R&D day, the company said that RNAi therapy is expected to benefit patients with common diseases.
Aerial view of the Alnylam Product Development Park. Picture according to Alnylam's official website
Alnylam has reached a multi-year R&D agreement with Regenerator to develop RNAi therapies for the treatment of ophthalmic and central nervous system diseases. The company also recently reached an agreement with Novartis to develop potential therapies to treat liver failure.
BioMarin
BioMarin Pharmaceutical has seven approved therapies, of which Voxzogo (vosoritide) for the treatment of children over the age of 5 with achondroplasia is the first therapy to receive FDA approval for the treatment of the disease.
The company's gene therapy valoctocogene roxaparvovec has yielded positive results in a Phase 3 clinical trial treating patients with hemophilia A, and based on trial data, BioMarin plans to resubmit a biologics licensing application (BLA) to the FDA in the second quarter of this year. In addition to hemophilia A gene therapy, the company has two gene therapies in Phase 1 clinical trials for the treatment of phenylketonuria and hereditary angioedema. It also struck a research and development agreement with Skyline Therapeutics last year to jointly develop gene therapies to treat inherited cardiovascular diseases.
CRISPR Therapeutics
The gene-editing therapy CTX001, jointly developed by CRISPR Therapeutics and Vertex, has shown curative potential in clinical trials treating patients with transfusion-dependent β thalassemia and severe sickle cell disease. The therapy is expected to submit a marketing application to the United States this year, becoming the first approved therapy based on CRISPR gene editing technology.
In addition, the company is also using CRISPR/Cas9 gene editing to develop allogeneic cell therapies for the treatment of cancer, and three allogeneic CAR-T cell therapies in the development pipeline using CRISPR gene editing have entered the clinical development stage. The allogeneic islet β cell replacement therapy developed by the company in collaboration with ViaCyte has also entered the clinical development phase. The therapy aims to produce islet β cells that avoid attack by the patient's immune system, with the ultimate goal of curing patients with type 1 diabetes.
Ionis Pharmaceuticals
Ionis Pharmaceuticals has developed an antisense oligonucleotide (ASO) drug discovery platform that targets RNA to treat or prevent the onset of disease by blocking the production of disease-associated proteins. The company already has three approved therapies, including Spinraza, which treats spinal muscular atrophy, the first FDA-approved ASO therapy.
The company has partnered with large pharmaceutical companies such as Roche, Pfizer, Bayer, AstraZeneca, and Novartis to develop a variety of ASO therapies for cardiovascular and rare diseases. Its ligand coupling antisense (LICA) technology makes it possible to accurately deliver ASO drugs to specific tissues or cells by coupling ligand molecules that bind to specific receptors with ASO molecules.
Karuna Therapeutics
Two Phase 3 clinical trials of KarXT, an investigative therapy developed by Karuna Therapeutics for the treatment of schizophrenia patients, are expected to yield results this year. KarXT consists of two active ingredients, xanomeline and rospium chloride, which relieve negative symptoms such as apathy and reduced social drive in patients with schizophrenia by stimulating muscarinic receptors M1 and M4 receptors, and the receptor agonist xanomeline. It can also improve cognitive abilities and is helpful in improving other psychiatric symptoms such as hallucinations and delusions.
In November last year, Zaiding Pharmaceutical and Karana Therapeutics entered into an exclusive licensing agreement for the development, production and commercialization of KarXT in Greater China. In addition to treating schizophrenia, KarXT is also used in clinical trials to treat psychotic episodes in Alzheimer's disease patients.
Targeted Therapeutics
Mirati Therapeutics' Kras G12C inhibitor a new drug application (NDA) for Adagrasib has been accepted by the FDA for the treatment of treated patients with non-small cell lung cancer with KRAS G12C mutations. In addition to non-small cell lung cancer, it has also shown promising activity in the treatment of colorectal, pancreatic and gastrointestinal cancers.
Another of the company's innovative therapies in the late stages of development is the polykinase inhibitor sitavatinib. This innovative therapy is currently being tested in Phase 3 clinical trials in combination with anti-PD-1 antibodies to treat patients with non-small cell lung cancer.
Sarepta Therapeutics
Sarepta already has three FDA-approved oligonucleotide therapies to treat specific types of DMD patients by promoting exon jumps. The company's R&D pipeline also includes more than 40 R&D projects that utilize oligonucleotides, gene therapies and gene editing to treat a range of genetic diseases, including DMD and Rett syndrome.
In addition, the gene therapy SRP-9001, developed in collaboration with Roche, has launched a global Phase 3 clinical trial. This therapy packages transgenic expressions of anti-micromustrophin in an AAV viral vector, and by transfecting the patient's muscle cells, allowing the muscle to produce a recombinant protein with partial anti-amyotrophin function, which can take effect in patients carrying any type of DMD gene variant.
UniQure
The gene therapy etranacogene dezaparvovec, jointly developed by uniQure and CSL Behring, has reached the primary endpoint in a Phase 3 clinical trial for the treatment of patients with hemophilia B. The therapy, which can perform normal coagulation function at lower expression levels, has been granted breakthrough therapy designation by the U.S. FDA, and CSL Behring plans to submit a regulatory application to the FDA in the first half of 2022.
In addition, the company's gene therapy for Huntington's disease, AMT-130, has also launched clinical trials in the United States and Europe, and is expected to obtain preliminary safety and efficacy results in the second quarter of this year.
Vertex Pharmaceuticals
Vertex has several approved therapies for cystic fibrosis. In recent years, the company has continued to lay out new treatment models such as gene editing, cell therapy, and RNA therapy. The gene-editing therapy CTX001, developed in collaboration with CRISPR Therapeutics, is expected to file a marketing application in the United States this year.
The company's islet cell replacement therapy, obtained through the acquisition of Semma Therapeutics, made a breakthrough last year. This stem cell-differentiated islet cell replacement therapy reduced daily insulin use by 91% in the first patient with type 1 diabetes mellitus (T1D) who was treated in a Phase 1/2 clinical trial. It shows its potential to significantly restore islet cell function in patients with T1D.
The company also collaborates with Moderna, which is working on mRNA vaccines and therapies, and Arbor Biotechnologies, which is working on next-generation gene-editing therapies. (Compiled by Liu Yingqi)
Source:
The top 10 M&A targets in biotech for 2022 | Fierce Biotech
https://www.fiercebiotech.com/special-report/top-10-m-a-targets-biotech-for-2022