▎ WuXi AppTec content team editor
The M&A market in the biomedical sector is in the spotlight in 2022, with analysts estimating that large pharmaceutical companies have more than $1.7 trillion in "ammunition" available for trading, and on the other hand, a number of technological innovations have entered a fruitful stage, with cell therapies, gene therapies, and RNA- and DNA-based drugs with the potential to cure diseases. According to a research report by the well-known Ernst & Young (EY), although large pharmaceutical companies are also internally laying out the development capabilities of these technologies, harvesting external innovation through transactions is also an important means for them to innovate their drug development models.
In this environment, which biotech companies are high-profile acquisition targets in 2022? A recent article published by Fierce Biotech listed 10 biotechnology companies, of which as many as 7 focus on innovative treatment models such as cell therapy, gene therapy, and gene editing therapy. Today we take a look at the innovative technologies of these companies.
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 afamitresgene autoleucel (afami-cel, formerly known as ADP-A2M4), which targets the MAGE-A4 antigen, has yielded positive results in a key Phase 2 clinical trial for the treatment of advanced synovial sarcoma or mucoid/round cell liposarcoma (MRCLS), supporting this year's application for licensing of biological products (BLA) with the U.S. FDA. It could be the first TCR cell therapy approved to treat solid tumors.
▲ Adaptimmune's engineered TCR-T cell therapy (Image source: Adaptimmune official website)
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 Pharmaceuticals
Alnylam Pharmaceuticals is one of the star companies in the field of RNAi therapy development. The company 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.
Alnylam's breakthrough in lipid nanoparticle (LNP) and GalNAc coupling technology solves the problem of delivering RNAi therapy to the liver, paving the way for the development of RNAi therapy.
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.
Image source: Alnylam 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 Pharmaceutical
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 yielded positive results in a Phase 3 clinical trial for the treatment of patients with hemophilia A, where the annual bleeding rate (ABR) was 85% lower than baseline after two years of treatment with one gene therapy treatment, outperforming factor VIII prophylaxis. Based on trial data, BioMarin plans to resubmit a Biological Product 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
CRISPR Therapeutics was co-founded by Nobel laureate Dr. Emmanuelle Charpentier. The gene-editing therapy CTX001, developed jointly by the company with Vertex, has shown curative potential in clinical trials treating transfusion-dependent β thalassemia and severe sickle cell anemia. If the clinic goes well, 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
Karuna Therapeutics is at a critical stage. Two Phase 3 clinical trials of the company's research therapy KarXT for schizophrenia patients are expected to yield results this year. KarXT consists of two active ingredients, xanomeline and rospium chloride, designed to activate muscarinic acetylcholine receptors in the brain while reducing the effect of peripheral muscarinic acetylcholine receptors. By stimulating muscarinic receptors M1 and M4 receptors, the receptor agonist xanomeline relieves negative symptoms such as apathy and reduced social drive in patients with schizophrenia. It can also improve cognitive abilities and is helpful in improving other psychiatric symptoms such as hallucinations and delusions.
Xanomeline "teamed up" with trospium chloride to promote the birth of KarXT (Image source: Karuna Therapeutics official website)
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.