Bayer has made exciting progress in transforming its pharmaceutical business with breakthrough innovations in healthcare that will significantly help patients suffering from conditions that are currently still difficult to treat. In particular, Bayer has big plans in cell and gene therapy.
The information was stressed at its virtual Pharma Media Day titled “Transforming Healthcare. Transforming Bayer” in Germany. The event highlighted Bayer’s focus on the cell, gene, and oncology pipelines.
“The biomedical and technological revolution that is transforming healthcare at an unprecedented pace is taking place now. Our company is at the forefront of the wave of innovation in cell and gene therapy as well as digital health,” said Stefan Oelrich, member of the board of management at Bayer AG and President of Bayer’s Pharmaceuticals Division. “We are driving this transformation and growing our promising development portfolio together with our partners. Our joint goal is to bring breakthrough treatments to patients and make healthcare systems more sustainable in the mid- and long-term.”
The company has recently heavily invested in external innovation with an unprecedented number of more than 25 collaboration agreements and acquisitions. In particular, the company has emerged as a leader in the field of cell and gene therapies.
Cell and gene therapies offer for the first time the possibility to address the root cause of the disease, providing options for conditions considered intractable or where the current standard of care only addresses symptoms to different degrees. Bayer’s increasing investments in the field are consolidating the company’s emerging leadership and confirm its strategic significance as a growth-driver for its pharmaceutical business.
Bayer has just established a new Cell and Gene Therapy Platform. This platform steers Bayer’s strategy in the area and orchestrates all activities along the value chain providing an innovation ecosystem for all partners, including BlueRock Therapeutics and Asklepios BioPharmaceutical (AskBio), two companies fully owned by Bayer but independently operated.
Bayer’s development portfolio of cell and gene therapies already comprises seven advanced assets in different stages of clinical development. These are focused on multiple therapeutic areas with high unmet need, such as neurodegenerative, neuromuscular, and cardiovascular indications, with leading programmes in Pompe disease, Parkinson’s disease, hemophilia A, and congestive heart failure. With over 15 preclinical assets in the cell and gene therapy field, the pipeline is expected to grow steadily year by year.
“Cell and gene therapies hold the promise to significantly impact patients’ lives by moving from treating symptoms to potentially curative approaches,” said Wolfram Carius, executive vice president and head of Cell and Gene Therapy at Bayer. “Together with our partners, we want to accelerate innovation at its source and along the whole value chain to ensure a fast translation of science into therapies for patients who have no time to wait.”
BlueRock Therapeutics recently announced that the US Food and Drug Administration (FDA) has cleared their Investigational New Drug application to proceed with a Phase I study in patients with advanced Parkinson’s disease. This will be the first trial in the US to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease and a big step forward for the stem cell field.
In addition, digital healthcare offerings have become an important pillar of modern healthcare. The combination of conventional healthcare approaches with innovative digital technologies makes it possible to offer efficient solutions addressing the needs of a person in his/her specific environment. Bayer is developing Integrated Care concepts which offer individual support of patients. This personalised approach addresses complex and interconnected health challenges within the individual circumstances.
Integrated Care is a key element of Bayer’s digital business strategy in pharma. In collaboration, Bayer and the digital health company Informed Data Systems Inc. (IDS/One Drop) are expanding their business based on IDS’s existing diabetes management platform. This solution was downloaded more than three million times. Together, the companies are now creating new health offerings to address the needs of patients in the areas of cardiovascular diseases, women’s health, and oncology. Experts from both companies are jointly working on the first two modules, which are to become available within a year.
Furthermore, Bayer is continuing to build a strong development pipeline advancing more than 50 projects through the clinic with a focus on cardiovascular diseases, oncology and women’s health. The company highlighted two promising pipeline programmes in mid-stage development demonstrating medical innovation at Bayer.
Factor XI (FXI)-targeting compounds, which are a new class of anticoagulants that is comprised of three investigational assets, and an anti-FXIa antibody and a FXI-ligand-conjugated antisense oligonucleotide (FXI-LICA) that are being studied in patients with end-stage kidney disease. FXI-pathway inhibition may offer protection from thromboembolic events without increased risk of bleeding.
With its P2X3 multi-indication programme, Bayer highlighted another important candidate in mid-stage development. At Bayer, the promise of P2X3 antagonists was first identified for endometriosis within the company’s strategic research alliance with Evotec, a Germany-based drug discovery and development company. Endometriosis is a clinical condition affecting approximately 10 per cent of women in reproductive age, many of whom experience severe chronic pain with debilitating effects on their professional, personal, and social lives. P2X3 also has a prominent role in several other medical conditions associated with pain and neurogenic hypersensitivity such as chronic cough, overactive bladder, and neuropathic pain.
“Our research in the fields of Factor XI inhibitors and P2X3 antagonists are just two promising examples of mid-stage programmes that demonstrate our ongoing commitment to building and advancing a strong development pipeline,” said Stefan Oelrich. “Positioning Bayer as a leader in the highly dynamic space of healthcare innovation, we will continue to lead the field in bringing new solutions to patients who need them.”
At the same time, the company is also successfully delivering on its late-stage pipeline in the areas of oncology and cardiovascular disease including also a number of potential blockbuster products. In the area of oncology, for example, darolutamide (jointly developed with Orion Corporation), a differentiated treatment option that extends survival for men with non-metastatic castration-resistant prostate cancer who are at high risk for developing metastatic disease (nmCRPC), and shows a favourable safety profile.
The product has received regulatory approval in several markets, including the US, the EU, Brazil, Canada, and Japan. The Phase III study ARASENS in the indication of metastatic hormone sensitive prostate cancer (mHSPC) is expecting data read-out in 2021. Larotrectinib is a first-in-class precision oncology treatment designed to treat patients with solid tumours harbouring a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion (1). The product is approved in more than 40 countries, including the US and the EU. It was the first treatment in the EU to receive a tumour-agnostic indication.
In the field of women’s healthcare, Bayer recently added a highly attractive asset to its development portfolio through the acquisition of KaNDy Therapeutics. BAY-342 (formerly NT-814) is a first in class, non-hormonal, once-daily, oral neurokinin-1,3 receptor antagonist for the treatment of vasomotor symptoms (hot flashes and night sweats) during menopause, planned to start Phase III development in 2021.