In a deal worth $2.6bn, Pfizer is to acquire Canadian immuno-oncology company Trillium. Under the terms of the deal, Pfizer will acquire all outstanding shares of Trillium and its investigational immuno-therapeutics for haematological malignancies.

Trillium’s portfolio includes biologics that are designed to enhance the ability of patients’ innate immune systems to detect and destroy cancer cells. Its two lead molecules, TTI-622 and TTI-621, are currently in phase 1b/2 development across several indications, with a focus on haematological malignancies.

The acquisition follows an investment of $25m in Trillium, made by Pfizer in January through the ‘Pfizer Breakthrough Growth Initiative’ (PBGI), a funding programme created to invest a total of $500m across a number of clinical-stage biotech companies.

Jeff Settleman, senior vice president and chief scientific officer of Pfizer’s oncology research & development group, was also appointed to Trillium’s Scientific Advisory Board.

The move follows Pfizer’s deal with Arvinas in July to jointly develop and commercialise ARV-471, an investigational oral protein degrader for breast cancer. Pfizer agreed to pay Arvinas $650m upfront and make a $350m equity investment in the company.

Arvinas will be eligible to receive up to $400m in approval milestones and up to $1bn in commercial milestones relating to ARV-471, and will also share in the drug’s worldwide profits.

US-based biotech, Vertex, has signed a deal worth up to $1.2bn to use Arbor Biotechnologies’ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.

The deal builds on an earlier collaboration with the company that started in 2018 and will see Vertex receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies.

Vertex hopes this will give it extra momentum in two key areas: the treatment of type 1 diabetes, where it hopes to generate fully differentiated, insulin-producing hypo-immune islet cells, and sickle cell disease and beta thalassemia.

“This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases,” said Vertex cell and genetic therapies head, Bastiano Sanna.
He added that Arbor’s technology combined with Vertex’s ongoing programmes and capabilities in diabetes, haemoglobinopathies and other diseases would lead to improved cell replacement therapies for “broad populations of patients”.

Under the agreement, Arbor will receive an upfront cash payment as well as up to $1.2bn in research, development, regulatory and commercial milestone payments, and tiered royalties on future net sales. Vertex will also make an investment in Arbor in the form of a convertible note.

Vertex’s existing portfolio of treatment for cystic fibrosis (CF) saw its Q2 revenues reach $1.79bn, up by 18% on the same period in 2020, with 2021 revenues expected to reach $7.4bn.

Only three weeks after gaining approval in Europe for its gene therapy treatment Lenti-D (elivaldogene autotemcel or eli-cel), bluebird bio announced it will “scale back operations in Europe to focus on the US market”.

The announcement comes after its trial in the rare neurological condition cerebral adrenoleukodystrophy (CALD) was halted after a boy was diagnosed with myelodysplastic syndrome, a condition that can develop into leukaemia.

The company said that current evidence suggested that “specific design features” of its therapy “contributed to this event”. Two other patients are also being monitored, and the FDA has placed the eli-cel programme on clinical hold.

This is not the first such incident for bluebird bio’s gene therapies. Earlier this year, two clinical studies in sickle cell disease were put on hold after a patient developed leukaemia and another was diagnosed with myelodysplastic syndrome. The trials were later resumed when the case of myelodysplastic syndrome was assessed to be a misdiagnosis and the cancer case was “very unlikely” to be linked to bluebird bio’s therapy.

However, this latest setback for the company will inevitably raise questions about the future of gene therapy. Shares in bluebird bio dropped 25.9% on the latest news, meaning its stock has now fallen 57.4% so far this year.

The company will continue its planned split into two independent, publicly traded companies – bluebird bio and 2seventy bio – by the end of 2021

For nine years Pfizer has argued that its decision to de-brand its epilepsy drug Epanutin (phenytoin) was made in the best interests of patients.

However, after the decision resulted in a 2,600% price increase that cost the NHS £50m in 2013 alone, the UK’s Competition and Markets Authority (CMA) took another view.

In 2016, a CMA investigation found that Pfizer and its UK partner Flynn Pharma, broke competition law by charging “unfairly high” prices for phenytoin, which is used by an estimated 48,000 epilepsy patients in the UK to prevent and control seizures.

After the CMA issued a fine of £84.2m, Pfizer’s objections and several years of legal wrangling led the UK watchdog to re-open its investigation this year.

According to the CMA’s provisional view: “As Pfizer and Flynn were the dominant suppliers of the drug in the UK, the NHS had no choice but to pay unfairly high prices for this vital medicine.”

NHS spending on phenytoin sodium capsules rose “overnight” from around £2m a year in 2012 to about £50m in 2013, said the CMA. “For over four years, Pfizer’s prices were between 780% and 1,600% higher than it had previously charged. Pfizer then supplied the drug to Flynn, which sold it to wholesalers and pharmacies at prices between 2,300% and 2,600% higher than those they had paid previously.”

Pfizer and Flynn now have an opportunity to respond to the provisional findings, which the CMA will “carefully consider” to decide whether they broke the law.

The US Food and Drug Administration has accepted a supplemental biologics licence application for Bristol Myers Squibb’s (BMS) Orencia (abatacept) for the prevention of moderate-to-severe acute graft versus host disease (aGvHD) in patients receiving a stem cell transplant from an unrelated donor.

Orencia is an immunomodulator that disrupts the continuous cycle of T-cell activation. It is already indicated in the US for rheumatoid and psoriatic arthritis in adults and polyarticular juvenile idiopathic arthritis.

“For patients who receive unrelated donor stem cell transplants, in particular for racial and ethnic minority patient populations, there is a heightened risk of developing aGvHD, a potentially life-threatening medical complication for which there are no approved preventive therapies,” said Mary Beth Harler, head of immunology and fibrosis development at BMS. “We look forward to working with the FDA to bring Orencia to this new patient population and employ pathbreaking science in an effort to address unmet needs of underserved patients.”

In GvHD, transplanted donor T-cells are triggered by antigenic differences between donor and host causing them to attack the recipient’s healthy tissue and organs. The activated T-cells commonly target the host’s skin, liver and gastrointestinal tract, where damage has been associated with increased morbidity and death.

The acute disease (aGvHD) impacts 30-70% of patients, with racial and ethnic minority patients “more likely to experience challenges” following a hematopoietic stem cell transplantation, said BMS.