In 2016, Roche signed an initial deal with Blueprint worth a potential $1bn, which included $45m upfront along with a further $965m in possible option fees and milestone payments, plus royalties, dependent on the clinical development and regulatory approval of five small-molecule candidates.

In 2016, Roche signed an initial deal with Blueprint worth a potential $1bn, which included $45m upfront along with a further $965m in possible option fees and milestone payments, plus royalties, dependent on the clinical development and regulatory approval of five small-molecule candidates.
At that point, Blueprint was still in the preclinical stages of development – now, pralsetinib is poised for approval, with new drug applications already submitted to the US Food and Drug Administration (FDA) for the treatment of RET fusion-positive non-small cell lung cancer (NSCLC), RET mutation-positive medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer.

Although RET activating fusions and mutations are only observed in a small number of cancer patients – around 1-2% in NSCLC and 10-20% in papillary thyroid cancer – they are thought to be key disease drivers. By inhibiting RET, researchers have found that significant benefits can be achieved in patients with these specific mutations.

Under the terms of the deal, Blueprint and Roche’s Genentech division will co-commercialise pralsetinib in the US, while Roche will have exclusive commercialisation rights for the drug outside the US, excluding Greater China.

Blueprint will gain a substantial $775m in upfront payments, including a cash payment of $675m and an equity investment by Roche of $100m. The biotech will be eligible to receive an additional $927m in contingent payments, including certain development, regulatory and sales-based milestones for pralsetinib. Roche will also have the right to opt in to a next-generation RET compound, co-developed under the collaboration.

“With Roche’s global reach and unparalleled expertise in personalised healthcare, this collaboration will accelerate our ability to bring pralsetinib to patients with significant medical needs around the world and expand development of pralsetinib across multiple treatment settings where there is potential to benefit even broader patient populations,” said Jeff Albers, chief executive officer of Blueprint Medicines.

Blueprint’s main rival, Loxo Oncology, also has an oral RET inhibitor – Retevmo (selpercatinib) – in its arsenal that is FDA-approved in RET fusion-positive NSCLC, advanced or metastatic RET-mutant MTC and advanced or metastatic RET fusion-positive thyroid cancer.

Gilead and Arcus Biosciences announced their intent to partner on the development and commercialisation of next-generation cancer immunotherapies in May, and the agreement has now been finalised.

Under the terms of the agreement, Arcus is set to receive $375m upon closing, consisting of a $175m upfront payment and a $200m equity investment from Gilead. Arcus is eligible to receive up to $1.225bn in opt-in and milestone payments with respect to its current clinical product candidates.

In return, Gilead will gain access to Arcus’ current and future investigational immuno-oncology products, as Gilead continues to expand its presence in the field. This includes the immediate rights to zimberelimab, an investigational anti-PD-1 monoclonal antibody, as well as the right to opt in to all of Arcus’ other current clinical candidates upon payment of an opt-in fee that ranges from $200m to $275m per programme.

Arcus has ten ongoing clinical studies of molecules in its portfolio, including a randomised phase 2 study in first-line non-small cell lung cancer evaluating combinations of three of its candidates – AB154, an investigational anti-TIGIT monoclonal antibody; AB928, an investigational A2aR/A2bR antagonist, and zimberelimab.

“By gaining access to its broad, diverse pipeline and Arcus’ clear strengths in discovery and development, we believe that our partnership with Arcus will significantly accelerate our progress in developing transformative new therapies for cancer,” said Daniel O’Day, chairman and chief executive officer, Gilead Sciences.

Gilead has been expanding into immuno-oncology since the start of 2019 under the direction of new chief exec O’Day, in a bid to bulk up its pipeline amid pressure on its HIV and hepatitis franchises.

That includes a $4.9bn deal signed in March to acquire Forty Seven, a company that specialises in cancer immunotherapies targeting the CD47 immune checkpoint. Studies have suggested that blocking CD47, a cell membrane receptor found on the surface of many tumour types, could send some tumour cells into programmed cell death (apoptosis).

Gilead also signed a deal with Galapagos, worth $5.1bn, which doubled its research and development capacity and added at least six drugs to its pipeline. It also signed a $2.35bn deal with Nurix for protein degradation drugs for cancer and other diseases, as well as a $11.9bn acquisition of CAR-T cell therapy player Kite Pharma.

The International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) has raised nearly $1bn to support clinical research into new antibiotics in a bid to tackle growing antimicrobial resistance (AMR).

The AMR Action Fund was created to bring two to four new antibiotics to patients by 2030 – treatments which are urgently needed to address the rapid rise of antibiotic resistance, which is a part of the broader issue of antimicrobial resistance. The launch of this new fund is the result of a collaboration among major pharmaceutical companies represented by the IFPMA, alongside the European Investment Bank (EIB), Wellcome Trust and the World Health Organization (WHO).
According to the IFPMA, each year 700,000 people die as a result of AMR, with some analysts estimating that by 2050 as many as ten million people could lose their lives each year because of the increasing prevalence of antibiotic resistant infections.

Despite the global need for new medicines to treat antibiotic resistant infections, there has been a drought in research and development – especially among big pharmaceutical companies. For many major drugmakers, there is a profound lack of financial incentive for the development of new antibiotics, given the fact that the most effective medicines are usually kept in reserve and only used when infections do not respond to older agents.

This investment drought has led to the collapse of a number of smaller companies focused solely on the development of antibiotics, such as Melinta – a biotech company whose pipeline was filled with new antibiotic candidates and research, but had to file for bankruptcy earlier this year. It seems that the tide is beginning to turn in the development of new antibiotics, however, signalled by the creation of AMR Action Fund, with a number of major companies investing in the scheme.
That includes Pfizer, which has pledged $100m to the newly launched fund to help address the global public health need for new AMR treatments. German drug developer Boehringer Ingelheim has also contributed $50m to the AMR Action Fund to boost the development of novel treatments against the global health threat.

Biogen has submitted an application to the US Food and Drug Administration (FDA) for the approval of its controversial Alzheimer’s disease drug, after reviving development of the treatment last year.

It’s been a rocky road for Biogen’s aducanumab – following disappointing results in March last year, Biogen and partner Eisai said they were pulling the plug on the phase 3 testing on the advice of an independent data monitoring committee.

In a surprise twist, Biogen then announced plans to revive the research into the experimental therapy in October, after further analysis of promising results from a subset of patients in the phase 3 EMERGE study.

In the EMERGE trial, data showed that patients who received sufficient exposure to high doses of the drug experienced significant benefits on measures of cognition and function, including memory, orientation and language. The second phase 3 ENGAGE trial, on the other hand, didn’t show clinical efficacy for the drug, with many critics saying that the data is not strong enough to warrant FDA approval.

Although experts have found that the data is complex and hard to interpret, the fact that there are currently no approved treatments for Alzheimer’s on the market could prove to be enough to get aducanumab to the finish line, considering the FDA will be eager to approve new therapies for the mind-wasting disease.

That fact is aptly summed up by GlobalData analyst Alessio Brunello, who suggested the FDA is “unlikely to turn down aducanumab even if its benefit is modest, given the lack of any therapy that is truly efficacious”.

Now that Biogen has completed the Biologics License Application (BLA) and submitted it to the FDA, the regulatory agency has up to 60 days to decide whether to accept it for review. If accepted, Biogen said it expects the FDA will also inform it whether the application has been granted a priority review designation, which would be a positive signal for the company.

Biogen is relying heavily on the success of aducanumab, given that Mylan recently won a patent challenge allowing it to manufacture and sell a generic version of the former company’s branded multiple sclerosis treatment Tecfidera.

The company’s spinal muscular atrophy Spinraza (nusinersen) is also facing increasing competition from the likes of new therapeutics that include Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec) and Roche’s investigational therapy risdiplam.