The past week has been a positive one for the sickle cell disease (SCD) community.
The FDA confirmed late last week approval of Novartis' Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in SCD patients aged 16 and over.
A second novel SCD therapy - Global Blood Therapeutics' once-daily oral treatment voxelotor - is on track to be approved early next year.
Looking further ahead, partners CRISPR Therapeutics and Vertex Pharmaceuticals announced on Tuesday that the first two patients to receive the CRISPR-based genome editing therapy CTX001 for SCD and beta thalassaemia have responded to the treatment and experienced treatable side effects. The SCD patient treated with CTX001 was free from VOCs four months after infusion, though it realistically remains too early to draw any conclusion beyond encouragement from these preliminary findings.
Two very different biotech acquisitions announced this week share one similarity - in both cases the company being acquired has been passed up by another player with the option to buy.
Roche will buy Promedior for $390 million upfront in a deal potentially worth $1.4 billion, gaining access to PRM-151; a recombinant form of human pentraxin-2 protein that has demonstrated significant lung function improvements on top of current therapies for idiopathic pulmonary fibrosis (including Roche's marketed therapy Esbriet) in Phase II studies.
Bristol-Myers Squibb had previously reached an agreement to buy Promedior for $150 million in 2015, but chose not to acquire outstanding shares in the company late last year (potentially due to its focus on acquiring Celgene, announced in January, which coincidently was confirmed this week as having been cleared by the FTC).
Elsewhere, Alkermes said it will acquire CNS-focused Rodin Therapeutics in a deal worth up to $950 million, including an upfront payment of $100 million. Biogen previously collaborated with Rodin in 2016 with an option to buy the biotech for $485 million, though it would appear the Phase I Alzheimer's disease asset that had piqued Biogen's interest will be dropped by Alkermes. Instead it says it will focus on advancing preclinical candidates in the Rodin pipeline.
Analysts at Stifel described the deal as offering "entry into an interesting emerging area of neuroscience at a reasonable price," and noting that Rodin's portfolio could be "applied to a wide array of CNS indications where synaptic loss or dysfunction is core to the pathology, including Alzheimer's, Huntington's or even psychiatric ailments like depression and schizophrenia."
Chinese biotech company BeiGene received US approval for its BTK inhibitor Brukinsa for the second-line treatment of mantle cell lymphoma (MCL) this week.
Whilst MCL is initially a small and competitive indication, the approval of Brukinsa is nevertheless an important landmark for BeiGene, coming 100 days ahead of schedule to boot. It represents the first approval of any product developed internally by BeiGene and the first clearance of a Chinese-developed innovative drug in the US.
Analysts at Credit Suisse expect Chinese approval of Brukinsa to occur next year, noting further that final clearance of BeiGene's PD-1 inhibitor tiselizumab for classical Hodgkin's lymphoma in China should occur later this month. BeiGene regained full rights for tiselizumab when a global collaboration with Celgene was terminated in advance of its pending acquisition by Bristol-Myers Squibb. Pivotal-stage data in bladder and non-small-cell lung cancer are expected to read out shortly.
Earlier this month Amgen acquired a 20.5% stake in BeiGene for approximately $2.7 billion to expand its oncology footprint in the Chinese pharmaceutical market.
A number of biotechnology companies witnessed stratospheric share price gains this week in response to under-the-radar data readouts.
Karuna Therapeutics saw its market cap quadruple on Monday in response to promising Phase II data for its experimental schizophrenia treatment KarXT. The company plans to initiate a pivotal-stage study by the end of 2020. Whether it can suitably compensate - at the very least - for placebo effect and circumvent the notoriously poor predictability of large clinical studies in psychiatric diseases remains to be seen.
Shares in Myovant Sciences jumped as much as 178% on Tuesday after the company reported that a Phase III study of relugolix in men with advanced prostate cancer met its primary efficacy endpoint and all six key secondary goals. The company said results of the HERO trial will be used to support a US marketing application for the oral GnRH receptor antagonist in the second quarter of 2020, as well as future regulatory submissions in Europe and Japan.
The FDA said Wednesday that it approved Alnylam Pharmaceuticals' Givlaari (givosiran) to treat adults with acute hepatic porphyria. Givlaari is the (and Alnylam's) second RNAi drug to be approved by the FDA, with regulatory clearance coming four months ahead of the PDUFA date.
The injectable therapy, which is dosed based on patient weight, will carry a list price of $575,000 per year, and will be available after discounts for $442,000 per year.
Alnylam said it is in talks with leading payers about value-based agreements for Givlaari and plans to incorporate a new "ultra-rare disease framework" as part of its negotiations. Under the framework, participating government and commercial payers will pay the full value for the treatment "only when it delivers patient outcomes in the real-world setting similar to results demonstrated in clinical trials," the company explained. Moreover, it said an additional prevalence-based adjustment feature will trigger rebates to participating payers "if the number of diagnosed patients they cover exceeds current epidemiologic estimates" for the condition.
In response to a Bloomberg story this week about the price of Pfizer's transthyretin amyloid cardiomyopathy (ATTR-CM) treatment Vyndaqel, the company also said it would consider lowering the price if it is used to treat a larger than expected number of patients. Sales of Vyndaqel recorded in the third quarter came in notably higher than analysts were expecting.
On a related note, Novartis is rumoured to be interested in acquiring The Medicines Company, which is near to submitting its first regulatory applications for the siRNA (small interfering RNA) cardiovascular treatment inclisiran, and Novo Nordisk has agreed to collaborate with Dicerna to develop RNA based therapies in the cardio-metabolic space. The Danish company said this week that clinical programmes such as inclisiran have suitably de-risked this approach.
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