Welcome to your weekly roundup of EP Vantage’s snippets – short takes on smaller news items.
This week, 11-15 July, we had thought on the following events: A2A inhibitors – not just for Parkinson’s disease any more; a new immuno-oncology focus has emerged; Santhera joins its Duchenne rivals in US purgatory; Pharming goes up against Shire in HAE battle; Coherus rises sharply on biosimilar hopes; next round of sarcoma events after CytRx failure; Diurnal on track for 2018 European launch; will Ono be the boost Celyad needs as Juno setback raises concerns over CD19 CAR-T?
A2A inhibitors – not just for Parkinson’s disease any more. A new immuno-oncology focus has emerged
15 July 2016
Juno Therapeutics’ decision to acquire RedoxTherapies for $10m up front to secure rights to the adenosine A2A receptor antagonist vipadenant has probably raised a few eyebrows given that this class has struggled to make headway in central nervous system disease. However, the company is looking at the project as a potential modulator of immune suppression in cancer patients in the hopes of augmenting the tumour-fighting effects of its CAR-T candidates. Although most of the research in A2A has been in Parkinson’s disease, a small immuno-oncology pipeline is now emerging, led by Corvus Pharmaceuticals’ CPI-444, which is being tested in phase I as a monotherapy and with Roche’s PD-L1 inhibitor Tecentriq. Most recently, AstraZeneca dosed the first patient in a phase I trial of HTL1071, from Sosei’s Heptares Therapeutics subsidiary, as a monotherapy and in combination with its PD-L1 durvalumab.
Santhera joins its Duchenne rivals in US purgatory
14 July 2016
The US FDA has made it abundantly clear that it won’t cut corners in approving Duchenne muscular dystrophy drugs, and today Santhera became the latest victim of this tough stance. Its stock is trading down 40% after the agency said it wanted to see results of the Sideros trial before approving Raxone, and not on a confirmatory basis afterwards, as Santhera had hoped. This came after a refuse-to-file letter on PTC Therapeutics’ Translarna, complete response letter for Biomarin’s Kyndrisa and negative adcom vote on Sarepta’s eteplirsen. With Sideros results not due until late 2019 it is possible – though not at all certain – that Raxone’s delay until at least 2020 might give Santhera’s rivals an advantage.
Pharming goes up against Shire in HAE battle
14 July 2016
Pharming faces the task of marketing its hereditary angioedema drug Ruconest alone in 21 additional countries after scaling back its partnership with Sobi. The Dutch group will be up against fierce competition, with six other drugs already approved for HAE and several more in development – including Shire’s lanadelumab, forecast to be the top seller by 2022. Ruconest, meanwhile, is forecast to sell $10m. In the US, Pharming can still count Valeant as a partner, through its acquisition of Salix, but Ruconest seems unlikely to make its list of priorities.
Coherus rises sharply on biosimilar hopes
12 July 2016
Usually reporting phase I pharmacokinetic and pharmacodynamic results for a product is a non-event. But judging by the 33% jump in Coherus BioScience shares its investors are betting that the company will succeed in carving out decent US sales for CHS-1701, its biosimilar version of Amgen’s Neulasta. This hope could be based on speculation that the relative simplicity of Neulasta might limit clinical requirements for biosimilar approval to just pharmacokinetics, pharmacodynamics and immunogenicity. Even if the bar is set higher Coherus has at least cleared one hurdle, meaning that there could be upgrades to current CHS-1701 forecasts. But carving out market share will not be easy as there are several other players going after the $4.72bn in sales Neulasta notched up last year. Bernstein analysts see up to seven companies poised to enter the US and EU markets by the end of 2017.
Next round of sarcoma events after CytRx failure
12 July 2016
CytRx shares are expected to plummet during in-market trading today after the California-based group said its “better chemotherapy” aldoxorubicin failed to improve survival in soft tissue sarcoma (STS) patients above that seen with standard treatments in a second-line setting. Novartis’s Votrient is the biggest seller in STS, and is used in the relapsed/refractory setting after treatment with an anthracycline-based first-line treatment, and PharmaMar’s Yondelis is used in the same setting in the EU. The sector is awaiting news on an FDA decision on Lilly’s olaratumab, which could alter the STS treatment landscape if it receives approval in a first-line setting. The following candidates could have data in coming months.
Diurnal on track for 2018 European launch
11 July 2016
In publishing positive phase three data for its paediatric adrenal insufficiency product, Infacort, Diurnal moves one step closer to launching the treatment for congenital adrenal hyperplasia (CAH) in Europe by 2018. If this regulatory hurdle is cleared preparations will begin for the EU launch of the orphan drug Chronocort, for adult CAH sufferers. Phase III results are expected by early 2018, and if positive could see a 2018 European launch. Analysts at Numis expect Diurnal shares eventually to hit 550-600p, a big jump from 144p when the group floated on London’s Aim last December, raising £30m ($44.7m). But, as Numis points out, this will depend on Chronocort unseating the current standard of care. As Diurnal is planning on launching alone, the next announcement is likely to be a capital raise to fund its ambitions.
Will Ono be the boost Celyad needs as Juno setback raises concerns over CD19 CAR-T?
11 July 2016
Celyad announced a licensing deal today with Ono Pharmaceutical for its tumour-targeting adaptive cell therapy using natural killer cell receptors. Celyad has dosed its autologous cell therapy in 10 patients at four different dose levels without reporting any adverse event signals, but trails more advanced projects from Novartis, Juno Therapeutics and Kite Pharma. A setback for Juno’s CD19-directed CAR-T announced last week could allow Celyad some time to catch up as the first wave of adoptive cell therapy projects take aim at the same target.