Upcoming events – Reneuron’s conference data and Provention takes on Crohn’s

Reneuron is set to release further data with its hRPC stem cell therapy in retinitis pigmentosa, while Provention Bio aims to target Crohn’s early with PRV-6527.

Welcome to your weekly roundup of approaching clinical readouts. On October 12 Reneuron will report results from 12 patients who have received its human retinal progenitor cell (hRPC) therapy in retinitis pigmentosa, an inherited, degenerative eye disease.

In the phase IIa study patients receive a single sub-retinal injection of the cells into their worst eye, while their other eye remains untreated. The latest data, which will be presented at the American Academy of Ophthalmology annual meeting, follows earlier impressive results from the first three patients.

Released in February, these three patients showed a mean 16-letter improvement on the ETDRS eye chart in the treated eyes, versus a mean one-letter worsening in the untreated eyes. The results were at two months for one patient, and 18 days for the other two. Shares rose 19% on the news (Reneuron eyes a partner with new stem cell data, February 20, 2019).

The latest results will be from 12 patients, and treatment duration is likely to range widely: Stifel analysts reckon this could be from a few weeks to 9 months. The analysts note a 25-30% response rate responders being those with a >15 letter gain – would be a "meaningful outcome".

The company have stated that in terms of duration of efficacy the aim would be a once yearly treatment due to the invasive nature of the procedure.

Reneuron plan to do a phase IIb next year with treatment in both eyes versus observational control. In terms of a primary endpoint for future studies, as guided by the FDA, a 15 letter improvement from baseline would be seen as clinically meaningful.

Data

American Academy of Ophthalmology annual meeting session

Phase IIa data from 12 patients, NCT02464436

9.25am (PST), Saturday October 12, RET15 - Section XI: Late Breaking Developments, Part II

‘Proventing’ disease

Provention Bio is due to report phase II data in Crohn’s disease in the fourth quarter. The Prince study tests twice daily PRV-6527 versus placebo for 12 weeks. The primary endpoint is change from baseline in Crohn’s disease activity index score.

The trial enrolled 93 patients with moderate to severe Crohn’s disease who were either naïve to biologic therapy or had previously failed at least one biologic drug.

Crohn’s disease is characterised by recurring episodes of inflammation and ulceration in the gastrointestinal tract and Provention is aiming to intervene before the disease progresses into a full blown, chronic condition. PRV-6527 is an oral, colony stimulating factor 1 (CSF-1) receptor inhibitor that is said to work by intercepting inflammatory dendritic cells and macrophages, preventing their movement into the gut. 

In 2017 Provention in-licensed the worldwide rights for PRV-6527 from Johnson & Johnson for an undisclosed amount. J&J had tested the agent in rheumatoid arthritis but it showed limited signs of efficacy, with safety signals including elevations in liver enzymes.

The pharma giant has the right to re-acquire the drug for $50 million, with Provention eligible to receive royalties on IBD-related revenue. Analysts believe this re-acquisition could happen next year, although presumably these phase II results would have to be very strong for that to happen. Provention has $41.3m in cash according to its second quarter call. 

The Crohn’s market is currently dominated by monoclonal antibodies such as Abbvie’s anti-TNF mAb Humira. Despite the threat of biosimilars, Humira’s stronghold on Crohn’s disease is set to continue, according to consensus from EvaluatePharma, which sees the product’s 2024 sales reaching $3.7bn. The other top four spots are all taken by MAbs, and although PRV-6527 would represent a first oral entrant, this would be a tough market to crack (see table).

Biggest selling Crohn's disease treatments in 2024

     

WW annual indication sales ($bn)

Product

Company

Pharma class

2019e

2024e

Humira

AbbVie, Eisai

Anti-TNFa MAb

4.8

3.7

Entyvio

Takeda

Anti-alpha 4 beta 7 integrin MAb

1.5

2.8

Stelara

Johnson & Johnson, Mitsubishi Tanabe Pharma

Anti-IL-12 & IL-23 MAb

2.3

2.5

Remicade

Johnson & Johnson, Merck & Co, Mitsubishi Tanabe Pharma

Anti-TNFa MAb

2.7

1.6

Skyrizi

AbbVie

Anti-IL-23 MAb

-

0.4

Source: EvaluatePharma

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