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Currency in EUR
Last close As at 25/03/2023
EUR2.15
▲ −0.04 (−1.83%)
Market capitalisation
EUR125m
Research: Healthcare
Oryzon Genomics has nominated a new central nervous system (CNS) clinical development candidate, ORY-4001, for the treatment of rare CNS disorders including Charcot-Marie-Tooth (CMT) disease and amyotrophic lateral sclerosis (ALS). The decision follows positive data in which ORY 4001 demonstrated both an encouraging selectivity and safety profile as well as efficacy signals, triggering a strong anti-inflammatory response in preclinical in vivo models; the compound also showed positive responses in a validated CMT1A peripheral neuropathy in vivo model, which reliably recapitulates many of the symptoms of this condition in humans. In July 2022, Oryzon collaborated with the CMT Research Foundation, which helped fund the preclinical studies for ORY-4001 in CMT and, in our view, highlights the positive impact Oryzon’s partnerships can have on expediting development. The company now intends to start investigational new drug enabling studies as it progresses ORY-4001 towards clinical trials, the initiation of which would represent a future catalyst for investor attention.
Oryzon Genomics |
New clinical candidate expands CNS portfolio |
Clinical update |
Pharma and biotech |
14 March 2023 |
Share price performance
Business description
Analysts
Oryzon Genomics is a research client of Edison Investment Research Limited |
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Oryzon Genomics has nominated a new central nervous system (CNS) clinical development candidate, ORY-4001, for the treatment of rare CNS disorders including Charcot-Marie-Tooth (CMT) disease and amyotrophic lateral sclerosis (ALS). The decision follows positive data in which ORY4001 demonstrated both an encouraging selectivity and safety profile as well as efficacy signals, triggering a strong anti-inflammatory response in preclinical in vivo models; the compound also showed positive responses in a validated CMT1A peripheral neuropathy in vivo model, which reliably recapitulates many of the symptoms of this condition in humans. In July 2022, Oryzon collaborated with the CMT Research Foundation, which helped fund the preclinical studies for ORY-4001 in CMT and, in our view, highlights the positive impact Oryzon’s partnerships can have on expediting development. The company now intends to start investigational new drug enabling studies as it progresses ORY-4001 towards clinical trials, the initiation of which would represent a future catalyst for investor attention.
Year end |
Revenue |
PBT* |
EPS* |
DPS |
P/E |
Yield |
12/21 |
10.6 |
(7.2) |
(0.09) |
0.0 |
N/A |
N/A |
12/22 |
15.7 |
(6.6) |
(0.07) |
0.0 |
N/A |
N/A |
12/23e |
17.3 |
(4.2) |
(0.03) |
0.0 |
N/A |
N/A |
12/24e |
19.0 |
(10.0) |
(0.14) |
0.0 |
N/A |
N/A |
Note: *PBT and EPS are normalised, excluding amortisation of acquired intangibles, other income and exceptional items.
CMT is a debilitating, progressive and chronic orphan disease that damages the peripheral nerves in the brain, leading to muscular degeneration and potentially severe mobility issues. CMT affects around one in 2,500 people, which includes approximately 150,000 patients in the United States and around three million people globally. With no therapies approved for the treatment of CMT to date, the disease represents a significant area of unmet need. ALS is another rare and life-threatening CNS indication with no approved treatments. Patients diagnosed with ALS are often met with extremely poor prognosis with median survival from disease onset to death ranging from 20 to 48 months.
While Oryzon’s existing clinical candidates, iadademstat and vafidemstat, target the epigenetic modulator lysine-specific demethylase 1, ORY-4001 is a histone deacetylase 6 inhibitor and represents the company’s expansion into new epigenetic targets. The positive preclinical results, to date, provide encouraging signs for ORY-4001 and, should the drug progress into clinical studies, it would become Oryzon’s second epigenetic programme in CNS indications.
Additionally, investigations into orphan diseases such as CMT and ALS may provide future benefits for the company such as market exclusivity on regulatory approval (if received), exemption of FDA application fees and tax credits for qualified clinical trials.
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Research: Investment Companies
Gresham House Energy Storage Fund (GRID) is the largest UK fund investing in utility-scale battery energy storage systems in Great Britain and Ireland. Yesterday’s update on performance for the financial year ending 31 December 2022 and the outlook for 2023 includes news of an NAV increase in Q422, with further increases forecast in Q123 and beyond, a 5% increase in the 2023 dividend and plans to significantly expand GRID’s project pipeline across Great Britain, Ireland, the US, the European Union and Australia. These developments are all consistent with projections made in our recent update note (see link below) and suggest 2023 will be a year of both income and capital growth for GRID. They also bode positively for GRID’s efforts to capitalise on the global opportunities available in the battery energy storage sector over the longer term.
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