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Research: Healthcare
Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.
Pharnext |
First patient enrolled in PREMIER extension study |
Clinical development update |
Pharma and biotech |
12 September 2022 |
Share price performance Business description
Analysts
Pharnext is a research client of Edison Investment Research Limited |
Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.
Year end |
Revenue (€m) |
PBT* |
EPS* |
DPS |
P/E |
Yield |
12/20 |
2.8 |
(21.4) |
(1.17) |
0.00 |
N/A |
N/A |
12/21 |
3.6 |
(30.6) |
(1.01) |
0.00 |
N/A |
N/A |
Note: *PBT and EPS are normalised, excluding amortisation of acquired intangibles, exceptional items and share-based payments.
The ongoing pivotal Phase III PREMIER trial is a randomised, double-blinded, placebo-controlled study evaluating the effectiveness and safety of PXT3003 in mild-to-moderate CMT1A patients, a genetic peripheral nerve disorder that causes progressive muscle weakness. The PXT3003 dosage used for the trial corresponds to the high-dose arm used in the previous Phase III PLEO-CMT trial as well as the ongoing open-label extension study (PLEO-CMT-FU). Patient enrolment to the PREMIER study was competed in May 2022, with 387 patients recruited (versus the target of 350) across 52 centres globally. The primary endpoint is improvement on the 12-point Overall Neuropathy Limitations Scale, which measures functional motor disability. The 15-month study is expected to conclude in Q423.
CMT1A is the most common type of CMT and Pharnext estimates that the disease afflicts over 150,000 people in the United States and Europe (1.5 million people worldwide), with the most severe cases (c 5% of patients) requiring wheelchairs. There are currently no approved therapies, with treatment restricted to supportive care such as orthotics, leg braces, physical and occupational therapy, followed by surgery on disease progression. If clinical development is successful, PXT3003 would be the first therapy approved for the indication and will have seven years and 10 years of market exclusivity courtesy of its orphan drug designation in the United States and Europe.
In May 2022, Pharnext presented positive data from the long-term open-label PLEO-CMT-FU study, which continued to show a sustained benefit for patients after five years of treatment. In total, 123 patients are still under treatment in this trial. We believe that similar results from the newly announced PREMIER-OLE extension study should complement the data readout from the Phase III PREMIER trial in building the regulatory case for Pharnext.
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Research: Healthcare
Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.
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