Dona Ferentes
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NEU - Neuren Pharmaceuticals
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Dona Ferentes
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And there has been solid buying in the last month, since FDA approval.
More traction with yesterday's Ann.
NEU reported that its North America partner Acadia Pharmaceuticals (NASDAQ: ACAD) has announced that DAYBUE™ (trofinetide) is now available for the treatment of Rett syndrome in adult and pediatric patients two years of age and older in the United States. DAYBUE was approved by the US Food and Drug Administration (FDA) on 10 March 2023 and is the first and only drug approved by the FDA for the treatment of Rett syndrome.
....longer term, this has been textbook, for progression of their drug, through the necessary steps to commercialisation:
More traction with yesterday's Ann.
NEU reported that its North America partner Acadia Pharmaceuticals (NASDAQ: ACAD) has announced that DAYBUE™ (trofinetide) is now available for the treatment of Rett syndrome in adult and pediatric patients two years of age and older in the United States. DAYBUE was approved by the US Food and Drug Administration (FDA) on 10 March 2023 and is the first and only drug approved by the FDA for the treatment of Rett syndrome.
....longer term, this has been textbook, for progression of their drug, through the necessary steps to commercialisation:
Dona Ferentes
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Neuren and Acadia expand global partnership for trofinetide (DAYBUE™)
Highlights:
• Acadia’s exclusive licence for trofinetide in North America expanded to worldwide licence
• Neuren to receive US$100 million up-front, plus additional potential milestone payments of up to US$427 million, plus royalties
• Existing milestone payments and royalties for trofinetide in North America unchanged
• Expanded partnership leverages Acadia’s unique knowledge and expertise from the successful development and commercialisation of trofinetide (marketed in the US as DAYBUE™) in the United States
• Update on DAYBUE launch in the US - Acadia expects net sales of $21-23 million in Q2 2023 and $45-55 million in Q3 2023
• Exclusive worldwide licence granted to Acadia for NNZ-2591 solely in Rett and Fragile X - enables coordinated global development and removes existing restrictions on Neuren for NNZ-2591 in those two indications. Milestone payments and royalties to Neuren for NNZ-2591 in Rett and Fragile X identical to trofinetide milestone payments and royalties.
• Neuren retains worldwide rights to NNZ-2591 in all other indications and is currently advancing Phase 2 trials in each of Phelan-McDermid, Pitt Hopkins, Angelman and Prader-Willi syndromes, with first results expected in December 2023
Dona Ferentes
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still hovering, though the news seems better with every week.
Neuren’s drug – branded Daybue – is taken in liquid form twice a day by children to treat Rett syndrome, which is a genetic mutation from birth.
It’s only been available for prescription in the US for about 12 weeks, but Neuren’s chief executive, John Pilcher, said doctors and families were already reporting patients’ cognitive function had improved in areas like walking, talking, sleeping, and hand movements, as the drug improves signalling between brain cells.
Neuren’s US distribution partner, Acadia, forecasts Daybue sales there will climb strongly and royalty payments from Acadia will help it swing from almost zero revenue over the first half of this year to a profit of $134.1 million in the second half and accumulated profits of $274.7 million by the 2025 financial year.
some analysts are tipping $20+ (but they would)
Neuren’s drug – branded Daybue – is taken in liquid form twice a day by children to treat Rett syndrome, which is a genetic mutation from birth.
It’s only been available for prescription in the US for about 12 weeks, but Neuren’s chief executive, John Pilcher, said doctors and families were already reporting patients’ cognitive function had improved in areas like walking, talking, sleeping, and hand movements, as the drug improves signalling between brain cells.
Neuren’s US distribution partner, Acadia, forecasts Daybue sales there will climb strongly and royalty payments from Acadia will help it swing from almost zero revenue over the first half of this year to a profit of $134.1 million in the second half and accumulated profits of $274.7 million by the 2025 financial year.
some analysts are tipping $20+ (but they would)
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Well there's the $20 level plus some, up 28.88% today. A nice start to the week and a couple of announcements below.
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Dona Ferentes
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solid second day after the TH and announcement , putting on another 8 per cent today.
.
Phase 2 trial shows significant improvements in Phelan-McDermid syndrome
Highlights:
• Significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures
• Improvements were consistently seen across clinically important aspects of Phelan-McDermid syndrome, including communication, behaviour, cognition/learning and socialisation
• Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful:
o Clinical Global Impression of Improvement (CGI-I) - mean score of 2.4, with 16 out of 18 children showing improvement assessed by clinicians
o Caregiver Overall Impression of Change – mean score of 2.7, with 15 out of 18 children showing improvement assessed by caregivers
• For 10 out of 14 efficacy endpoints, improvement from baseline on overall/total scores was statistically significant (Wilcoxon signed rank test p<0.05)
• NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values or other safety parameters during treatment
.
Phase 2 trial shows significant improvements in Phelan-McDermid syndrome
Highlights:
• Significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures
• Improvements were consistently seen across clinically important aspects of Phelan-McDermid syndrome, including communication, behaviour, cognition/learning and socialisation
• Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful:
o Clinical Global Impression of Improvement (CGI-I) - mean score of 2.4, with 16 out of 18 children showing improvement assessed by clinicians
o Caregiver Overall Impression of Change – mean score of 2.7, with 15 out of 18 children showing improvement assessed by caregivers
• For 10 out of 14 efficacy endpoints, improvement from baseline on overall/total scores was statistically significant (Wilcoxon signed rank test p<0.05)
• NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values or other safety parameters during treatment
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NEU's distributor of DayBlue, ACAD(US) is under attack by a short seller.
From Reuters via TradingView.
Trading halted one hour after opening and NEU's price sold down hard to $20 (-$3).
From Reuters via TradingView.
Trading halted one hour after opening and NEU's price sold down hard to $20 (-$3).
Dona Ferentes
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"Neuren tumbled 14.2 per cent to $19.78 after a US short seller issued a report attacking the data behind its Daybue drug to treat Rett syndrome in children."
... & despite posting on it, I've never jumped on-board.
... & despite posting on it, I've never jumped on-board.
Dona Ferentes
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pushing 15 per cent higher today
Phase 2 trial shows significant improvements in Pitt Hopkins syndrome
Highlights:
• Statistically significant improvement from baseline assessed by both clinicians and caregivers in all four efficacy measures specifically designed for Pitt Hopkins syndrome (Wilcoxon signed rank test p<0.05)
• Clinician and caregiver global efficacy measures showed a level of improvement considered clinically meaningful:
o Clinical Global Impression of Improvement (CGI-I) - mean score of 2.6, with 9 out of 11 children showing improvement assessed by clinicians
o Caregiver Overall Impression of Change – mean score of 3.0, with 8 out of 11 children showing improvement assessed by caregivers
• Improvements were seen in clinically important aspects of Pitt Hopkins syndrome, including communication, social interaction, cognition and motor abilities
• NNZ-2591 was safe and well tolerated, with no serious or severe adverse events and no meaningful trends in laboratory values or other safety parameters during treatment
• Second positive Phase 2 trial result further strengthens confidence in NNZ-2591’s potential relevance for multiple neurodevelopmental disorders
Phase 2 trial shows significant improvements in Pitt Hopkins syndrome
Highlights:
• Statistically significant improvement from baseline assessed by both clinicians and caregivers in all four efficacy measures specifically designed for Pitt Hopkins syndrome (Wilcoxon signed rank test p<0.05)
• Clinician and caregiver global efficacy measures showed a level of improvement considered clinically meaningful:
o Clinical Global Impression of Improvement (CGI-I) - mean score of 2.6, with 9 out of 11 children showing improvement assessed by clinicians
o Caregiver Overall Impression of Change – mean score of 3.0, with 8 out of 11 children showing improvement assessed by caregivers
• Improvements were seen in clinically important aspects of Pitt Hopkins syndrome, including communication, social interaction, cognition and motor abilities
• NNZ-2591 was safe and well tolerated, with no serious or severe adverse events and no meaningful trends in laboratory values or other safety parameters during treatment
• Second positive Phase 2 trial result further strengthens confidence in NNZ-2591’s potential relevance for multiple neurodevelopmental disorders
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NEU has turned the corner and cash rich now.
FDA signal and Phase 3 - in next 6 months it is all blue sky (DYOR) .
Some relevant announcements are attached to support the above.
FDA signal and Phase 3 - in next 6 months it is all blue sky (DYOR) .
Some relevant announcements are attached to support the above.
Dona Ferentes
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here are some words sumarising one of the Announcements 
Trading 5% higher at $12.90.
MORE NEUREN PHARMACEUTICALS LIMITED CONTENT
Neuren Pharmaceuticals (ASX:NEU) is set to receive a substantial payout following the sale of a Rare Pediatric Disease Priority Review Voucher by its US-based partner, Acadia Pharmaceuticals (NASDAQ: ACAD), for $US150m. Under the agreement, Neuren will receive one-third of the net proceeds, estimated to be approximately $US50m.
The PRV was issued by the US Food and Drug Administration following its approval of DAYBUE (trofinetide), a treatment for Rett syndrome in patients aged two years and older. PRVs are awarded as part of the FDA's Rare Pediatric Disease Program, an initiative aimed at encouraging the development of therapies for severe and life-threatening pediatric diseases with limited treatment options. These vouchers allow companies to receive an expedited review for a future drug, reducing the FDA's standard review time from ten months to six months, potentially accelerating a drug’s market entry. Companies that receive a PRV may apply it to their own drug pipeline or sell it to other companies seeking faster FDA review.
The voucher awarded for DAYBUE reflects its impact on treating Rett syndrome, a rare neurological disorder that almost exclusively affects females. Rett syndrome typically manifests in early childhood and is characterised by a period of normal development followed by a gradual loss of motor and cognitive skills. Symptoms include difficulty with movement, loss of speech, repetitive hand movements and breathing irregularities. Most individuals with Rett syndrome require lifelong care, and there is no known cure. Trofinetide, now marketed as DAYBUE, addresses some of the disorder’s symptoms by targeting underlying inflammatory and neurodevelopmental processes.
Neuren’s licensing agreement with Acadia grants Acadia the global rights to develop and commercialise DAYBUE.
Neuren’s CEO, Jon Pilcher, highlighted the partnership’s success, emphasising that the proceeds from the PRV sale will enable continued investment in Neuren’s neurodevelopmental pipeline.
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Trading 5% higher at $12.90.Neuren receives U$50m boost from FDA voucher sale
By Finance News Network |MORE NEUREN PHARMACEUTICALS LIMITED CONTENT
Neuren Pharmaceuticals (ASX:NEU) is set to receive a substantial payout following the sale of a Rare Pediatric Disease Priority Review Voucher by its US-based partner, Acadia Pharmaceuticals (NASDAQ: ACAD), for $US150m. Under the agreement, Neuren will receive one-third of the net proceeds, estimated to be approximately $US50m.
The PRV was issued by the US Food and Drug Administration following its approval of DAYBUE (trofinetide), a treatment for Rett syndrome in patients aged two years and older. PRVs are awarded as part of the FDA's Rare Pediatric Disease Program, an initiative aimed at encouraging the development of therapies for severe and life-threatening pediatric diseases with limited treatment options. These vouchers allow companies to receive an expedited review for a future drug, reducing the FDA's standard review time from ten months to six months, potentially accelerating a drug’s market entry. Companies that receive a PRV may apply it to their own drug pipeline or sell it to other companies seeking faster FDA review.
The voucher awarded for DAYBUE reflects its impact on treating Rett syndrome, a rare neurological disorder that almost exclusively affects females. Rett syndrome typically manifests in early childhood and is characterised by a period of normal development followed by a gradual loss of motor and cognitive skills. Symptoms include difficulty with movement, loss of speech, repetitive hand movements and breathing irregularities. Most individuals with Rett syndrome require lifelong care, and there is no known cure. Trofinetide, now marketed as DAYBUE, addresses some of the disorder’s symptoms by targeting underlying inflammatory and neurodevelopmental processes.
Neuren’s licensing agreement with Acadia grants Acadia the global rights to develop and commercialise DAYBUE.
Neuren’s CEO, Jon Pilcher, highlighted the partnership’s success, emphasising that the proceeds from the PRV sale will enable continued investment in Neuren’s neurodevelopmental pipeline.
nh,nh
