Journal of Inherited Metabolic Disease

Novel Genetic Screening Tool Offers Hope for Babies Born With Life-Threatening Metabolic Disorder

Retrieved on: 
Thursday, May 4, 2023

SEATTLE, May 4, 2023 /PRNewswire-PRWeb/ -- Every expectant parent hopes to welcome a healthy baby into the world. Unfortunately, infants with some genetic diseases appear healthy at birth, but then rapidly deteriorate—they become lethargic, stop eating, develop seizures, and may progress to coma—symptoms common to a variety of metabolic disorders. For the physicians charged with their care, it is a race against time to determine which disorder is causing their fragile patient to decline.

Key Points: 
  • For the physicians charged with their care, it is a race against time to determine which disorder is causing their fragile patient to decline.
  • A study published today in the American Journal of Human Genetics describes the tools that the Dudley Lab developed to assess the changes in a genetic sequence (variant) associated with ornithine transcarbamylase deficiency ( OTC deficiency ), a rare, inherited metabolic disorder.
  • "We hope that our data will also accelerate the development of new treatments and cures for OTC deficiency."
  • The technology built by the Dudley Lab at PNRI offers hope for a brighter future for infants with life-threatening metabolic diseases.

CENTOGENE to Participate in Upcoming Conferences in March

Retrieved on: 
Wednesday, March 8, 2023

CENTOGENE representatives will be attending events in Brazil, the U.S., Saudi Arabia, Thailand, and the U.K. and will be available to discuss Pharma, CRO, and Diagnostic collaboration opportunities.

Key Points: 
  • CENTOGENE representatives will be attending events in Brazil, the U.S., Saudi Arabia, Thailand, and the U.K. and will be available to discuss Pharma, CRO, and Diagnostic collaboration opportunities.
  • Please see additional details below:
    To learn more about the 3rd Brazilian Congress of Neurogenetics 2023 and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( https://link.centogene.com/3KK0jgi ).
  • To learn more about the 6th Asian Congress on Inherited Metabolic Diseases 2023 and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( https://link.centogene.com/3ZpRrRe ).
  • To learn more about the European Laboratory Research & Innovation Group’s (ELRIG) Research & Innovation 2023 conference and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( https://link.centogene.com/3YKdpOh ).

Poxel Reports Financial Results for First Half 2022 and Provides a Corporate Update

Retrieved on: 
Wednesday, September 21, 2022

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its financial results for the period ended June 30, 2022 and provided a corporate update.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its financial results for the period ended June 30, 2022 and provided a corporate update.
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: Thus far, 2022 has been marked by important achievements for Poxel.
  • Over the summer, we also extended our cash runway through the restructuring of our debt and an equity-linked financing facility.
  • This accomplishment provides us further flexibility to secure additional financing solutions necessary to execute our rare disease strategy.

Poxel Provides Corporate Update and Reports Cash and Revenue for the Second Quarter and First Half 2022

Retrieved on: 
Monday, August 8, 2022

POXEL SA (Euronext : POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today provided a corporate update and announced its cash position and revenue for the second quarter and first half of 2022.

Key Points: 
  • POXEL SA (Euronext : POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today provided a corporate update and announced its cash position and revenue for the second quarter and first half of 2022.
  • "The next major milestone for Poxel will be the results of our Phase 2 DESTINY-1 study for PXL065 in NASH, which are expected later this quarter.
  • As of June 30, 2022, royalty revenue to Poxel based on TWYMEEG net sales in Japan under the Sumitomo Pharma license agreement has been limited following TWYMEEGs commercial launch on September 16, 2021.
  • As of June 30, 2022, cash and cash equivalents were EUR 16.1 million (USD 16.8 million), as compared to EUR 32.3 million (USD 36.6 million) as of December 31, 2021.

Poxel Announces the Publication of Two Preclinical Articles on X-Linked Adrenoleukodystrophy for PXL065 and PXL770

Retrieved on: 
Tuesday, July 5, 2022

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announces the publication of two preclinical articles on X-Linked Adrenoleukodystrophy (ALD) for PXL065 and PXL770.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announces the publication of two preclinical articles on X-Linked Adrenoleukodystrophy (ALD) for PXL065 and PXL770.
  • An article on PXL065 has been published in The Journal of Inherited Metabolic Disease (JIMD) and is entitled Therapeutic potential of deuterium-stabilized (R)-pioglitazone - PXL065 - for X-linked adrenoleukodystrophy.
  • These publications describe similar beneficial preclinical profiles of PXL770 and PXL065 in models of ALD.
  • Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

CytRx Comments on Quarterly Results and Recent Strategic Initiatives

Retrieved on: 
Friday, November 12, 2021

CytRx concluded the quarter ended September 30, 2021 with cash and cash equivalents of approximately $16.5 million.

Key Points: 
  • CytRx concluded the quarter ended September 30, 2021 with cash and cash equivalents of approximately $16.5 million.
  • General and administrative expenses were $1.5 million for the quarter, compared with $2.2 million for the same period in 2020.
  • All forward-looking statements are based upon information available to CytRx on the date the statements are first published.
  • CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Orphazyme reports business highlights and financial results in Interim Report First Half 2021

Retrieved on: 
Tuesday, August 31, 2021

Copenhagen,Denmark,August 31,2021 Orphazyme A/S (ORPHA.CO; ORPH) (the Company), a late-stage biopharmaceutical company, today announces its Interim Report First Half 2021 for the period January 1 June 30, 2021.

Key Points: 
  • Copenhagen,Denmark,August 31,2021 Orphazyme A/S (ORPHA.CO; ORPH) (the Company), a late-stage biopharmaceutical company, today announces its Interim Report First Half 2021 for the period January 1 June 30, 2021.
  • We anticipate reaching net revenues of between DKK 30 and DKK 40 million by year-end December 31, 2021.
  • Orphazyme will host an investor call during which Management will present the Interim Report First Half 2021.
  • The call will be held on Tuesday, August 31, 2021 at 2.00 PM CEST/8.00 AM EDT.

CytRx Highlights Orphazyme’s Published Results From Its Phase 2/3 Trial of Arimoclomol in Niemann-Pick Disease Type C

Retrieved on: 
Tuesday, August 24, 2021

Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:

Key Points: 
  • Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:
    "CytRx is encouraged by Orphazymes published results of its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C, which exhibited a statistically significant and clinically meaningful treatment effect on reducing disease progression.
  • Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease.
  • 2 Patterson MC, Lloyd-Price L, Guldberg C, et al (2021) Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale.
  • 4 Patterson MC, Lloyd-Price L, Guldberg C, et al (2021) Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale.