Ultragenyx | Jotup

Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update

Retrieved on:

Thursday, May 2, 2024

Osteogenesis imperfecta, Burosumab, Patient, OI, Ageing, Ultragenyx

NOVATO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended March 31, 2024 and reaffirmed its financial guidance for 2024.

Key Points:

Total operating expenses for the first quarter of 2024 were $274 million, including non-cash stock-based compensation of $37 million.
Net cash used in operations for the quarter ended March 31, 2024 was $191 million.
For the full year 2024, the company reaffirms:
Crysvita revenue in the range of $375 million to $400 million.
ET to discuss the first quarter 2024 financial results and provide a corporate update.

Athira Pharma Appoints Javier San Martin, M.D., as Chief Medical Officer

Retrieved on:

Monday, April 15, 2024

BOTHELL, Wash., April 15, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the appointment of Javier San Martin, M.D., as Chief Medical Officer.

Key Points:

BOTHELL, Wash., April 15, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced the appointment of Javier San Martin, M.D., as Chief Medical Officer.
“We are pleased to welcome Javier to the team and are confident his insights and guidance will be instrumental as we advance the Company’s next phase of growth,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira.
Before that, Dr. San Martin served as Senior Vice President of Clinical Development at Alder Biopharmaceuticals, where he managed medical, regulatory, and clinical operations.
In addition, Athira today announced, as required by The Nasdaq Stock Market Rules, an equity inducement award to Dr. San Martin, the Company’s new Chief Medical Officer.

Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting

Retrieved on:

Friday, April 12, 2024

American Academy, Safety, Patient, Angelman syndrome, Abstract (summary), UBE3A, Trial of the century, Science, AAN, Neurology, Ultragenyx, Child

GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).

Key Points:

GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).
The GTX-102 emerging science presentation will include data beyond what is currently available online.
Title: Clinical Activity and Safety of GTX-102, an Investigational Antisense Oligonucleotide for the Treatment of Patients With Angelman Syndrome
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Ultragenyx will host a conference call at 8:00 a.m.
ET on Wednesday, April 17, 2024, to discuss the new efficacy and safety data from the GTX-102 Phase 1/2 clinical study being presented at the conference.

North River Leerink’s Successful 100 Chestnut Street Development Continues Somerville’s Growth as a Leading Life Science and Innovation Hub

Retrieved on:

Thursday, February 29, 2024

At the heart of this transformation stands 100 Chestnut, a purpose-built 200,000 sq.

Key Points:

At the heart of this transformation stands 100 Chestnut, a purpose-built 200,000 sq.
ft. Life Science building that is 90% leased, reflecting NRL's passion and focus on developing world-class buildings that enable innovation and support the creation of sustainable and vibrant communities.
Our goal is to contribute towards the development of a rich, vibrant and diverse district focused on fostering Art and enabling innovation in the Life Science/Biotech industry,” said Jeff Leerink, Managing Partner at NRL.
And most recently, ADA Forsyth, a leading institute dedicated to improving oral and overall health through innovative research and community outreach, has leased 76,000 sq.

Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

Retrieved on:

Thursday, February 15, 2024

Patient, Burosumab, Ultragenyx, OI

NOVATO, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2023 and reaffirmed its financial guidance for 2024.

Key Points:

Total operating expenses for the fourth quarter of 2023 were $249 million, including non-cash stock-based compensation of $34 million.
Total operating expenses for the year ended December 31, 2023 were $1,003 million, including $135 million of non-cash stock-based compensation.
For the fourth quarter of 2023, Ultragenyx reported net loss of $123 million, or $1.52 per share basic and diluted, compared with a net loss for the fourth quarter of 2022 of $152 million, or $2.16 per share, basic and diluted.
ET to discuss the fourth quarter and full year 2023 financial results and provide a corporate update.

Disc Medicine Strengthens Leadership Team with Appointment of Seasoned Industry Executive Jean Franchi as Chief Financial Officer, and Promotion of Jonathan Yu to Chief Operating Officer

Retrieved on:

Wednesday, February 7, 2024

Merrimack Pharmaceuticals, Business administration, Policy, Corporate finance, Growth, Genetics, Senior, Bachelor, Common stock, Patient, Corporation, IRON, RSU, Franchi, Sale, Hematology, Ultragenyx, Hofstra University, Genzyme

Ms. Franchi will be responsible for finance, investor relations, and other functions and will lead the company’s capital formation and financial strategy.

Key Points:

Ms. Franchi will be responsible for finance, investor relations, and other functions and will lead the company’s capital formation and financial strategy.
“We are excited to welcome an executive of Jean’s caliber and depth of experience to the Disc team, and I’m delighted to announce the promotion of a key executive, Jonathan Yu, to Chief Operating Officer,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
The team has built a unique company with a strong foundation and pipeline with enormous potential,” said Ms. Franchi.
Prior to Replimune, Ms. Franchi served as Chief Financial Officer for Merrimack Pharmaceuticals, Dimension Therapeutics, which was acquired by Ultragenyx, and Good Start Genetics.

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Retrieved on:

Thursday, January 4, 2024

Marketing, Cholesterol, Ultragenyx, NHS England, Adolescence, Hammersmith Hospital, Diet, Ageing, Clinical trial, Trauma, Heart UK, Patient, NHS, ANGPTL3, Lipid, Pharmaceutical industry

Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

Key Points:

Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).
Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.
"We are very pleased that NICE has approved the use of evinacumab within the NHS.
The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough.

Amylyx Pharmaceuticals Appoints Camille L. Bedrosian, MD, as Chief Medical Officer

Retrieved on:

Tuesday, November 28, 2023

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).

Key Points:

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).
In this role, Dr. Bedrosian will lead global functions in medical affairs, regulatory, safety and pharmacovigilance, biometrics, clinical development, and clinical operations.
I’m excited to join Amylyx and contribute to bringing innovative, new treatment options to individuals and communities suffering from neurodegenerative diseases.
“We also want to thank our longtime Chief Medical Officer, Dr. Patrick Yeramian, for his many years of dedication and commitment to building Amylyx into the successful company it is today.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on:

Monday, December 18, 2023

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points:

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on:

Monday, December 18, 2023

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points:

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.