MHRA

Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

Retrieved on: 
torsdag, maj 9, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, STAC, FDA, CTA, Haemophilia, Enzyme replacement therapy, Kite, Investment, Nav1.7, Fabry disease, Poster, Disease, ERT, Sigma-Aldrich, STAAR, Health care, MAA, Partnership, Sangamo Therapeutics, MINT, AAV, Ligand Pharmaceuticals, IND, Tauopathy, Novartis, DNA, Patient, Pivotal, DSM-IV codes, EMA, Prion, BBB, Genome, BLA, MHRA, Manuscript, BioRxiv, Capsid, Modularity, Biogen, Therapy, Food and Drug Administration, European Medicines Agency, Gene, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.
  • “We are pleased with the progress being made in business development discussions across our portfolio, including our Fabry disease program.
  • An IND submission is expected in the fourth quarter of 2024.
  • Revenues for the first quarter ended March 31, 2024 were $0.5 million, compared to $158.0 million for the same period in 2023.

MeiraGTx Reports First Quarter 2024 Financial and Operational Results

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torsdag, maj 9, 2024
FDA, Research, UCL Business, Weight loss, IND, Oxyntomodulin, Visual acuity, RIX, Oral medicine, American Academy, Brain, STN, Facial muscles, Technology transfer, Janssen Pharmaceuticals, Appetite, BLA, MHRA, Messenger, Subthalamic nucleus, Safety, Diabetes, Insurance, RNA, AAOM, AAV, Dopamine, Obesity, DNA, Autoimmune disease, Motor cortex, Treatment, Pros, GMP, GIP, Clinical trial, File, PSMA, Sale, Radiation, Neurodegenerative disease, DSM-IV codes, Saliva, Health care, Xerostomia, Patient, Riboswitch, Gene, Peptide, Head, PYY, Pharmaceutical industry

LONDON and NEW YORK, May 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the first quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • MeiraGTx anticipates receiving an additional $15 million in near-term milestone payments later in 2024.
  • As of March 31, 2024, MeiraGTx had cash and cash equivalents of approximately $119.2 million and in April 2024, the Company collected $19.7 million in receivables which were due in the first quarter of 2024, effectively increasing the cash balance to $138.9 million.
  • The increase of $0.6 million was due to higher interest rates and cash balances during 2024.
  • Interest expense was $3.3 million for the three months ended March 31, 2024, compared to $3.1 million for the three months ended March 31, 2023.

Pharming Group reports first quarter 2024 financial results and provides business update

Retrieved on: 
onsdag, maj 8, 2024
VWAP, VUS, FDA, Phase, Haploinsufficiency, IRP, Iberian languages, Laboratory, Safety, Euronext Amsterdam, Pharmacokinetics, PTEN, Epidemiology, CTLA4, Marketing, World Games, APDS, Health care, MAA, CHMP, Trial of the century, Genetic testing, Disease, Patient, Physician, EMA, Primary immunodeficiency, Ministry, Variant, Committee, MHRA, Diagnosis, Pids, PID, Lymphocyte, Clinical trial, Government, Microsoft Access, Prevalence, Ageing, Pharmaceutical industry

“Pharming delivered a strong first quarter, increasing quarterly revenues by 31% year-over-year to US$55.6 million and keeping the Company on track for our 2024 total revenue guidance of US$280 - US$295 million.

Key Points: 
  • “Pharming delivered a strong first quarter, increasing quarterly revenues by 31% year-over-year to US$55.6 million and keeping the Company on track for our 2024 total revenue guidance of US$280 - US$295 million.
  • Pharming made continued progress in the first quarter of 2024 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.
  • Pharming announced completion of enrollment in the clinical trial for children ages 4 to 11 years old on April 8, 2024.

Leading Academic Neurosurgeons Describe New NICO Myriad SPECTRA™ System as a 'Game-Changing Advancement' in Surgical Removal of High-Grade Glioma Brain Tumors

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fredag, maj 3, 2024
AANS, FDA, University, Medication, Brain, Food, NICO, UAMS, Neuroscience, Health care, Surgeon, Doctor of Philosophy, Medicine, Neurosurgery, Patient, King, MIPS, MHRA, Fluorescence, Neoplasm, Collection, King's College Hospital, Medical imaging

New NICO SPECTRA is only hand-held technology delivering blue light closer to target to enhance tissue fluorescence.

Key Points: 
  • New NICO SPECTRA is only hand-held technology delivering blue light closer to target to enhance tissue fluorescence.
  • "Sending light deep into the brain is not easy and can make these cases extremely difficult.
  • The SPECTRA System provides illumination, resection, collection and biological preservation of tissue for the surgical removal of brain tumors and intracerebral hemorrhages using minimally invasive and open craniotomy surgical approaches.
  • Learn more about NICO technologies at NICOneuro.com ; follow us on LinkedIn and Twitter , view surgical and patient videos on YouTube .

Integrated cancer biotech Infinitopes secures £12.8m seed financing to enhance its Precision Immunomics™ antigen discovery technologies to target five more cancers

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torsdag, maj 2, 2024
Neoplasm, Medication, Cancer, Laboratory, Bias, Innovation, Big Pharma, University of Oxford, Octopus Group, Health care, Recurrence, Kindred, Vaccine, CRUK, Cancer Research Institute, Patient, LC-MS/MS, MHRA, Immune system, Associate, Precision, Growth

OXFORD, England, May 2, 2024 /PRNewswire/ -- Infinitopes Precision Immunomics, an integrated cancer biotech combining world leading platforms in precision antigen discovery with vaccine vectors capable of durably stimulating protective immune responses, today announced the completion of a £12.8m seed funding round led by Octopus Ventures.  The round was heavily oversubscribed, also drawing in funds from Cancer Research Horizons, Cancer Research Institute, CRIS Cancer Foundation, Kindred Capital, Manta Ray, Martlet Capital, Meltwind Advisory, Saras Capital, Wilbe Capital, and expert angel investors. The total funds raised combine new equity investment and a prestigious non-dilutive award from Innovate UK's Future Economy Investor Partnership scheme.

Key Points: 
  • Since incorporation in 2021, Infinitopes has moved swiftly to cement its leadership position in cancer vaccines by overcoming three key challenges in the field.
  • Firstly, using its Precision ImmunomicsTM antigen discovery technologies, the Company can identify and rank tumour signatures, without prior bias, enabling the accurate selection of novel, synergistic tumour targets.
  • Secondly, Infinitopes continues to develop safe and effective, proprietary vector delivery systems, capable of stimulating durable T-cell responses necessary to prevent the recurrence of life-threatening cancer metastases.
  • Infinitopes' lead asset, a novel cancer vaccine called ITOP1, is scheduled to commence a phase I/IIa study in first line cancer patients in the third quarter of 2024.

Integrated cancer biotech Infinitopes secures £12.8m seed financing to enhance its Precision Immunomics™ antigen discovery technologies to target five more cancers

Retrieved on: 
tisdag, april 30, 2024
Neoplasm, Medication, Cancer, Laboratory, Bias, Innovation, Big Pharma, University of Oxford, Octopus Group, Health care, Recurrence, Kindred, Vaccine, CRUK, Cancer Research Institute, Patient, LC-MS/MS, MHRA, Immune system, Associate, Precision, Growth

OXFORD, England, April 30, 2024 /PRNewswire/ -- Infinitopes Precision Immunomics, an integrated cancer biotech combining world leading platforms in precision antigen discovery with vaccine vectors capable of durably stimulating protective immune responses, today announced the completion of a £12.8m seed funding round led by Octopus Ventures.  The round was heavily oversubscribed, also drawing in funds from Cancer Research Horizons, Cancer Research Institute, CRIS Cancer Foundation, Kindred Capital, Manta Ray, Martlet Capital, Meltwind Advisory, Saras Capital, Wilbe Capital, and expert angel investors. The total funds raised combine new equity investment and a prestigious non-dilutive award from Innovate UK's Future Economy Investor Partnership scheme.

Key Points: 
  • Since incorporation in 2021, Infinitopes has moved swiftly to cement its leadership position in cancer vaccines by overcoming three key challenges in the field.
  • Firstly, using its Precision ImmunomicsTM antigen discovery technologies, the Company can identify and rank tumour signatures, without prior bias, enabling the accurate selection of novel, synergistic tumour targets.
  • Secondly, Infinitopes continues to develop safe and effective, proprietary vector delivery systems, capable of stimulating durable T-cell responses necessary to prevent the recurrence of life-threatening cancer metastases.
  • Infinitopes' lead asset, a novel cancer vaccine called ITOP1, is scheduled to commence a phase I/IIa study in first line cancer patients in the third quarter of 2024.

Santhera Announces Preliminary Unaudited 2023 Annual Results and Provides Corporate Update Ahead of Full Report Publication in May

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torsdag, april 25, 2024
FDA, Annual, Pharmacy, NMPA, Private, Partnership, CDE, PDUFA, CEPS, European Medicines Agency, News, Annual report, Intangibles, DMD, U.S. FDA, Catalyst Pharmaceuticals, Tax, CHF, MHRA, Duchenne muscular dystrophy, Quality of life, Leber's hereditary optic neuropathy, Vamorolone, BST, Public, EMA, Marketing, File, Sale, Medication, Treasury, NDA, Health care, Duchenne, Bond, Patient, Bank statement, AGM, Growth, Cryptocurrency

As permitted by SIX Exchange Regulation, the Company will publish the full 2023 Annual Report in May.

Key Points: 
  • As permitted by SIX Exchange Regulation, the Company will publish the full 2023 Annual Report in May.
  • Financially, Santhera reported a 2023 revenue of CHF 103.4 million and net income of CHF 54.8 million, driven by the Catalyst licensing deal.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • SIX Exchange Regulation has permitted Santhera to publish its 2023 Annual Report by May 31, 2024, at the latest.

United Kingdom’s MHRA Approves YORVIPATH® (palopegteriparatide) in Great Britain for the Treatment of Adults with Chronic Hypoparathyroidism

Retrieved on: 
onsdag, april 24, 2024
Hypoparathyroidism, United, Parathyroid hormone, European Commission, Marketing, PTH, Health care, European, Commission, MHRA, European Medicines Agency, Pharmaceutical industry

YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily.

Key Points: 
  • YORVIPATH is a prodrug of parathyroid hormone (PTH 1-34) administered once daily.
  • “With today’s MHRA approval of YORVIPATH, we are expanding our global geographic reach to meet the needs of adults with chronic hypoparathyroidism in Great Britain,” said Camilla Harder Harvig, Executive Vice President and Chief Commercial Officer at Ascendis Pharma.
  • Orphan status provides 10 years of market exclusivity in Great Britain with respect to similar medicinal products in the approved orphan indication of chronic adult hypoparathyroidism.
  • TransCon PTH is also in development for the treatment of adults with chronic hypoparathyroidism in the United States, Japan, and other countries.

AriBio Receives EMA Phase 3 Clinical Trial Authorisation for AR1001 in Treatment of Alzheimer’s Disease (POLARIS-AD)

Retrieved on: 
måndag, april 29, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, Clinical trial, Safety, Food, Plasma, Mirodenafil, Patient, POLARIS, EMA, GDS, CSF, MHRA, Biomarker, European Medicines Agency, Pharmaceutical industry

AriBio Co., Ltd. (AriBio) announced authorisation from the European Medicines Agency (EMA) for POLARIS-AD (AR1001-ADP3-US01), a global, phase 3, early Alzheimer’s disease clinical trial.

Key Points: 
  • AriBio Co., Ltd. (AriBio) announced authorisation from the European Medicines Agency (EMA) for POLARIS-AD (AR1001-ADP3-US01), a global, phase 3, early Alzheimer’s disease clinical trial.
  • EMA now aligns with the U.S. Food and Drug Administration (FDA) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) on the Clinical Trial Authorisation of POLARIS-AD.
  • The final decision from EMA was completed on April 24th with clinical trial approvals in the following member states: Czech Republic, Denmark, France, Germany, Italy, Spain and The Netherlands.
  • Currently, 1,150 participants are expected to be randomized into the Phase 3 clinical trial across 200 sites globally.

Pharvaris Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
onsdag, april 10, 2024
Abele, AAAAI, FDA, Citizenship, Research, Healthcare Improvement Scotland, Trial of the century, SMC, IND, Record, Intellectual property, National Congress, American Academy, Attack, Food, ACAAI, Conference, Hereditary angioedema, MHRA, HAE, Therapy, Network, EOP, Book, PRO, Chemistry, CMC, G&A, Investigational New Drug, Doctor of Philosophy, Medication, Research and development, American College, Mass meeting, Health care, Administration, Bank statement, Annual general meeting, Heredity, Pharmaceutical industry

Pharvaris is currently enrolling in RAPIDe-3, a global pivotal Phase 3 study of deucrictibant immediate-release capsule (PHVS416) for the on-demand treatment of HAE attacks.

Key Points: 
  • Pharvaris is currently enrolling in RAPIDe-3, a global pivotal Phase 3 study of deucrictibant immediate-release capsule (PHVS416) for the on-demand treatment of HAE attacks.
  • Effective April 15, 2024, David Nassif, J.D., will join Pharvaris as Chief Financial Officer and will be responsible for refining and implementing Pharvaris’ corporate financial strategy and activities including financial reporting and operations.
  • Loss for the fourth quarter of 2023 was €32.7 million, resulting in basic and diluted loss per share of €0.74.
  • Pharvaris is a Foreign Private Issuer and prepares and reports consolidated financial statements and financial information in accordance with IFRS as issued by the International Accounting Standards Board.