Tubulin

Tenaya Therapeutics Announces Publication of Preclinical HDAC6 Inhibitor Data for Heart Failure with Preserved Ejection Fraction in Nature Communications

Retrieved on: 
Monday, February 26, 2024

SOUTH SAN FRANCISCO, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced the publication of preclinical research related to Tenaya’s small molecule inhibitors of histone deacetylase 6 (HDAC6), including TN-301, in the February 26, 2024, issue of Nature Communications. The article, titled “Targeting HDAC6 to Treat Heart Failure with Preserved Ejection Fraction in Mice,” details the potential of inhibiting HDAC6 for the treatment of Heart failure with preserved ejection fraction (HFpEF), a form of heart failure that effects more than three million people in the U.S. alone1.

Key Points: 
  • The article, titled “Targeting HDAC6 to Treat Heart Failure with Preserved Ejection Fraction in Mice,” details the potential of inhibiting HDAC6 for the treatment of Heart failure with preserved ejection fraction (HFpEF), a form of heart failure that effects more than three million people in the U.S. alone1.
  • Tenaya’s highly selective small molecule inhibitors of the enzyme HDAC6 were discovered using the company’s modality-agnostic target discovery and validation capabilities.
  • For preclinical studies, Tenaya researchers used TYA-018, an HDAC6 inhibitor structurally and functionally similar to the company’s clinical candidate, TN-301.
  • The selective effects of HDAC6 inhibition were reaffirmed through genetic deletion studies, in which treatment of Hdac6 knockout mice did not display any of the beneficial effects that wild-type HFpEF mice did following treatment.

Could protecting our proteins help us prevent ageing?

Retrieved on: 
Wednesday, February 7, 2024

This is Deinococcus radiodurans, one of the most resistant bacteria known to date, which lives in arid environments such as desert sand.

Key Points: 
  • This is Deinococcus radiodurans, one of the most resistant bacteria known to date, which lives in arid environments such as desert sand.
  • It can also overcome an irradiation dose 5,000 times greater than the lethal dose for humans.
  • Its DNA isn’t resistant, it’s simply repaired immediately by proteins that are indestructible in the face of this extreme radiation.
  • Hence the secret of the robustness of this extremophilic bacterium depends on the robustness of its “proteome” – the sum of all its proteins – and in particular its DNA repair proteins.

The keys to ageing

  • Numerous models have been proposed to explain the molecular basis of ageing, such as the theory of cellular senescence, the reduction in DNA repair capacity, telomere shortening, mitochondrial dysfunction and oxidative stress or chronic inflammation.
  • These different models all attempt to understand the consequences of ageing, not the causes.


Indeed, rather than focusing on our DNA and trying to protect it to slow down our ageing, what if we protected our proteome?

What is the proteome?

  • Proteins – from the Greek protos meaning “first” – are only exceeded as a constituent of the human body by water, and account for around 20% of its mass.
  • The term was coined by analogy with the genome: the proteome being to proteins what the genome is to genes, i.e.
  • the set of genes/proteins of an individual – this protein set varying according to the activity of the genes.


A structural role: many proteins are central to the maintenance and cohesion of our tissues. For example, actin and tubulin are involved in cell architecture. Keratin is involved in the architecture of our epidermis, hair and nails. Collagen is a protein that plays an important role in the structure of bones, cartilage and skin.
A functional role: enzymatic (for example, proteases are involved in cleaning up dysfunctional proteins and in desquamation), hormonal (for example, insulin regulates glycaemia), transport (for example, aquaporins transport water in the different layers of the skin) or defence (for example, immunoglobulins are involved in the immune response). All vital functions are thus ensured by the activity of proteins.

Carbonylation, the leading cause of irreparable damage to our proteome

  • It is under constant threat, because protein synthesis and degradation also depend on proteins.
  • Over time and as a result of external aggression, the proteome is subjected to various alterations, the most formidable of which is carbonylation, irreversible damage linked to the oxidation of proteins.
  • These carbonylated protein aggregates are found in most age-related diseases, as well as in the main signs of skin ageing.

Antioxidant chaperone molecules to act on the causes of ageing


To correctly perform their many jobs, proteins need to fold into a range of shapes and are helped out by specialised proteins called “chaperones”. These help out the proteins after their synthesis by ribosomes, or their correct folding after stress, such as heat.

  • In short, chaperones (protein or chemical) are the doctors of malformed proteins.
  • At the same time, their antioxidant effectiveness neutralises the causes of carbonylation.
  • In Deinococcus radiodurans, because its proteome is protected against oxidative damage by chaperone molecules, it remains intact and can then able to repair damage to its genome.


Miroslav Radman is founder and scientific director of the Mediterranean Institute for Life Sciences (MedILS). The MedILS has received funding from the NAOS company for several research collaborations. He is a consultant and member of the NAOS Scientific Committee.

Human medicines European public assessment report (EPAR): Mevlyq, eribulin, Status: Opinion

Retrieved on: 
Tuesday, January 2, 2024

Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates.

Key Points: 
  • Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates.
  • Since Mevlyq is administered intravenously and is 100% bioavailable, a bioequivalence study versus the reference product Halaven was not required.
  • Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
  • - Marketing authorisation applicant
    - YES Pharmaceutical Development Services GmbH
    - Opinion adopted
    - 14/12/2023
    - Opinion status
    - Positive

Human medicines European public assessment report (EPAR): Mevlyq, eribulin, Status: Opinion

Retrieved on: 
Monday, December 18, 2023

Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates.

Key Points: 
  • Eribulin inhibits the growth phase of microtubules without affecting the shortening phase and sequesters tubulin into nonproductive aggregates.
  • Since Mevlyq is administered intravenously and is 100% bioavailable, a bioequivalence study versus the reference product Halaven was not required.
  • Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
  • - Marketing authorisation applicant
    - YES Pharmaceutical Development Services GmbH
    - Opinion adopted
    - 14/12/2023
    - Opinion status
    - Positive

Tenaya Therapeutics Presents Encouraging New Clinical and Preclinical Data from HDAC6 Inhibitor Program TN-301 for the Potential Treatment of Heart Failure with Preserved Ejection Fraction at the 2023 HFSA Annual Scientific Meeting

Retrieved on: 
Monday, October 9, 2023

SOUTH SAN FRANCISCO, Calif. and CLEVELAND, Oct. 09, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today released new data for TN-301 at the 2023 Heart Failure Society of America (HFSA) Annual Scientific Meeting. TN-301 is Tenaya’s highly selective small molecule inhibitor of histone deacetylase 6 (HDAC6) being developed for the potential treatment of heart failure with preserved ejection fraction (HFpEF).

Key Points: 
  • TN-301 is Tenaya’s highly selective small molecule inhibitor of histone deacetylase 6 (HDAC6) being developed for the potential treatment of heart failure with preserved ejection fraction (HFpEF).
  • "TN-301 continues to generate promising early data as a differentiated candidate for the treatment of HFpEF, a form of heart failure with few treatment options and unacceptably low survival rates.
  • In Stage 1, participants received single ascending doses (SAD) of either TN-301 at doses ranging from 1mg – 700mg or placebo.
  • Plasma exposure and target engagement observed in this healthy participant study met or exceeded those required for maximal efficacy in preclinical studies.

Tenaya Therapeutics Reports Second Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, August 9, 2023

SOUTH SAN FRANCISCO, Calif., Aug. 09, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today reported financial results and provided a corporate update for the second quarter ended June 30, 2023.

Key Points: 
  • Second Quarter Cash and Investments of $152 Million;
    SOUTH SAN FRANCISCO, Calif., Aug. 09, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today reported financial results and provided a corporate update for the second quarter ended June 30, 2023.
  • Tenaya anticipates dosing its first patient in the “MyPeak-1” Phase 1b multi-center, open-label, dose-escalation clinical trial of TN-201 during the third quarter of 2023 (ClinicalTrials.gov #NCT05836259).
  • Non-cash stock-based compensation included in G&A expense was $2.1 million for the quarter ended June 30, 2023.
  • Net Loss: Net loss for the quarter ended June 30, 2023, was $33.3 million, or $0.45 per share.

Tenaya Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 8, 2023

SOUTH SAN FRANCISCO, Calif., March 08, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today reported financial results for the fourth quarter and full year ended December 31, 2022, and provided a corporate update.

Key Points: 
  • “Tenaya enters 2023 well capitalized and with meaningful milestones ahead,” said Faraz Ali, Chief Executive Officer of Tenaya.
  • Research & Development (R&D) Expenses: R&D expenses were $25.7 million for the fourth quarter and $94.5 million for the full year ended December 31, 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $8.8 million for the fourth quarter and $31.1 million for the full year ended December 31, 2022.
  • Net Loss: Net loss was $33.5 million, or $0.61 loss per share for the fourth quarter ended December 31, 2022.

FLAG Therapeutics Receives Rare Pediatric Disease (RPD) Designation for FLAG-003 for the Treatment of Children with Diffuse Intrinsic Pontine Glioma (DIPG)

Retrieved on: 
Tuesday, January 31, 2023

RALEIGH, N.C., Jan. 31, 2023 (GLOBE NEWSWIRE) -- FLAG Therapeutics, Inc., announced today that FLAG-003, an investigational small molecule therapy for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare, highly aggressive and difficult to treat brain tumor found in children, has been granted Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA).

Key Points: 
  • RALEIGH, N.C., Jan. 31, 2023 (GLOBE NEWSWIRE) -- FLAG Therapeutics, Inc., announced today that FLAG-003, an investigational small molecule therapy for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare, highly aggressive and difficult to treat brain tumor found in children, has been granted Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA).
  • The FDA grants RPD designation to drugs intended for the prevention or treatment of rare pediatric diseases.
  • FLAG-003 was previously granted Orphan Drug Designation (ODD) for the treatment of all gliomas, including glioblastoma multiforme (GBM) and DIPG, by the FDA.
  • “Receiving Rare Pediatric Disease designation for FLAG-003 for children with DIPG underscores the dire need for a treatment option for this devastating, fatal form of brain cancer,” stated Frank Sorgi, Ph.D., President and Chief Executive Officer of FLAG Therapeutics.

Testicular Cancer - Global Pipeline Insights, 2022: Featuring Sanofi, Seagen, BioNTech, Context Therapeutics & Leadartis - ResearchAndMarkets.com

Retrieved on: 
Wednesday, January 11, 2023

This "Testicular Cancer - Pipeline Insight, 2022" report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Testicular Cancer pipeline landscape.

Key Points: 
  • This "Testicular Cancer - Pipeline Insight, 2022" report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Testicular Cancer pipeline landscape.
  • Testicular cancer is one of the most common malignancies in men aged 15 to 45 years.
  • Complex environmental and genetic factors are involved in the development of testicular cancer; common risk factors include cryptorchidism, family history of testicular cancer, personal history of testicular cancer in the contralateral testis, age, and ethnicity.
  • Testicular Cancer pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

Tenaya Therapeutics Announces TN-201 IND Clearance and Anticipated 2023 Milestones

Retrieved on: 
Monday, January 9, 2023

SOUTH SAN FRANCISCO, Calif., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, announced today that the U.S. Food and Drug Administration (FDA) has provided clearance of its Investigational New Drug (IND) application to initiate clinical testing of TN-201. In addition, Tenaya shared anticipated 2023 program milestones and updated cash runway guidance.

Key Points: 
  • In addition, Tenaya shared anticipated 2023 program milestones and updated cash runway guidance.
  • We are enrolling patients across three non-interventional studies for our gene therapy programs and plan to file an IND for TN-401.
  • Tenaya anticipates dosing the first patient in the Phase 1b clinical trial of TN-201 in the third quarter 2023.
  • Tenaya plans to submit an IND application to the FDA in the second half 2023 to enable clinical development of TN-401.