23 June 2020

Sarepta and Codiak BioSciences to develop rare disease therapies

Sarepta Therapeutics has signed a global research and option agreement with Codiak BioSciences for the design and development of engineered exosome therapeutics to treat rare, neuromuscular diseases.

The therapies will be designed to deliver gene therapy, gene editing and RNA technologies.

This engineered exosome approach is intended to deliver genetic therapies without inducing the adaptive immune response.

Up to five neuromuscular targets are covered under the two-year agreement.

Codiak is eligible for up to $72.5m in upfront and near-term licence payments, and research funding. Meanwhile, Sarepta received an option to any of the candidates resulting from this collaboration.

Exosomes act as the body’s natural intercellular communication system and enable the transfer of molecular payloads between cells. Codiak uses its engEx Platform engineer exosomes with select cargos and guides tropism to cell types of interest.

As part of the partnership, Codiak’s exosome engineering capabilities will be combined with Sarepta’s precision genetic medicine expertise to create therapeutics for neuromuscular diseases with limited or no treatment options.

Sarepta Therapeutics president and CEO Doug Ingram said: “Codiak’s engEx technology could potentially address some of the limitations of current treatment approaches and offers broad utility across Sarepta’s therapeutic modalities — gene therapies, gene editing and RNA.

“Codiak’s exosomes are engineered for precise tissue targeting and offer a non-viral delivery approach with non-immunogenic potential, thus opening up avenues for more efficient delivery and potential re-dosing.”

According to the terms of the deal, Sarepta holds an exclusive option to licence Codiak’s technology to develop and commercialise engineered exosome therapeutics for up to five neuromuscular targets.

The partners will work together to design exosomes that can deliver and functionally release specific payloads, such as nucleic acids and gene therapy, in neuromuscular diseases.

If Sarepta decides to exercise its option for a target, Codiak will carry out research and preclinical development through investigational new drug (IND) preparation, and Sarepta will carry out clinical development and commercial activities.

Last month, Sarepta Therapeutics partnered with Dyno Therapeutics to develop next-generation AAV vectors for muscle diseases.

23 June 2020

Sanofi and Translate Bio team up to develop mRNA vaccines

Sanofi vaccines business unit Sanofi Pasteur has expanded its existing 2018 partnership with messenger RNA (mRNA) therapeutics firm Translate Bio for the development of mRNA vaccines against various infectious diseases.

The expanded collaboration will combine Sanofi’s vaccine research and development capabilities with Translate Bio’s expertise and knowledge on mRNA research and development.

Translate Bio is leveraging its mRNA platform for the discovery, design and manufacturing of vaccine candidates while Sanofi Pasteur’s vaccine expertise will help advance the candidates’ development.

Furthermore, Translate Bio will transfer technology and processes for Sanofi Pasteur to create and manufacture mRNA vaccines against infectious diseases.

Currently, the partners are analysing several Covid-19 vaccine candidates in-vivo for immunogenicity and neutralising antibody activity. Findings are intended to facilitate lead candidate selection.

The aim is to launch a first-in-human clinical trial in the fourth quarter of this year.

The companies are working on an mRNA vaccine development candidate against influenza. They plan to advance the candidate through preclinical studies and begin human trials mid next year.

Sanofi Pasteur executive vice-president Thomas Triomphe said: “We are excited by the novel technology and expertise Translate Bio brings, and we believe that adding this mRNA platform to our vaccines development capabilities will help us advance prevention against current and future infectious diseases.”

Under the expansion agreement, Sanofi will make an upfront payment of $425m, including a $300m cash payment and a common stock investment of $125m at $25.59 per share.

In addition, Translate Bio will be eligible for potential milestones and other payments of up to $1.9bn, including $450m of milestones covered by the 2018 agreement, as well as royalty payments on global sales.

Nearly $360m of the potential milestones and other payments are expected over the coming years, including Covid-19 vaccine development milestones.

Sanofi Pasteur will be responsible for all costs during the partnership term. Under this agreement, the company will gain exclusive global rights for infectious disease vaccines.

22 June 2020

Sanofi-Regeneron Dupixent secures China approval for atopic dermatitis

Sanofi and Regeneron Pharmaceuticals’ Dupixent (dupilumab) has received approval from China’s National Medical Products Administration (NMPA) to treat adults with moderate to severe atopic dermatitis.

The drug is indicated for patients who did not experience adequate disease control using topical prescription therapies or when these therapies are not advisable.

Dupixent is a fully human monoclonal antibody designed to block the signalling of the interleukin-4 and interleukin-13 proteins. The drug already holds approval in multiple other countries for moderate to severe atopic dermatitis in some patients.

The China approval was based on positive results from the global LIBERTY AD clinical trial programme involving approximately 3,000 patients with inadequately controlled moderate to severe atopic dermatitis.

During the trials, Dupixent was assessed as monotherapy or in combination with topical corticosteroids for safety and efficacy measures, including skin clearance, overall disease severity and itch.

Results from an ongoing Phase III trial in China in adults with moderate to severe atopic dermatitis will also be submitted to NMPA in the second half of next year.

Sanofi CEO Paul Hudson said: “The approval of Dupixent in China offers a new treatment option with an established safety and efficacy profile.

“This is a meaningful advance for patients and their physicians who have struggled to treat the debilitating symptoms of moderate-to-severe atopic dermatitis which can seriously impact quality of life.”

Meant for use as a subcutaneous injection, Dupixent will be available as a 300mg dose pre-filled syringe in China.

Apart from atopic dermatitis, Sanofi and Regeneron are assessing dupilumab for a variety of diseases driven by allergic and type 2 inflammation such as asthma, eosinophilic esophagitis and chronic obstructive pulmonary disease.

Regeneron Pharmaceuticals co-founder, president and chief scientific officer George Yancopoulos said: “More than 150,000 people have already been treated with Dupixent globally and today’s approval brings this novel treatment to those in China who are in urgent need of new options.”

Last October, Sanofi and Regeneron received European Commission approval for Dupixent to treat adults with severe chronic rhinosinusitis with nasal polyposis (CRSwNP).

19 June 2020

Epizyme’s Tazverik secures FDA approval to treat follicular lymphoma

Epizyme has secured accelerated approval from the US Food and Drug Administration (FDA) for Tazverik to treat relapsed / refractory follicular lymphoma.

Tazverik has been approved for use in previously treated adults with follicular lymphoma and an EZH2 mutation, as well as in relapsed or refractory patients with no other satisfactory treatment options.

Earlier this year, the US regulator approved Tazverik as a treatment for metastatic or locally advanced epithelioid sarcoma patients not eligible for surgery.

In clinical trials, follicular lymphoma patients with an EZH2 activating mutation who had previously received two or more therapies had an overall response rate of 69% to Tazverik, including 12% who achieved a complete response.

In 53 patients with wild-type EZH2, the overall response rate was 34%, including 4% who achieved a complete response and 30% achieving a partial response.

Epizyme said eight patients discontinued because of an adverse reaction during the trial and there were no reported deaths on the study, and no black box warnings or contraindications.

Epizyme chief medical officer Shefali Agarwal said: “In our view, there remains no clear standard of care in the relapsed and/or refractory FL population as not all patients benefit from today’s available therapies.

“Based on this label, physicians will have the ability to use their clinical discretion to prescribe Tazverik for their relapsed or refractory patients regardless of EZH2 mutational status and without regard to a specific line of treatment where other options are not satisfactory.”

The recommended dosage of Tazverik is 800mg, taken orally twice daily, with or without food.

18 June 2020

Orca Bio raises $192m to support cell therapies development

US-based biotechnology firm Orca Bio has raised $192m in a Series D financing round to support the development of cell therapies intended to provide an alternative for standard bone marrow transplants.

The financing round, which brings the company’s total capital to approximately $300m, was led by Lightspeed Venture Partners and an undisclosed investor.

8VC, DCVC Bio, ND Capital, Mubadala Investment Company, Kaiser Foundation Hospitals, Kaiser Permanente Group Trust and IMRF are among other investors who participated in the funding round.

Orca Bio will use the latest funds to advance its cell therapy pipeline and manufacturing platform designed to create therapeutic mixtures of immune and stem cells. These therapeutic mixtures are expected to help treat haematological malignancies, genetic diseases and autoimmune disorders.

The company’s lead candidate, TRGFT-201 is a controlled formulation of T-cells, including subsets of regulatory T-cells. The product is being assessed in Phase I/II clinical trial involving patients with certain blood cancers.

Orca Bio will use the Series D financing to support clinical development of TRGFT-201.

The company is also developing OGFT-001, a controlled cell product candidate with a next-generation formulation of T-cells. It is in a Phase I study for treating patients with blood cancers.

These two trials are said to be one of the largest Phase I cell therapy studies.

Orca Bio co-founder and CEO Ivan Dimov said: “Replacing bone marrow transplants is a logical first step in next-generation allogeneic cell therapy. While a conventional bone marrow transplant administers an uncontrolled cell product, Orca Bio has been the first to deliver a high precision cell therapy.

“We are initially focused on advancing two clinical programmes in patients with blood cancers and have successfully treated the largest-ever number of patients with high precision cell therapy.”

In addition to the indications being studied, the product candidates are expected to provide curative outcomes in an expanded patient population, while decreasing toxicities associated with standard bone marrow transplants.

17 June 2020

Takeda licences psychiatry programmes to Neurocrine Biosciences

Takeda Pharmaceutical has collaborated with US-based Neurocrine Biosciences for the development and commercialisation of therapeutic compounds in its early-to-mid-stage psychiatry pipeline.

As part of the strategic deal, Takeda granted Neurocrine Biosciences an exclusive licence for seven programmes in the pipeline, including three clinical-stage assets.

Clinical-stage assets include TAK-831 in Phase II clinical trials to treat negative symptoms of schizophrenia, Phase II study-ready compound TAK-653 for treatment-resistant depression and another Phase II study-ready compound TAK-041 for anhedonia treatment.

TAK-831 is designed to inhibit D-Amino Acid Oxidase (DAAO), while TAK-653 is an Alpha-Amino-3-Hydroxy-5-Methyl-4-Isoxazole Propionic Acid (AMPA) potentiator and TAK-041 is a G Protein-Coupled Receptor 139 (GPR139) agonist.

Neurocrine Biosciences CEO Kevin Gorman said: “With our deep understanding in the fields of psychiatry and neurology, we look forward to developing new treatments for schizophrenia, treatment-resistant depression and anhedonia as part of our diverse clinical development pipeline.

“This strategic partnership enhances our growing pipeline and strengthens our position as a leading neuroscience-focused biopharmaceutical company.”

According to the terms of the collaboration, Neurocrine Biosciences will develop and commercialise all pipeline compounds covered under the agreement.

The company will pay $120m in upfront payment to Takeda, which is also eligible for development milestones of up to $495m, commercial milestones of up to $1.4bn, as well as sales royalties.

Takeda Pharmaceutical Neuroscience Therapeutic Area Unit head Sarah Sheikh said: “The strategic partnership with Neurocrine Biosciences allows us to continue to build on our leadership in psychiatry and deliver future medicines for these patients while advancing our clinical assets for rare neurological diseases, such as narcolepsy, developmental and epileptic encephalopathies and neurodegenerative conditions.”

Last week, Takeda signed an agreement to sell certain non-core over-the-counter (OTC) and prescription pharmaceutical assets available only in the Asia Pacific to South Korean biopharmaceutical firm Celltrion for $266m in an upfront payment.

16 June 2020

FDA revokes authorisation for antimalarials to treat Covid-19

The US Food and Drug Administration (FDA) has revoked the emergency use authorisation (EUA) for antimalarials, chloroquine phosphate and hydroxychloroquine sulfate, to treat some hospitalised Covid-19 patients.

In March, the FDA awarded the EUA for the use of these drugs donated to the Strategic National Stockpile when a clinical trial was not available or trial participation was not feasible.

According to the regulatory agency, chloroquine and hydroxychloroquine no longer meet the legal criteria for EUA.

Data from an ongoing analysis of the EUA and scientific research showed that the drugs are not likely to be effective against Covid-19 for the authorised EUA uses.

Also, based on the emerging data on serious cardiac adverse events and other potentially serious side effects, the known and potential benefits of the drugs no longer outweigh the known and potential risks for authorised use.

The Biomedical Advanced Research and Development Authority (BARDA) of the US Department of Health and Human Services originally filed for the EUA.

In consultation with the FDA, BARDA sent a letter asking the regulator to revoke the EUA based on the science and data available to date.

FDA Medical and Scientific Affairs deputy commissioner Anand Shah said: “We’ve made clear throughout the public health emergency that our actions will be guided by science and that our decisions may evolve as we learn more about the SARS-CoV-2 virus, review the latest data, and consider the balance of risks versus benefits of treatments for Covid-19.

“We will continue to examine all of the emergency use authorisations the FDA has issued and make changes, as appropriate, based on emerging evidence.”

The FDA noted that a large randomised clinical trial revealed no benefit on mortality or in speeding recovery when hospitalised patients were treated with hydroxychloroquine.

These results are consistent with other emerging data, including evidence that the suggested dosing regimens for the drugs are not likely to kill or inhibit SARS-Cov-2.

16 June 2020

Sanofi commits €610m for vaccine research and production in France

Sanofi has announced plans to invest €610m to boost its vaccine research and production capacities in France, in line with the company’s corporate strategy.

The company will establish a new production site and research centre in the country.

The Evolutive Vaccine Facility (EVF), the plant in Neuville sur Saône, will leverage the latest vaccine production technologies. The project will see an investment of €490m over five years and is estimated to generate 200 new jobs.

The facility will allow the company’s vaccine unit, Sanofi Pasteur, to secure vaccine supplies in case of new pandemic outbreaks, noted Sanofi.

The EVF features an innovative design with a central unit that has multiple fully digital production modules, enabling the simultaneous production of three to four vaccines. This design can help prioritise the production of a specific vaccine depending on public health problems.

The company will also invest €120m to build a research and development (R&D) centre focused on developing vaccines in France at the Sanofi Pasteur site in Marcy-l’Etoile.

This facility will have specialised laboratories for the development of vaccines against emerging diseases and pandemic risks, expected to serve as a world reference for pre-clinical research and pharmaceutical and clinical development.

Sanofi CEO Paul Hudson said: “By investing in a new industrial site and an R&D centre, Sanofi positions France at the core of its strategy, aiming to make France a world-class centre of excellence in vaccine research and production.

“Sanofi is a major healthcare player in France, in Europe, and worldwide. It is our responsibility to focus our resources and expertise against the current pandemic, but also to invest in preparing for future ones.”

The company’s portfolio includes vaccine to protect children, adolescents and adults from influenza, infectious diseases and endemic diseases.

It operates 33 sites in France, including 22 industrial sites and employs nearly 25,000 people.

In April, Sanofi partnered with GlaxoSmithKline (GSK) to develop a vaccine against Covid-19.

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