15 November 2019

Sarepta and StrideBio sign gene therapy development deal

Sarepta Therapeutics has partnered with StrideBio for the development of in-vivo adeno-associated viral (AAV) based therapies for up to eight targets of the central nervous system (CNS) and neuromuscular conditions.

Under the collaboration and licence agreement, Sarepta obtained exclusive rights to certain targets that will be based on StrideBio’s capsid technology to improve specific tropism to tissues and avoid neutralising antibodies.

The companies will also work on approaches to address re-dosing problems in individuals who received AAV-delivered gene therapy.

StrideBio is responsible for investigational new drug (IND) related research, development and manufacturing for the first four CNS targets.

The initial four targets are MECP2 for Rett syndrome, SCN1A for Dravet syndrome, UBE3A for Angelman syndrome and NPC1 for Niemann-Pick disease.

Sarepta Therapeutics president and CEO Doug Ingram said: “Our partnership with StrideBio expands our research portfolio by up to eight new targets and, through our strategic partnering approach that has our collaborator lead all IND-enabling research and development, ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities.”

As part of the alliance, StrideBio will carry out AAV capsid development and non-clinical activities. The company will also manufacture preclinical candidates selected for clinical studies.

The companies agreed to share early clinical development activities for certain targets and Sarepta will conduct late-stage development and commercialisation.

Sarepta will make an upfront payment of $48m in cash and stock to StrideBio, along with development, regulatory and commercial milestones for the first four programmes.

StrideBio is also eligible for royalties on global net sales of commercial products under the alliance.

Sarepta will hold an exclusive option for four additional targets based on the StrideBio capsid technology. This involves an upfront payment of up to $42.5m and downstream milestones.

StrideBio can choose to gain rights to co-develop and co-commercialise one of the partnership targets. Sarepta also agreed to participate in StrideBio’s next financing round.

15 November 2019

Bayer to invest $100m for alliance with Dewpoint Therapeutics

Bayer has entered an option, research and licence agreement valued at approximately $100m with Dewpoint Therapeutics to develop therapies for cardiovascular and gynaecological conditions.

Under the alliance, Bayer will use Dewpoint’s drug platform, which targets biomolecular condensates, to analyse its small molecule compound library for new treatments.

Bayer noted that nearly 80% of the human proteome cannot be still targeted by small molecules.

Small molecules account for more than 80% of all marketed therapies and biomolecular condensates research is expected to expand the conventional drug target space.

Bayer added that biomolecular condensates could help identify new targets and new methods for targets that were previously intractable.

Bayer research and development head Dr Joerg Moeller said: “As we continue to broaden our capabilities in research and development, the collaboration with Dewpoint gives us access to breakthrough innovation potential.

“New analytic tools and a growing understanding of biomolecular condensates could provide new insights into cellular functions that previously have not been considered by scientists in drug development, enabling us to identify novel pharmacological targets for future therapies.”

The agreement will provide Bayer with an option for exclusive licence of certain new therapies resulting from the collaboration.

The companies will also work together to enhance Dewpoint’s footprint in Germany and leverage local capabilities to develop new drugs.

Dewpoint Therapeutics CEO Amir Nashat said: “We look forward to combining Bayer’s expertise in chemistry and drug development and Dewpoint’s novel platform and insights into the role of biomolecular condensates in disease.

“We also look forward to working closely with Bayer to expand our capabilities in Germany and put the local biotech ecosystem at the forefront of this important and emerging area.”

In September, Bayer committed more than $30m to support research projects on therapies for chronic lung diseases in the US.

14 November 2019

WHO announces pilot for insulin prequalification

The World Health Organization (WHO) has launched a pilot programme for insulin prequalification to boost access to diabetes treatment.

Under the two-year programme, primarily meant for low and middle-income countries, WHO will assess insulin developed by manufacturers for quality, safety, efficacy and affordability.

It is in line with the organisation’s measures to address rising diabetes burden.

Discovered nearly ten decades ago, insulin is required by patients with type 1 or type 2 diabetes. However, high prices are known to hinder access to the medication.

WHO prequalification is intended to improve the availability of quality products on the international market, offering more options at lower prices.

WHO director-general Dr Tedros Adhanom Ghebreyesus said: “Diabetes is on the rise globally, and rising faster in low-income countries. Too many people who need insulin encounter financial hardship in accessing it, or go without it and risk their lives.

“WHO’s prequalification initiative for insulin is a vital step towards ensuring everyone who needs this life-saving product can access it.”

The organisation noted that currently, three manufacturers cater to the majority of the global insulin market with prices not accessible for many people and countries.

Data collected from 24 countries between 2016 and 2019 suggests that insulin and analogue insulins were available in 61% and 13% health facilities, respectively.

According to data, insulin supply for one month will cost 22% of a worker’s earnings in Accra, Ghana.

In developed countries, patients usually have to store insulin, which could have a life-threatening impact on those who do not obtain the appropriate amount.

Prequalified products from added companies are expected to improve competition and allow a steadier insulin supply in all countries.

In addition to insulin prequalification, WHO is planning to create price reduction strategies for analogues, update diabetes treatment guidelines, as well as enhance delivery systems and access to diagnostics to lower disease burden.

13 November 2019

US researchers launch neurohub for brain disease therapies

US researchers have launched a research network, Weill Neurohub, to develop treatments for brain and nervous system diseases and disorders.

Weill Neurohub will involve neuroscientists and researchers from the University of California, Berkeley (UC Berkeley), the University of California, San Francisco (UC San Francisco) and the University of Washington (UW).

The hub is supported by $106m from the Weill Family Foundation.

It will leverage the expertise and resources from the 17 National Laboratories under the US Department of Energy (DOE), which collaborated with the Weill Family Foundation in August.

The collaboration uses DOE’s artificial intelligence (AI) and supercomputing capabilities for research on traumatic brain injury (TBI) and neurodegenerative diseases.

Also, the hub will offer funds for UW, UC Berkeley and UC San Francisco researchers working on cross-disciplinary various projects.

However, the majority of the funds will go to cross-institutional projects involving research on the brain and techniques to disease, including imaging, engineering, genomics, molecular therapeutics, computation and data analytics.

Weill Neurohub could also form other partnerships for additional resources, research and development efforts, information and data sharing.

Weill Family Foundation president Joan Weill said: “Despite amazing advances in neuroscience, new therapies are not reaching patients with mental illness and neurological disorders nearly as quickly as they have for heart disease and cancer.

“And in addition to the terrible personal toll these illnesses exact on patients and their families, they also have a massive impact on our healthcare system and on the global economy. Our goal, through the broad and multifaceted approach of the Weill Neurohub, is to begin to change that.”

An Information Technology & Innovation Foundation study in 2016 revealed that neurological and psychiatric disorders and diseases cost more than $1.5tn annually to the US economy alone.

12 November 2019

Lupin to divest Kyowa Pharmaceutical for $526m

Pharmaceutical company Lupin has agreed to divest its stake in Kyowa Pharmaceutical Industry to private equity firm Unison Capital Partners’ entity Plutus for JPY57.4bn ($526m).

The transaction will be carried out by Lupin subsidiary Nanomi, which holds 99.82% in Kyowa Pharmaceutical.

Founded in 1954, Kyowa Pharmaceutical is engaged in the development, manufacturing and sale of branded and generic pharmaceutical products in Japan.

Its portfolio includes products across various therapeutic areas, including cardiovascular and gastroenterology fields. The company sells central nervous system / neurology products under the Amel brand.

Lupin acquired majority ownership in the company in 2007. Currently, Kyowa Pharmaceutical has manufacturing facilities in Sanda and Tottori, along with a research centre in Osaka.

Kyowa Pharmaceutical generated revenues of JPY14.241bn ($131m) in the first half of the fiscal year 2020 and JPY28.335bn ($260m) in the fiscal year 2019.

Lupin estimates the divestiture to generate net cash inflow of approximately JPY32.596bn ($299m). The proceeds from the deal will be used to boost the company’s balance sheet and as growth capital for organic and inorganic initiatives.

Lupin CEO Vinita Gupta said: “We acquired Kyowa in 2007 and since then have made a material difference to the Japanese healthcare system with the affordable medicines we brought to market.

“This transaction is aligned with our vision to focus on our key markets and strategic priorities to achieve sustainable growth in the mid to long-term.”

Unison’s investments in healthcare will help boost Kyowa’s footprint in the CNS field.

In a statement, Unison said: “Unison aims to support Kyowa’s management team as the company continues to strengthen its existing business, based on pursuing a hybrid (generic / brand) strategy.

“The company will also work on realising its unique growth strategy of expanding its product and service portfolio beyond drugs, which Unison will support through its healthcare ecosystem of researchers, advisors and strategic partners, including Unison’s own healthcare portfolio companies.”

Lupin’s board of directors approved the transaction, which is expected to close in March next year.

12 November 2019

EC approves Merck’s Ebola vaccine Ervebo

Merck (MSD) has secured conditional approval from the European Commission (EC) to market its Ervebo vaccine for active immunisation against Ebola Virus Disease (EVD) caused by the Zaire Ebola virus.

The V920 Ebola Zaire vaccine received a positive response from the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) last month, for use in patients aged 18 years and above.

The EC authorisation will allow marketing of Ervebo in 28 European Union member countries and Iceland, Liechtenstein and Norway.

Furthermore, Merck can now start manufacturing licensed doses of the vaccine in Germany, set to be available in the third quarter of next year.

The company also partnered with the World Health Organization (WHO), the US government and Gavi, the Vaccine Alliance, for improved access to the investigational V920 vaccine in the Democratic Republic of the Congo (DRC).

To support ongoing efforts in the DRC, Merck will produce additional doses over the next year.

Merck is also seeking the WHO seeking prequalification status for the vaccine and filed submissions with several African country National Regulatory Authorities in alliance with the African Vaccine Regulatory Forum (AVAREF).

The vaccine will be registered in multiple markets in Africa if approved.

Merck chairman and CEO Kenneth Frazier said: “We also look forward to continuing to work with the FDA and the African countries on their regulatory reviews over the coming months and with the WHO on vaccine prequalification, which will help broaden access to this important vaccine for those who need it most.”

Ervebo also received priority review status from the US Food and Drug Administration (FDA). The regulator is expected to provide a decision in March next year.

Earlier this month, Janssen Pharmaceutical submitted applications to the EMA for approval of a vaccine regimen to prevent EVD by Zaire Ebola virus.

11 November 2019

Johns Hopkins researchers develop anti-cancer compound

Researchers at Johns Hopkins Medicine in the US have developed a compound to block glutamine metabolism, slowing tumour growth, altering the tumour microenvironment and boosting the anti-tumour T-cell generation.

The compound, JHU083, is a prodrug version of glutamine antagonist, DON, designed to become active and functional within the tumour.

As glutamine is necessary for tumour metabolism, JHU083 is expected to help treat various cancers. Researchers added that the drug selectively targets tumour cells.

Johns Hopkins Kimmel Cancer Center Bloomberg~Kimmel Institute for Cancer Immunotherapy associated director Jonathan Powell said: “By targeting glutamine metabolism, we were not only able to inhibit tumour growth and change the tumour microenvironment but also alter the T-cells in a way that we markedly enhanced immunotherapy for cancer.”

When tested in mice models of colon cancer, lymphoma and melanoma, the drug candidate was able to significantly reduce tumour growth and improve survival by targeting tumour cell metabolism and tumour microenvironment.

Permanent cures were observed with JHU083 in mice via triggering of the anti-tumour immune response.

Furthermore, the cancer-free mice rejected the reinjection of new tumours, suggesting an immune memory that could identify and attack cancer.

Further research showed improved anti-tumour effects with simultaneous administration of JHU083 plus an anti-PD-1 checkpoint inhibitor, compared to anti-PD-1 therapy alone.

Powell added: “Initially, we thought we would need to use the two therapies sequentially in order to avoid any potential impact of the metabolic therapy on the immunotherapy.

“Remarkably, however, it turned out that the combined treatment worked best when we gave them simultaneously.”

Researchers also found that the compound could boost the efficacy of adoptive cellular therapy indicating its use to enhance CAR-T cell therapy.

The team is also planning to study JHU083 in combination with various types of immunotherapy.

8 November 2019

Janssen files for European authorisation of Ebola vaccine

Johnson & Johnson subsidiary Janssen Pharmaceutical has filed marketing authorisation applications (MAAs) with the European Medicines Agency (EMA) for the approval of an Ebola vaccine regimen.

The two-dose regimen is intended to prevent Ebola virus disease (EVD) caused by Zaire ebolavirus species. Janssen submitted two MAAs for each vaccine in the regimen.

Based on Janssen’s AdVac technology, Ad26.ZEBOV is the first dose of the Ebola vaccine regimen. The second MVA-BN-Filo dose is based on MVA-BN technology from Bavarian Nordic.

The doses are to be administered approximately eight weeks apart.

For the vaccine regimen, adenovirus serotype 26 (Ad26) and Modified Vaccinia Virus Ankara (MVA) are genetically modified to prevent replication in human cells.

The vectors are also modified to generate one of the Ebola virus proteins to induce an immune response.

In the MAAs, Janssen included results from Phase I, II and III clinical trials, which were conducted to assess the safety and immunogenicity of the vaccine regimen in adults and children.

The applications are also supported by data from preclinical studies and immunobridging analyses.

More than 6,500 individuals in the US, Europe and Africa have participated in more than ten trials of the Ebola vaccine to date.

Janssen Pharmaceutical global therapeutic area head Johan Van Hoof said: “Our goal is to deliver a vaccine that can be used both in response to Ebola outbreaks and also more proactively as a prophylactic tool to help countries protect their populations.”

This September, EMA’s Committee for Medicinal Products for Human Use awarded an accelerated assessment for the MAAs.

Janssen is in discussions with the US Food and Drug Administration about the data set necessary to seek approval for its vaccine regimen.

The company also partnered with the WHO to register the product in African markets.

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