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Bayer and Mammoth Biosciences to collaborate on novel gene editing technology

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Bayer and Mammoth Biosciences to collaborate on novel gene editing technology

January 10, 2022: “Bayer AG and Mammoth Biosciences, Inc., which is harnessing the diversity of nature to power the next-generation CRISPR products announced a strategic collaboration and option agreement for the use of Mammoth’s CRISPR systems to develop in vivo gene-editing therapies.

Mammoth Biosciences’ groundbreaking gene-editing technology is a key enabling technology, as well as a stand-alone therapeutic modality.

It will significantly enhance Bayer’s efforts to develop transformative therapies for patients faster and strengthen the company’s recently established new cell and gene therapy platform.

Under the terms of the agreement the two companies will start their collaboration with a focus on liver-targeted diseases.

“Bringing together Mammoth’s novel CRISPR systems with our existing gene augmentation and our induced pluripotent stem cell (iPSC) platforms will allow us to unleash the full potential of our cell and gene therapy strategy,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of the Bayer’s Pharmaceuticals Division.

“Partnering with Mammoth’s cutting edge scientific team is a fundamental pillar for our company to improve the lives of patients suffering from conditions that are currently still difficult to treat.”

“We’re excited to be working together with Bayer, building on the technology leap of our novel CRISPR systems, along with Bayer’s expertise in successful drug development,” said Dr. Peter Nell, Chief Business Officer and Head of Therapeutic Strategy at Mammoth.

“This joint effort has the potential to benefit patients by developing CRISPR-based approaches for the clinic with the appropriate urgency, while ensuring scientific excellence and safety.”

Cell and gene therapies are the next step in the evolution of drug development. By addressing the root cause of diseases, they are potentially capable of permanently reversing diseases with a one-time treatment.

Gene editing serves as a key enabler for cell therapies when used outside the living body (ex vivo) and allows therapeutic targeting of a wide range of genetic diseases with a high unmet medical need when used inside the living body (in vivo).

Mammoth Biosciences’ proprietary toolkit of ultra-small Cas enzymes, including Cas14 and Casɸ, allows for expanded high-fidelity gene editing to be combined with targeted systemic delivery.

Under the agreement, Bayer gains access to this novel gene-editing technology, which offers the potential of an advanced in vivo applicability due to the ultra-compact size of these novel CRISPR systems.

Under the terms of the agreement, Mammoth Biosciences will receive an upfront payment of USD 40 million and is eligible to receive target option exercise fees as well as potential future payments in the magnitude of more than one billion USD upon successful achievement of certain research, development, and commercial milestones across five preselected in vivo indications with a first focus on liver-targeted diseases.

In addition, Bayer will pay research funding and tiered royalties up to low double-digit percentage of net sales. The companies are also exploring work on ex vivo projects on a nonexclusive basis.”

https://media.bayer.com/baynews/baynews.nsf/id/Bayer-and-Mammoth-Biosciences-to-collaborate-on-novel-gene-editing-technology?Open&parent=news-overview-category-search-en&ccm=020

Novartis and Molecular Partners report positive topline data from Phase 2 study for ensovibep

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Novartis and Molecular Partners report positive topline data from Phase 2 study for ensovibep

January 10, 2022: “Novartis and Molecular Partners announced that Part A of the EMPATHY clinical trial that compared single intravenous doses of ensovibep, a DARPin antiviral therapeutic candidate vs. placebo to treat COVID-19, met the primary endpoint of viral load reduction over eight days.

The two secondary endpoints also showed clinically meaningful benefit over placebo – (1) composite endpoint of hospitalization and/or Emergency Room (ER) visits or death, and (2) time to sustained clinical recovery.

Novartis confirms it will now exercise its option to in-license ensovibep from Molecular Partners and, following exercise of the option, will seek expedited access globally, first via the FDA’s EUA process.

The global EMPATHY clinical trial, which is being conducted by Novartis, with Molecular Partners as sponsor, is a randomized, double-blind, placebo controlled study in ambulatory (non-hospitalized) adult patients with COVID-19.

EMPATHY Part A enrolled 407 patients to identify a dose of ensovibep with optimal safety and efficacy and recruited patients in the USA, South Africa, India, the Netherlands and Hungary to explore three doses: 75mg, 225mg and 600mg.

Results from the study showed that the primary endpoint was met with a statistically significant reduction in viral load over eight days, compared to placebo, for all three dosing arms.

The secondary endpoint of hospitalization and/or ER visits related to COVID-19, or death showed an overall 78% reduction in risk of events across ensovibep arms compared to placebo.

Treatment arms were generally balanced in terms of demographic, baseline and disease characteristics.

The placebo arm with 99 patients had a total of six events (event rate of 6.0%); five patients were hospitalized, two of whom died due to worsening of COVID-19 and one patient had an ER visit only.

In the 301 patients treated with ensovibep, there were four events, hospitalizations occurred in two patients and two needed to visit ER (event rate of 1.3%). No deaths occurred in any of the patients treated with ensovibep.

All doses were well-tolerated and no unexpected safety issues were identified for any of the doses.

The lowest dose of 75mg is the planned dose for further development. The data will now undergo further review so that Novartis and Molecular Partners can determine the appropriate next steps for the program.

“We are pleased that the results from the EMPATHY trial demonstrate the positive therapeutic effect of ensovibep, with the potential to be an important new treatment option to combat the rapidly evolving SARS-CoV-2 pandemic,” said Vas Narasimhan, CEO of Novartis.

“As COVID-19 continues to burden healthcare systems across the globe, a range of treatments will be needed, and Novartis is proud to continue our collaboration with Molecular Partners on this unique treatment for COVID-19 and contribute ensovibep to this suite of options.” 

With the decision made to exercise the option, Novartis will become responsible for development, manufacturing, distribution and commercialization activities of ensovibep. Novartis has already initiated scale-up activities in its large-scale biologics production facilities.

As the SARS-CoV-2 virus evolves, a multi-solution strategy is needed to combat the pandemic and there will be a need for antiviral treatments to complement the global vaccination efforts.

Despite availability of vaccinations, there continues to be disease transmission, either through pockets of unvaccinated populations, in patients with compromised immune systems and co-morbidities or through emerging variants, and breakthrough infections are likely to continue.

A recent in vitro analysis also showed that ensovibep maintains full neutralization of the pseudoviruses containing the mutations identical to the Omicron variant of concern.

“These encouraging results come at a time when the need for therapies with pan-variant activity, such as ensovibep, has never been greater. 

We are incredibly excited about the opportunity to provide a potential therapeutic option for patients around the world who require access to effective COVID-19 treatments,” said Patrick Amstutz, Ph.D., CEO of Molecular Partners.

“Today’s data are a culmination of a persistent team effort, between ourselves and Novartis, to deliver a tailored antiviral with demonstrated safety and efficacy in global clinical trials.

As pioneers of DARPin therapeutics, our team has the unique ability to rapidly generate and develop multi-specific DARPin therapeutics. We look forward to continue to demonstrate our capabilities and the potential of our pipeline in oncology, virology and for patients in need.”   

Given the pressing public health emergency and the rapid spread of the Omicron variant across the world, Novartis and Molecular Partners are in close liaison with regulatory bodies to seek expedited review and approval of ensovibep as soon as possible.

If approved, ensovibep will be the first multi-specific antiviral molecule for the treatment of COVID-19. 

About ensovibep
Ensovibep is a DARPin therapeutic candidate, designed specifically to inactivate
SARS-CoV-2, the virus that causes COVID-19. DARPins (Designed Ankyrin Repeat Proteins) are mono or multi-specific protein-based therapies, designed to specifically engage their targets for various effects.

Ensovibep was designed to include three individual DARPin domains, each highly neutralizing to SARS-CoV-2. With these domains constructed into a single molecule, ensovibep can block the receptor-binding domain (RBD) of the SARS-CoV-2 spike protein through highly potent and cooperative binding.

This design ensures strong neutralization, even in the presence of mutations of the spike protein and limits the development of escape mutants.

Several characteristics of DARPin therapeutics make them suitable for COVID-19 treatment, including multi-specific binding, the rapid onset of action, and scalable bacterial production.

In vitro testing has shown high neutralization activity of ensovibep against all known SARS-CoV-2 variants, including the variants of concern: Alpha, Beta, Gamma, Delta and Omicron.3

About the EMPATHY clinical trial program
Following promising Phase 1 clinical data for ensovibep the global EMPATHY clinical trial was initiated by Novartis, with Molecular Partners as sponsor, in May 2021.

EMPATHY is a Phase 2 and 3 study looking at the safety and efficacy of ensovibep in symptomatic COVID-19 patients in the ambulatory (non-hospitalized) setting. Ensovibep is administered via a single dose IV infusion.

The EMPATHY clinical trial plans to enroll 2,100 patients. 407 patients were randomized into four arms of Part A of the study to identify a dose with optimal safety and efficacy.

The clinical efficacy and safety of this dose vs. placebo will be further evaluated in Part B, the Phase 3 component of the EMPATHY study which will enroll an additional 1,700 patients globally.

The EMPATHY clinical trial enrolled both vaccinated and unvaccinated adult patients who have experienced at least two mild/ moderate symptoms of COVID-19 within seven days of onset and had a positive rapid antigen test on the day of dosing, confirmed by a PCR test at baseline.

The COVID-19 symptoms include fever, cough, sore throat, low energy, tiredness, headache, muscle or body aches, chills and/ or shortness of breath.

The collaboration with Molecular Partners
Novartis is proud to collaborate with Molecular Partners on the development of ensovibep, a DARPin therapeutic candidate designed for potential use against COVID-19.

With the decision made to exercise the option, Novartis will be responsible for further development, manufacturing, distribution and commercialization activities of ensovibep.

Under the terms of the agreement, Molecular Partners had received an upfront payment of CHF 60 million, including equity.

Molecular Partners will receive a further payment of CHF 150 million, upon Novartis electing to take up the option on the therapeutic candidate, and significant royalties on sales.

Molecular Partners has agreed to forgo royalties in lower income countries, and is aligned with Novartis’ plans to ensure affordability based on countries’ needs and capabilities.  

Novartis response to the COVID-19 pandemic
Novartis is making a number of contributions to the global fight against the COVID-19 pandemic and supporting the stability of global health systems.

The company announced an initial agreement with BioNtech to provide manufacturing capacity for a COVID-19 vaccine at its site in Stein, Switzerland.

Novartis has also donated US $40 million to help communities affected by the pandemic around the world.”

https://www.novartis.com/news/media-releases/novartis-and-molecular-partners-report-positive-topline-data-from-phase-2-study-ensovibep-mp0420-darpin-antiviral-therapeutic-covid-19

FDA Shortens Interval for Booster Dose of Moderna COVID-19 Vaccine to Five Months

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FDA Shortens Interval for Booster Dose of Moderna COVID-19 Vaccine to Five Months

January 07, 2022: “The U.S. FDA amended the emergency use authorization (EUA) for the Moderna COVID-19 Vaccine to shorten the time between the completion of a primary series of the vaccine and a booster dose to at least five months for individuals 18 years of age and older. 

“The country is in the middle of a wave of the highly contagious omicron variant, which spreads more rapidly than the original SARS-CoV-2 virus and other variants that have emerged,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

“Vaccination is our best defense against COVID-19, including the circulating variants, and shortening the length of time between completion of a primary series and a booster dose may help reduce waning immunity.

Today’s action also brings consistency in the timing for administration of a booster dose among the available mRNA vaccines.

We encourage everyone to get vaccinated—it’s never too late to get your COVID-19 vaccine or booster.” 

The most commonly reported side effects by individuals who received a booster dose of the Moderna COVID-19 Vaccine after completion of a two-dose primary series were pain, redness and swelling at the injection site, as well as fatigue, headache, muscle or joint pain and chills.

The fact sheets for recipients and caregivers and for healthcare providers include information about the potential side effects, as well as the risks of myocarditis and pericarditis.

The amendment to the EUA was granted to ModernaTx Inc.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-shortens-interval-booster-dose-moderna-covid-19-vaccine-five-months

Sosei Heptares and Verily will collaborate on immune-mediated diseases

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Sosei Heptares and Verily will collaborate on immune-mediated diseases

January 2022: “Sosei Group Corporation and Verily, an Alphabet precision health company announced they have entered into a strategic research collaboration.

The research agreement brings together the complementary capabilities of Verily’s immune profiling and Sosei Heptares’ G-protein-coupled receptors (GPCR) structure-based drug design.

The collaboration aims to:

  • Advance the understanding of GPCR biology in immune cells, particularly in the fields of immunology, gastroenterology, immuno-oncology and other disorders with immunoprotective or immunopathogenic mechanisms
  • Prioritize and validate GPCR targets with strong potential as drug targets
  • Discover and develop novel drug candidates that modulate these targets

Verily’s proprietary Immune Profiler is a next generation immune mapping platform that combines high-resolution molecular phenotyping performed in Verily’s labs and advanced computational analysis techniques to generate insights into immune system functions.

It will be used to identify GPCR targets that represent new opportunities to modulate immune cell function and ameliorate disease pathology.

The companies will collaborate to prioritize the GPCR targets using Sosei Heptares’ world-leading StaR® (stabilized receptor) platform and structure-based drug design expertise, with the goal of generating lead molecules for further development or out-licensing.

Matt Barnes, Vice President, Drug Discovery at Sosei Heptares, commented: “We are extremely pleased to collaborate with Verily on this exciting and expansive project, which aims to identify GPCRs expressed in immune cells, enhance our understanding of their functional relevance and prosecute as potential drug targets in immunological diseases.

Immunology is a key area of focus for Sosei Heptares and this new collaboration brings together two world-class technologies and teams with the skills and expertise to define key GPCRs as targets for the discovery of new medicines that could have significant impact on patients with immune-based diseases worldwide.”

Jessica Mega, Chief Medical & Scientific Officer, Co-Founder, Verily, added: “We recognize Sosei Heptares as a leader in targeting GPCRs, one of the most valuable families of protein targets to date.

Our complementary expertise in data-driven prioritization of novel therapeutic targets and expertise in GPCR drug development make for a perfect match to accelerate the development of future therapeutic options for patients suffering with immune-mediated disease.”

https://soseiheptares.com/news/744/129/Sosei-Heptares-and-Verily-will-collaborate-to-generate-novel-drug-candidates-against-GPCR-targets-for-immune-mediated-diseases.html

AZ and Neurimmune sign exclusive global collaboration and licence agreement to develop and commercialise NI006

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AZ and Neurimmune sign exclusive global collaboration and licence agreement to develop and commercialise NI006

January 07, 2022: “Alexion, AstraZeneca’s Rare Disease group, has entered into an exclusive global collaboration and licence agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody currently in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

NI006 specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart, with the potential to treat patients with advanced ATTR-CM.

Under the agreement, Alexion will be granted an exclusive worldwide licence to develop, manufacture and commercialise NI006.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis. 

It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.

Marc Dunoyer, Chief Executive Officer, Alexion, said: “With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed.

We look forward to applying this expertise to the development of NI006, which is designed to clear cardiac amyloid fibril deposits with the potential to improve cardiac function for patients living with advanced ATTR-CM, who are currently underserved by existing treatment options.”

There is a significant unmet medical need for patients with various types and levels of severity of amyloidosis that may require multiple mechanisms of action to address those needs.

NI006, an ATTR depleter, adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens our broader commitment to addressing cardiomyopathies that can lead to heart failure.

Financial considerations
Alexion will pay Neurimmune an upfront payment of $30m with the potential for additional contingent milestone payments of up to $730m upon achievement of certain development, regulatory and commercial milestones, as well as low-to-mid teen royalties on net sales of any approved medicine resulting from the collaboration.

Neurimmune will continue to be responsible for completion of the current Phase Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial costs.

Alexion will be responsible for further clinical development, manufacturing and commercialisation.

The transaction is expected to close following satisfaction of customary closing conditions and regulatory clearances.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-and-neurimmune-sign-deal-for-ni006.html

Exscientia and Sanofi establish strategic research collaboration to develop AI-driven pipeline of precision-engineered medicines

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Exscientia and Sanofi establish strategic research collaboration to develop AI-driven pipeline of precision-engineered medicines

January 7, 2022: “Sanofi and Exscientia announced a groundbreaking research collaboration and license agreement to develop up to 15 novel small molecule candidates across oncology and immunology, leveraging Exscientia’s end-to-end AI-driven platform utilizing actual patient samples.

The companies have been working together since 2016 and in 2019, Sanofi in-licensed Exscientia’s novel bispecific small molecule candidate capable of targeting two distinct targets in inflammation and immunology.

“We look forward to deepening our work with Exscientia, a leader in leveraging AI to modernize all aspects of drug discovery and development,” said Frank Nestle, Global Head of Research and Chief Scientific Officer, Sanofi. 

“Sanofi’s collaboration with Exscientia aims to transform how we discover and develop new small molecule medicines for cancer and immune-mediated diseases.

Application of sophisticated AI and machine learning methods will not only shorten drug discovery timelines, but will also help to design higher quality and better targeted medicines for patients.”

Exscientia and Sanofi will collaborate to identify and select target projects, leveraging Exscientia’s personalised medicine platform.

The platform enables a “patient-first” approach through integrating primary human tissue samples into early target and drug discovery research.

By doing so, Exscientia scientists can integrate patient, disease, and clinically relevant data into decisions on potential new medicine candidates earlier in the drug creation process.

In addition to target discovery, Exscientia will lead small molecule drug design and lead optimization activities up to development candidate nomination, with Sanofi assuming responsibility for preclinical and clinical development, manufacturing and commercialization.

“It is immensely exciting to collaborate with Sanofi with our goal of realizing the full potential of AI to deliver the next generation of cancer and immunology medicines,” said Andrew Hopkins, DPhil, CEO and founder of Exscientia. 

“Our AI-driven platform can be leveraged across drug discovery, translational research and development, with applications ranging from improving the precision medicine and quality of drug candidates to enriching for patient selection in clinical trials.

Our expanded collaboration with Sanofi will utilise the breadth of our platform to test AI-designed drug candidates against patient tissue models, potentially providing far better accuracy than conventional approaches such as mouse models.

When you consider the change this represents – testing candidates against actual human tissue years before a clinical trial – it’s transformative.”

Under the terms of the agreement, Exscientia will receive an upfront cash payment of $100 million from Sanofi and will be eligible to receive future research, translational, clinical development, regulatory and commercial milestone payments of up to approximately $5.2 billion in aggregate, if all milestones for all programs are achieved.

In the case that Sanofi commercializes a therapeutic from the collaboration, Exscientia will also be eligible to receive tiered royalties on product sales ranging from high-single-digits to mid-teens and an option for clinical co-investment to increase the royalty rate up to 21% on net sales of co-funded products.

The upfront cash payment of $100 million is expected to be reflected in the first quarter 2022 financial results of Exscientia as cash inflows from collaborations and recognized as revenue over the duration of the agreement.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-01-07-07-00-00-2362917

BlueRock Therapeutics Announces Closeout of First Cohort in Phase 1 Trial for Advanced Parkinson’s Disease

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BlueRock Therapeutics Announces Closeout of First Cohort in Phase 1 Trial for Advanced Parkinson’s Disease

January 6, 2022: “BlueRock Therapeutics, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced the closeout of the first of two cohorts in its ongoing Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with advanced Parkinson’s disease.

The purpose of the Ph1 clinical trial is to evaluate the safety, tolerability and preliminary efficacy of DA01 in patients with Parkinson’s disease.

Enrollment of the first cohort of patients was completed at three active sites in the United States and Canada: Weill Cornell Medicine, University of California, Irvine and University Health Network.

Patients in the first cohort received a bilateral surgery to transplant dopaminergic neurons in the midbrain region.

No severe adverse events were observed in any participating patients.

“By replacing lost neurons in the midbrain region of patients with Parkinson’s disease, we have the potential to regenerate lost function and possibly improve and reverse the disease,” states Joachim Fruebis, Ph.D., Chief Development Officer of BlueRock Therapeutics.

“This is an incredible step forward as we execute on our goal of bringing authentic cellular medicines to patients in desperate need of treatment.”

About the DA01 Phase 1 Trial
Enrollment is currently open for the second and final high-dose cohort in both the United States and Canada.

The primary objective of the Ph1 trial is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant.

The secondary objectives of the trial are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation.

The trial is being conducted at Weill Cornell Medicine, under the guidance of Dr. Sarva, M.D., Principal Investigator (PI), University of California, Irvine, under the guidance of Dr. Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. (PI) and at the University Health Network, under the guidance of Dr. Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S. (PI).

More information about this trial is available at clinicaltrials.gov (NCT#04802733).

About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels.

The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons.

At diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons.Parkinson’s disease often starts with a tremor in one hand.

Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with one million living in the United States.

DA01 is being clinically evaluated in a subset of patients with advanced Parkinson’s disease, where regular medications are no longer fully effective.”

https://media.bayer.com/baynews/baynews.nsf/id/BlueRock-Therapeutics-Announces-Closeout-First-Cohort-Phase-1-Trial-Patients-Advanced-Parkinsons?Open&parent=news-overview-category-search-en&ccm=020

Novartis announces collaboration with Alnylam to explore targeted therapy to restore liver function

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Novartis announces collaboration with Alnylam to explore targeted therapy to restore liver function

January 6, 2021: “Novartis announced a collaboration with Alnylam to leverage Alnylam’s proven, proprietary siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research, potentially leading to development of a treatment designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure.

“There remains an enormous unmet need for new types of medicines to address end-stage liver disease,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research.

“Building on a legacy of leadership in regenerative medicine, we have devised a restorative strategy that could potentially deliver a transformative benefit to patients with liver failure.

We’re delighted now to work alongside Alnylam in this new collaboration, as the Alnylam siRNA platform is optimally suited to translate this concept to clinical investigation.”

End-stage liver disease (ESLD) is a progressive illness, most often resulting from cirrhosis, that is characterized by the destruction of healthy liver tissue and the loss of critical liver function.

The disease has a profound impact on patients’ quality of life, and accounts for over one million deaths globally each year.

Currently, liver transplantation is the only treatment for ESLD, but transplants are invasive procedures and there is a limited supply of organs available for patients in need.

A significant need exists for medicinal alternatives to transplantation that regenerate liver tissue and restore the essential metabolic and synthetic processes that are managed by the liver.

“We are so pleased to collaborate with Novartis,” said Kevin Fitzgerald, Ph.D., Chief Scientific Officer at Alnylam.

“We believe collaborations like this serve as an excellent example of how Alnylam’s leadership in RNAi can fuel new frontiers of medicine with highly innovative targets coming from some of the most admired pharmaceutical companies.”

During the exclusive three-year research collaboration, Alnylam will develop and test potential siRNAs using target-specific assays developed by Novartis.

Once a lead candidate is identified, further development and clinical research will be conducted by Novartis.”

https://www.novartis.com/news/media-releases/novartis-announces-collaboration-alnylam-explore-targeted-therapy-restore-liver-function

FDA Takes Multiple Actions to Expand Use of Pfizer-BioNTech COVID-19 Vaccine

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FDA Takes Multiple Actions to Expand Use of Pfizer-BioNTech COVID-19 Vaccine

January 03, 2022: “Tthe U.S. Food and Drug Administration amended the emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 Vaccine to:

  • Expand the use of a single booster dose to include use in individuals 12 through 15 years of age.
  • Shorten the time between the completion of primary vaccination of the Pfizer-BioNTech COVID-19 Vaccine and a booster dose to at least five months.
  • Allow for a third primary series dose for certain immunocompromised children 5 through 11 years of age.

“Throughout the pandemic, as the virus that causes COVID-19 has continuously evolved, the need for the FDA to quickly adapt has meant using the best available science to make informed decisions with the health and safety of the American public in mind,” said Acting FDA Commissioner Janet Woodcock, M.D.

“With the current wave of the omicron variant, it’s critical that we continue to take effective, life-saving preventative measures such as primary vaccination and boosters, mask wearing and social distancing in order to effectively fight COVID-19.”

What you need to know:

Boosters are now authorized for people 12 years of age and older

Today’s action expands the use of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine to include its use in individuals as young as 12 years of age.

  • The agency has determined that the protective health benefits of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine to provide continued protection against COVID-19 and the associated serious consequences that can occur including hospitalization and death, outweigh the potential risks in individuals 12 through 15 years of age.
  • The FDA reviewed real-world data from Israel, including safety data from more than 6,300 individuals 12 through 15 years of age who received a booster dose of the vaccine at least 5 months following completion of the primary two-dose vaccination series. 
  • These additional data enabled the FDA to reassess the benefits and risks of the use of a booster in the younger adolescent population in the setting of the current surge in COVID-19 cases. 
  • The data shows there are no new safety concerns following a booster in this population. There were no new cases of myocarditis or pericarditis reported to date in these individuals. 

Booster interval updated to five months for people 12 years of age and older 

The FDA is also authorizing the use of a single booster dose five months after completion of the primary vaccination series of the Pfizer-BioNTech COVID-19 Vaccine.

  • Since Pfizer initially submitted safety and effectiveness data on a single booster dose following primary vaccination, additional real-world data have become available on the increasing number of cases of COVID-19 with the omicron variant in the U.S. 
  • No new safety concerns have emerged from a population of over 4.1 million individuals 16 years of age and older in Israel who received a booster dose at least five months following completion of the primary vaccination series.
  • Additionally, peer-reviewed data from multiple laboratories indicate that a booster dose of the Pfizer-BioNTech COVID-19 Vaccine greatly improves an individual’s antibody response to be able to counter the omicron variant.
    Authorizing booster vaccination to take place at five months rather than six months may therefore provide better protection sooner for individuals against the highly transmissible omicron variant.
    Given the demonstrated safety and effectiveness of a booster dose when administered five months after the primary vaccination series, and the fact that a booster dose may help provide better protection against the rapidly spreading omicron variant, the FDA has determined that the known and potential benefits of administering a booster to individuals ages 12 and older at least five months following completion of the primary vaccination series, outweighs the known and potential risks.
  • While today’s action applies to the Pfizer-BioNTech COVID-19 Vaccine, the FDA continues to review data concerning all available vaccines and will provide additional updates as appropriate.

A third primary series dose for certain immunocompromised children ages 5 through 11

Children 5 through 11 years of age who have undergone solid organ transplantation, or who have been diagnosed with conditions that are considered to have an equivalent level of immunocompromise, may not respond adequately to the two-dose primary vaccination series.

Thus, a third primary series dose has now been authorized for this group. This will now allow these children to receive the maximum potential benefit from vaccination.

  • The FDA previously authorized a third primary series dose for use as part of the primary immunization series in individuals 12 years and older.
    The potential effectiveness of an additional dose in children 5 through 11 years of age was extrapolated from data in adults. 
  • The agency used prior analyses conducted as part of the authorization process for healthy children to inform safety in this population and determined that the potential benefits of the administration of a third primary series dose at least 28 days following the second dose of the two-dose regimen, outweighed the potential and known risks of the vaccine. To date, the FDA and CDC have seen no new safety signals in this age group. 
  • Children 5 through 11 years of age who are fully vaccinated and are not immunocompromised do not need a third dose at this time, but the FDA will continue to review information and communicate with the public if data emerges suggesting booster doses are needed for this pediatric population.

“Based on the FDA’s assessment of currently available data, a booster dose of the currently authorized vaccines may help provide better protection against both the delta and omicron variants.

In particular, the omicron variant appears to be more resistant to the antibody levels produced in response to the primary series doses from the current vaccines,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

“With this in mind, the FDA has extended the range of individuals eligible to receive a booster, shortened the length of time between the completion of the Pfizer primary series for individuals to receive a booster and is authorizing a third protective vaccine dose for some of our youngest and most vulnerable individuals.”

The fact sheets for recipients and caregivers and for healthcare providers contain information about the potential side effects, as well as the risks of myocarditis and pericarditis.

The FDA and the U.S. Centers for Disease Control and Prevention have several systems in place to continually monitor COVID-19 vaccine safety and allow for the rapid detection and investigation of potential safety concerns.

The most commonly reported side effects by individuals who received a booster dose or an additional dose as part of a primary series were pain, redness and swelling at the injection site, as well as fatigue, headache, muscle or joint pain and chills.

Of note, swollen lymph nodes in the underarm were observed more frequently following the booster dose than after the second dose of a two-dose primary series. 

The FDA will publicly post documents regarding the agency’s decision on its website following authorization. 

The amendment to the EUA was granted to Pfizer Inc.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-takes-multiple-actions-expand-use-pfizer-biontech-covid-19-vaccine

Novartis announces CAFC upholds validity of Gilenya® dosage regimen patent

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Novartis announces CAFC upholds validity of Gilenya® dosage regimen patent

January 4, 2022: “Novartis announced that the U.S. Court of Appeals for the Federal Circuit (CAFC) issued its decision upholding the validity of US Patent No. 9,187,405 covering a dosing regimen for Gilenya. 

In August 2020, the U.S. Federal District Court for the District of Delaware issued a favorable decision in the Gilenya patent litigation and a permanent injunction was granted against HEC Pharma until the expiration of the ‘405 patent in December 2027 (including pediatric exclusivity). 

HEC Pharma was the only remaining Abbreviated New Drug Application (ANDA) filer challenging this patent. 

This decision confirms the validity of the patent and allows that injunction to remain in place.

Previously, Novartis entered into settlement agreements with a number of ANDA filers.

Those ANDA filers will not be able to launch a generic version of Gilenya, if approved by the FDA, before an agreed upon date that is prior to the expiration of the dosage regimen patent in December 2027.

The potential generic entry date and other terms of the settlement agreement are confidential. 

With this decision, Novartis confirms that it expects no generic versions of Gilenya in the US market for at least the next two years.”

https://www.novartis.com/news/media-releases/novartis-announces-us-court-appeals-federal-circuit-cafc-upholds-validity-gilenya-fingolimod-dosage-regimen-patent

Sanofi’s Availability of the Q4 2021 Memorandum for modelling purposes

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Sanofi’s Availability of the Q4 2021 Memorandum for modelling purposes

January 4, 2022 – “Sanofi announced today that its Q4 2021 Memorandum for modelling purposes is available on the “Investors” page of the company’s website:
https://www.sanofi.com/en/investors/financial-results-and-events/financial-results/Q4-results-2021

As for each quarter, Sanofi prepared this document to assist in the financial modelling of the Group’s quarterly results.

This document includes a reminder on various non-comparable items and exclusivity losses as well as the foreign currency impact and share count. Sanofi’s fourth-quarter 2021 results will be published on February 4, 2022.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-01-04-07-30-00-2360386

Pfizer Invites Public to Listen to Webcast of Pfizer Discussion at Healthcare Conference

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Pfizer Invites Public to Listen to Webcast of Pfizer Discussion at Healthcare Conference

December 30, 2021: “Pfizer Inc. invites investors and the general public to listen to a webcast of a discussion with Albert Bourla, Chairman and CEO, at the Goldman Sachs 14th Annual Healthcare CEOs Unscripted Conference on Thursday, January 6, 2022 at 10:00 a.m. Eastern Standard Time.

To view and listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today.

The transcript and webcast replay of the discussion will be made available on our web site at www.pfizer.com/investors within 24 hours after the end of the live discussion and will be accessible for at least 90 days.

About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-invites-public-listen-webcast-pfizer-discussion-9

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