Jazz and Zymeworks sign license agreement for antibody development

Jazz Pharmaceuticals has entered into an exclusive licensing agreement with Zymeworks’ subsidiary, Zymeworks BC to develop and commercialise the latter’s HER2-targeted bispecific antibody, Zanidatamab.

Under the deal terms, Jazz will get exclusive rights to develop and market the antibody across all indications in the US, Japan, Europe, as well as all other territories, excluding Asia/Pacific territories, which were previously licensed by Zymeworks.

Zymeworks will receive an upfront payment of $50m upon receiving regulatory clearance, as well as $325m in second one-time payment.

The company is also eligible to receive nearly $525m upon achieving certain regulatory milestones and approximately $862.5m in commercial milestones with a total deal value of around $1.76bn along with royalties between 10% and 20% on Jazz’s net sales.

With new mechanisms of action, Zanidatamab has showed compelling anti-tumour activity in many HER2-expressing cancers as monotherapy and along with chemotherapy and other agents.

At present, the antibody is in pivotal trials as a first-line treatment for HER2-positive gastroesophageal adenocarcinoma (GEA) and as a second-line treatment for HER2-expressing biliary tract cancer (BTC).

Jazz Pharmaceuticals research and development global head and executive vice-president Rob Iannone said: “Zanidatamab is a novel HER2-targeted bispecific antibody with biparatopic binding and the potential to transform the current standard of care in multiple HER2 expressing cancers.

“This agreement reflects Jazz’s strategic focus on opportunities where we can not only apply advanced technologies to address critical unmet patient needs, but where we can also leverage Jazz’s existing integrated capabilities and global infrastructure to commercialise efficiently.”

Zymeworks is developing the antibody in several Phase I, Phase II and pivotal global clinical trials as a targeted treatment option for solid tumour patients.

Jazz and Zymeworks sign license agreement for antibody development

Jazz Pharmaceuticals has entered into an exclusive licensing agreement with Zymeworks’ subsidiary, Zymeworks BC to develop and commercialise the latter’s HER2-targeted bispecific antibody, Zanidatamab.

Under the deal terms, Jazz will get exclusive rights to develop and market the antibody across all indications in the US, Japan, Europe, as well as all other territories, excluding Asia/Pacific territories, which were previously licensed by Zymeworks.

Zymeworks will receive an upfront payment of $50m upon receiving regulatory clearance, as well as $325m in second one-time payment.

The company is also eligible to receive nearly $525m upon achieving certain regulatory milestones and approximately $862.5m in commercial milestones with a total deal value of around $1.76bn along with royalties between 10% and 20% on Jazz’s net sales.

With new mechanisms of action, Zanidatamab has showed compelling anti-tumour activity in many HER2-expressing cancers as monotherapy and along with chemotherapy and other agents.

At present, the antibody is in pivotal trials as a first-line treatment for HER2-positive gastroesophageal adenocarcinoma (GEA) and as a second-line treatment for HER2-expressing biliary tract cancer (BTC).

Jazz Pharmaceuticals research and development global head and executive vice-president Rob Iannone said: “Zanidatamab is a novel HER2-targeted bispecific antibody with biparatopic binding and the potential to transform the current standard of care in multiple HER2 expressing cancers.

“This agreement reflects Jazz’s strategic focus on opportunities where we can not only apply advanced technologies to address critical unmet patient needs, but where we can also leverage Jazz’s existing integrated capabilities and global infrastructure to commercialise efficiently.”

Zymeworks is developing the antibody in several Phase I, Phase II and pivotal global clinical trials as a targeted treatment option for solid tumour patients.

Charles River, Nanoscope to manufacture gene therapies for vision disorders

Charles River Laboratories International and Nanoscope Therapeutics have announced a collaboration to manufacture gene therapies for vision disorders.

The collaboration will use Charles River’s contract development and manufacturing (CDMO) services to manufacture plasmid DNA and viral vectors for late-stage clinical trials that target degenerative ocular diseases, which have no cure.

Through the collaboration, Nanoscope will gain access to manufacturing platforms as well as several Charles River CDMO centres of excellence.

The new partnership builds on Charles River’s purchase of Vigene Biosciences, Cognate BioServices, and Cobra Biologics last year.

This expanded the company’s complete portfolio pf cell and gene therapy (C&GT) to cover every major CDMO platforms, such as viral vector, cell therapy, and plasmid DNA production.

Clinical-stage biopharmaceutical company Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies to cure genetic diseases which cause vision impairment and blindness.

The diseases include Dry Age-related Macular Degeneration (AMD), Retinitis Pigmentosa, and Stargardt Disease.

The company is developing a platform technology that uses Multi-Characteristic Opsins (MCO) which has potential to treat several genetic illnesses.

Its optogenetic therapy uses AAV2 vector for delivering MCO genes into retinal cells for enabling vision in different colour environments.

Charles River Biologics Solutions Corporate senior vice-president Kerstin Dolph said: “We are excited to continue to support Nanoscope Therapeutics’ efforts in the production of gene therapies that are focused on restoring vision for people suffering from retinal degenerative diseases with no known cure.”

Nanoscope’s optogenetic therapy will be given as a single intravitreal injection without requiring any other devices or interventions.

Gilead’s Kite gets EC approval for Yescarta to treat DLBCL and HGBL

Gilead Sciences company Kite has secured approval from the European Commission (EC) for Yescarta (axicabtagene ciloleucel) to treat adult patients with diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL).

The approval was granted based on data obtained from the pivotal Phase III ZUMA-7 study, which is said to be the largest and longest trial of a Chimeric Antigen Receptor (CAR) T-cell therapy against the standard of care (SOC) in this patient population.

ZUMA-7 is an ongoing, randomised, open-label, global and multicentre Phase III study that is assessing the safety and efficacy of a single infusion of Yescarta against current SOC for second-line therapy in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy.

Event-free survival (EFS) is the primary endpoint of the trial, while objective response rate (ORR) and overall survival (OS) are the key secondary endpoints. Patient-reported outcomes and safety are additional secondary endpoints.

According to Gilead, patients receiving Yescarta demonstrated statistically significant improvements in Quality of Life (QoL) at Day 100 as against those who received SOC in the analysis of patient-reported outcomes (PROs).

Kite CEO Christi Shaw said: “We are very proud to announce Kite’s fifth approved indication in Europe in our continued commitment to the research and delivery of cell therapies with curative potential to patients who might benefit around the world.

“Today’s approval marks an important step by providing patients in Europe this option of CAR T-cell therapy earlier in their treatment journey.”

Yescarta is claimed to be the first CAR T-cell therapy approved for patients in Europe who do not respond to first-line treatment.

First approved in Europe in 2018, it is presently used to treat five types of blood cancers, including large B-Cell lymphoma (LBCL), primary mediastinal large B-Cell lymphoma (PMBCL), and follicular lymphoma (FL) besides DLBCL and HGBL.

Bavarian Nordic to supply smallpox and monkeypox vaccine to Switzerland

Bavarian Nordic has received an order from the Swiss Armed Forces and the Swiss Federal Office of Public Health for its Modified Vaccinia Ankara-Bavarian Nordic (MVA-BN) smallpox and monkeypox vaccine doses.

Under the terms of the deal, the company will be responsible for the supply of 100,000 doses of the vaccine.

The company will seek regulatory approval of the vaccine with the Swiss Agency for Therapeutic Products, Swissmedic, in three months as part of the deal.

Developed in collaboration with the US government, MVA-BN is a non-replicating smallpox vaccine that has been approved by the US Food and Drug Administration (FDA), Health Canada and the European Commission as the only vaccine against monkeypox.

It is marketed as Jynneos in the US, Imvanex in Europe, and Imvamune in Canada.

Bavarian Nordic president and CEO Paul Chaplin said: “As the epicenter of the outbreak, Europe has seen a wide spread of monkeypox as well as a high number of cases in many countries.

“Thankfully, the case numbers are now going down, but efforts are still required to end the outbreak and hopefully preventing the disease becoming endemic in the region.

“We are therefore pleased to enter this agreement with the Swiss authorities, complementing the efforts by the EU Commission to provide access to the vaccine for at-risk populations across the entire region.”

First deliveries under this contract are planned to commence imminently, with the final deliveries expected early next year.

In August this year, the company signed an agreement with the Pan American Health Organization (PAHO) for its monkeypox vaccine.

EMA’s CHMP recommends approval for Novartis’ prostate cancer therapy

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion and recommended a marketing authorisation for Novartis’ Pluvicto (177Lu-PSMA-617 or lutetium vipivotide tetraxetan) to treat prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC).

The regulator has recommended the radioligand therapy along with androgen deprivation therapy (ADT) with or without androgen receptor (AR) pathway inhibition to treat progressive PSMA- positive mCRPC adult patients, who have received treatment with AR pathway inhibition and taxeme based chemotherapy.

This opinion is based on the data obtained from the prospective, randomised, open-label, multicentre, international Phase III VISION study.

The study was designed for evaluating the safety and efficacy of Pluvicto (lutetium (177Lu) vipivotide tetraxetan) along with investigator-chosen standard of care (BSoC) compared to BSoC.

It was conducted in PSMA PET-scan positive mCRPC patients who have received androgen receptor (AR) pathway inhibition as well as taxane-based chemotherapy.

In the trial, Pluvicto and BSoC combination showed significantly improved overall survival in PSMA-positive mCRPC patients.

Novartis chief commercial officer and Innovative Medicines International president Marie-France Tschudin said: “This positive CHMP opinion for Pluvicto is an important step forward in our goal of bringing transformative innovation to more patients around the world.

“If approved by the European Commission, Pluvicto would be the first and only commercial radioligand therapy for people with advanced prostate cancer in Europe.

“We are committed to exploring the potential of radioligand therapy to address unmet needs in prostate cancer, including in earlier stages of disease.”

The findings showed that Pluvicto plus BSoC treated group had 60% reduction in the radiographic disease progression or death (rPFS) risk and 38% reduction in death risk compared to BSoC alone.

Decreased appetite, anemia, fatigue, nausea, dry mouth and constipation are the most common adverse events in the study’s Pluvicto arm.

The regulatory decision is expected to come in about two months.

AdAlta, GPCR Therapeutics partner on new cancer treatment approach

AdAlta has announced collaboration with clinical-stage biotech company GPCR Therapeutics to evaluate a new cancer treatment approach.

The companies will assess AdAlta’s CXCR4 inhibiting i-bodies as cancer therapeutics, using GPCR’s combination inhibition technique.

AdAlta owns an i-bodies panel that inhibit CXCR4 signalling in different ways.

The panel includes the company’s lead drug candidate, AD-214, which has progressed to clinical development to treat fibrotic diseases.

GPCR Therapeutics discovered that the combination of CXCR4 inhibitors and molecules that inhibit other GPCRs associated with CXCR4 in cancer can lead to CXCR4’s superior inhibition.

Under the partnership deal, AdAlta will be responsible for supplying its CXCR4 inhibiting i-bodies’ panel.

GPCR Therapeutics will assess the i-bodies along with a generic beta-blocker molecules series which are chosen from its platforms, that inhibit a GPCR, called B2AR.

The studies will assess the combined CXCR4-B2AR inhibition’s effect on cell migration, in vitro cell signalling, and cell killing.

The company will evaluate the combined inhibition of the compounds in vivo in mouse cancer models, if the studies are successful.

AdAlta CEO and managing director Tim Oldham said: “Through the programme, we hope to demonstrate that AdAlta’s i-bodies, when combined with other GPCR inhibitors can have enhanced therapeutic outcomes in cancer, in comparison with the typical approach of inhibiting individual GPCRs.

“This collaboration is consistent with our strategy of expanding the commercial use of our i-bodies in a cost-effective way.”

If studies are successful, AdAlta will have the first option to licence and further market the products resulting from the collaboration.

Meanwhile, GPCR Therapeutics will also have the similar option if it is not exercised by AdAlta.

SiSaf to seek FDA Orphan Drug Designation for SIS-101-ADO

SiSaf has initiated the process to obtain an Orphan Drug Designation from the US FDA for its siRNA therapeutic, SIS-101-ADO, to treat rare genetic skeletal disorders.

The company is seeking regulatory approval for SIS-101-ADO to treat Autosomal Dominant Osteopetrosis Type 2 (ADO2) patients.

CSSi LifeSciences will be responsible for coordinating the regulatory process that SIS-101-ADO needs to undergo.

A systemic Bio-Courier siRNA, SIS-101-ADO has been designed for blocking the CLCN7 gene’s dominant-negative mutations and rescue the ADO2’s phenotype.

It combines an siRNA which suppress the CLCN7 expression, using the company’s Bio-Courier next generation silicon stabilised hybrid lipid nanoparticles (sshLNPTM) technology.

By combining lipid nanoparticle technology with inorganic bioabsorbable silicon, the sshLNPTM technology addresses the limitations of other RNA delivery technologies.

SiSaf founder and CEO Dr Suzanne Saffie-Siebert said: “In recent years, there has been an explosion of interest in RNA therapeutics for a wide range of medical concerns.

“Initiating the regulatory process to have our ADO2 therapeutic obtain Orphan Drug Designation will move this revolutionary treatment closer to the goal of alleviating the pain and suffering that this disease inflicts on people.

“Provided successful, SIS-101-ADO and other Bio-Courier formulated drugs will not only be able to treat rare skeletal disorders but can clear the way for therapeutics for other rare diseases once thought impossible to treat.”

According to the company, genetic skeletal disorders, including ADO2 account for 5% of all birth defects across the world.

The company’s Bio-Courier technology has been designed for accelerating the technical advances and application of promising RNA therapeutics.

Eligo’s EB003 receives FDA Orphan Drug Designation and RPD designation

Eligo Bioscience has received Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation from the US Food and Drug Administration (FDA) for its microbiome gene therapy, EB003.

The CRISPR-based gene therapy has been indicated to treat Shiga-toxin producing bacterial infection to prevent hemolytic uremic syndrome (HUS).

It has been designed to target Shiga toxins from E. coli (STEC) bacteria in the gut of infected patients.

The company stated that the preclinical data supports the capacity of EB003 in efficiently eliminating Shiga-toxin genes from the gut of infected patients.

This will lead to quick decrease in toxin levels and the associated symptoms, as well as in preventing the evolution to HUS.

Eligo Bioscience CEO Xavier Duportet said: “Granting of Orphan Drug Designation and Rare Pediatric Disease designation for EB003 highlights the FDA’s recognition of the potential of how our unique CRISPR-based modalities can be used to address devastating diseases driven by the expression of bacterial genes, such as hemolytic uremic syndrome.

“We are grateful that the FDA is providing additional support for the development of therapies geared towards rare pediatric diseases and encourages us in our mission to propose highly innovative solutions to patients in need.”

According to the company, children aged less than five years are mainly sensitive to the expression of STEC bacteria after having contaminated foods.

The production of toxin in the person’s gut triggers bloody diarrhea, and its translocation and accumulation in the systemic compartment can lead to HUS, which affects kidneys and blood platelets.

China’s NMPA approves sNDA for JW Therapeutics’ follicular lymphoma therapy

The China National Medical Products Administration (NMPA) has approved the supplemental New Drug Application (sNDA) for JW Therapeutics’ relmacabtagene autoleucel injection to treat relapsed or refractory follicular lymphoma.

The injection (abbreviated as relma-cel ) has been approved to treat adult patients with follicular lymphoma that relapses or is refractory in 24 months of second-line or above systemic treatment (r/r FL).

Developed by JW Therapeutics, relma-cel is an autologous anti-CD19 CAR-T cell immunotherapy product which has been developed based on Juno Therapeutics’ CAR-T cell process platform.

The regulatory approval marks relma-cel as the first cell immunotherapy product approved in China to treat r/r FL patients and the second approved indication after its launch in September last year.

It is based on the six months clinical data obtained from the cohort B of a multi-centre, single-arm pivotal RELIANCE study conducted on Carteyva in relapsed or refractory B cell non-Hodgkin lymphoma adult patients in China.

The findings demonstrated that Carteyva showed 92.58% overall response rate (ORR), 77.78% complete response rate (CRR) at month six, and controllable CAR-T associated toxicities in r/r FL patients.

JW Therapeutics co-founder, chairman and CEO James Li said: “Thanks to the patients and investigators who contributed to the clinical studies of Carteyva, and thanks to the regulators for the recognition of Carteyva.

“We are pleased with the second approved indication, which provides a new and breakthrough treatment option for r/r FL patients.

“JW Therapeutics is committed to maximising the value of Carteyva, continuously advancing technology innovation and pipeline development, and improving the accessibility of cell immunotherapy products.”