CHMP Gives Positive Opinion on Takeda’s Qdenga 

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its approval to Takeda GmbH’s Qdenga, a live, attenuated dengue tetravalent vaccine (TAK-003). It is the first time the CHMP has reviewed a medicinal product for the EU market in a centralized procedure at the same time as a medicinal product for non-EU countries under the ‘Eu-Medicines for all’ program (EU-M4all). The EMA’s approval is expected within a few months.

Dengue fever, a mosquito-borne tropical disease, is prevalent in over 100 countries, including Europe (only nine countries had experienced severe dengue epidemics pre-1970). Qdenga, which was approved by the BADAN POM in August this year for use in those aged six to 45 years old, will be used to prevent disease caused by dengue virus (DENV) serotypes 1, 2, 3, and 4 in people as young as four years old.

There is already an approved dengue fever vaccine (Sanofi’s Dengvaxia), but TAK-003 has been shown to provide better protection for young children and those over 45 years old: the tetravalent vaccine was evaluated for safety and benefits in 19 clinical trials involving over 27,000 people aged 15 months to 60 years old from both endemic and non-endemic regions.

The studies’ findings demonstrated the prevention of fever, severe disease, and hospitalization caused by any of the four DENV serotypes. The most common side effects of any dose of Qdenga were injection site pain, headaches, muscle pain, and a general feeling of being ill.

“We are one step closer towards the approval of a dengue vaccine that could benefit many of the millions of individuals around the world exposed to dengue. This is a major moment for the global health community, European countries and the dengue-endemic countries that participated in the EU-M4all procedure,” said Gary Dubin, M.D., president of the Global Vaccine Business Unit at Takeda. “We have been working for many years to help improve the way dengue can be prevented. Our efforts to provide a new option for dengue prevention support Takeda’s overall goal to provide long-term societal value to the people we serve.”

Takeda will host a TAK-003 Investor Event in December 2022 to provide more information on commercial plans and regulatory updates.

FDA Sets Feb Review Date for Gilead Sciences’ Trodelvy

Gilead Sciences expects to receive a response from the FDA in February next year regarding its marketing application for Trodelvy in hormone receptor-positive, HER2-negative metastatic breast cancer, which the company describes as a key new indication for the drug.

Based on the results of the recently reported TROPiCs-02 trial, the US FDA has granted Trodelvy (sacituzumab govitecan) priority review as a treatment for adults with unresectable locally advanced or metastatic HR+/HER2- breast cancer who have progressed on endocrine-based therapy and at least two additional systemic therapies.

It has set a deadline of February 23 to complete its review, which, if positive, would significantly expand the patient population eligible for Trodelvy beyond its current use in triple-negative breast cancer (TNBC) and urothelial cancer.

The priority review continues Trodelvy’s renaissance in HR+/HER2- breast cancer, after the first TROPiCs-02 readout at this year’s ASCO congress revealed that the Trop2-targeting antibody-drug conjugate failed to have a significant impact on overall survival, despite meeting its primary endpoint of an improvement in progression-free survival (PFS). At the recently concluded ESMO meeting, Gilead was more optimistic about the drug’s prospects, noting that a trend toward improved survival with Trodelvy had now become a statistically significant improvement over the control group.

“Trodelvy has already changed the treatment landscape in second-line metastatic triple-negative breast cancer and pre-treated metastatic urothelial cancer, and today’s news marks our third supplemental application acceptance within the last two years,” said Bill Grossman, MD, Ph.D., Senior Vice President, Therapeutic Area Head, Gilead Oncology.

There is no doubt that approval of the new indication will provide Gilead with an opportunity to accelerate sales of Trodelvy, the centerpiece of its oncology growth strategy. It made USD 305 million from TMBC and urothelial cancer in the first half of this year, and Gilead is hoping for significant growth as it seeks to recoup the USD 21 billion it paid for Trodelvy’s original developer, Immunomedics, 2years ago.

Odyssey Therapeutics Announces Oversubscribed USD 168 Million Series B Financing

Odyssey Therapeutics, Inc., a biotech company, has announced a USD 168 million Series B financing led by global venture capital firm General Catalyst, an active healthcare investor. The funds will be used to expand Odyssey’s precision immunomodulator and oncology medicine portfolio.

The latest funding announcement comes on the heels of the company’s USD 218 million Series A financing in December 2021, bringing the total capital raised to USD 386 million, allowing Odyssey to continue its mission of developing innovative medicines for serious human diseases.

All Series A investors are participating in the Series B financing, as are new investors Fidelity Management & Research Company and T. GreatPoint Ventures, Catalio Capital Management, Walleye Capital, Alexandria Venture Investments, and The Healthcare Innovation Investment Fund LLC are among the investors.

“Odyssey is pioneering next generation therapeutics by marrying a powerful drug discovery engine integrating machine learning, biology, and medicinal and structural chemistry with an exceptional leadership team of proven drug developers. We are pleased to partner with General Catalyst and our world-class syndicate of investors and believe the interest in our immunology and oncology pipeline and capabilities further validates our approach to therapeutic development,” said Gary D. Glick, Ph.D., Founder and Chief Executive Officer of Odyssey Therapeutics. “We have made tremendous strides since our launch in 2021, and we value the support as we advance multiple candidates into IND-enabling studies in 2023.”

The ‘drug hunting engine’ of the company works by combining “both established and newly emerging drug discovery technologies to tackle immunology and oncology targets with the greatest clinical and commercial potential.” This includes targets that were previously thought to be “undruggable.”

Tavros Therapeutics Signs Agreement with Bayer Unit on Targeted Oncology Programs

On October 12, 2022, Tavros Therapeutics and Vividion Therapeutics announced a strategic collaboration agreement to discover or target four oncology targets across an initial five-year term. Both players will come up with a uniqure set of expertise to achieve the desired goals. As per the update, Tavros will receive $17.5 million in a cash upfront payment from Vividion. Upon attaining the prespecified preclinical, clinical development, and commercial milestones, Tavros is eligible to receive up to $430.5 million in potential future payments. Moreover, Tavros will get a low-single-digit royalty on sales of specific potential programs. Vividion has options to pursue up to five additional targets with up to $482 million in potential additional future payments.

Vividion Therapeutics is a leading biopharmaceutical company and a wholly owned and independently operated subsidiary of Bayer AG. Vividion utilizes novel discovery technologies to unlock high-value, traditionally undruggable targets with precision therapeutics for devastating cancers and immune disorders. Based in Durham (North Carolina), Tavros Therapeutics, through its precision oncology platform, is working on the new frontier of targeted therapies exploiting tumors’ genetic vulnerabilities. As per the agreement, Tavros utilizes functional and computational genomics technologies to uncover unique vulnerabilities within tumors, discover novel targets and biomarkers in areas of high unmet clinical need, and identify novel clinical positioning strategies for existing molecules. 

Pharming Submits Leniolisib for Approval in the EU

On Oct. 11, 2022, Pharming Group N.V. announced that it had submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for leniolisib. Pharming’s Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor designed to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency in adults and adolescents 12 years or older. 

Earlier in August 2022, EMA’s Committee for Medicinal Products for Human Use (CHMP) granted permission to accelerate the assessment for the leniolisib MAA. Under accelerated assessment, the review timeframe decreases from 210 days to 150 days. As per the updates from Pharming, the marketing authorization for leniolisib in the EEA is anticipated in H1 2023. To date, leniolisib has been well tolerated during both the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration-enabling study.

Activated Phosphoinositide 3-kinase δ syndrome (APDS) is a recently recognized rare primary immunodeficiency disease.  It is estimated that APDS affects approximately 1 to 2 people per million. Variants in either of two genes, PIK3CD or PIK3R1, lead to the development of APDS. These two genes regulate the maturation of white blood cells. Pharming’s Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 is an important cellular messenger activating AKT (via PDK1) and regulates many cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism.

FDA’s Fast Track Designation to Mereo’s Alvelestat for Treatment of AATD-associated Lung Disease

On October 17, 2022, Mereo BioPharma Group announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track designation for its investigational oral neutrophil elastase inhibitor, alvelestat (MPH-966). Earlier, in May 2022, Mereo reported positive top-line safety and efficacy data from the ASTRAEUS Phase 2 study of alvelestat in severe Alpha-1 Antitrypsin Deficiency (A1ATD)-associated emphysema. The Company plans to provide a further update on the program on October 31, 2022.

Mereo is looking forward to having an End-of-Phase 2 meeting with the FDA to discuss the design of a registration-enabling study for alvelestat as a treatment for AATD-associated lung disease, including the potential opportunity for an accelerated approval pathway, around the end of the year.
AATD is a rare genetic disease that results in a deficiency of the alpha-1 antitrypsin protein, which protects the lungs against damaging enzymes that the body releases during inflammation. As per the estimates, about 50,000 people in North America and 60,000 in Europe are affected with severe AATD. AATD can lead to pulmonary emphysema, a progressive, life-threatening lung disease. To improve the AATD treatment scenario globally, several key companies are actively working in the therapeutics market.