Pharmaceutical Technology Editors

Takeda and Hutchmed, a commercial-stage biopharmaceutical company, announced an exclusive licensing agreement on Jan. 23, 2023. Per the terms of the agreement, Takeda will pay Hutchmed $400 million for the rights to further develop and commercialize fruquintinib outside of mainland China, Hong Kong, and Macau. An additional $730 million, as well as royalties on net sales, is contingent on the drug meeting various regulatory, development, and sales milestones.

Fruquintinib is an orally administered, tyrosine kinase inhibitor of vascular endothelial growth factor receptors (VEGFR)-1, 2, and 3; according to a company press release, the inhibition of VEGFR has been linked to blocking tumor angiogenesis. The treatment was designed to improve kinase selectivity, with the goal of minimizing off-target toxicities, improving tolerability, and providing more consistent target coverage. The drug was approved for use in colorectal cancer (CRC) in China in 2018.

“Fruquintinib has the potential to change the treatment landscape for patients with refractory metastatic CRC who are in need of additional treatment options. We look forward to utilizing our development and commercial capabilities to expand the potential of this innovative medicine to patients beyond China,” said Teresa Bitetti, president, Global Oncology Business Unit, Takeda, in the press release. “Working with Hutchmed will enable us to expand our oncology portfolio, bringing us one step closer to achieving our aspiration to cure cancer.”

“We are pleased to be partnering with a company that shares our mission to improve treatment outcomes for cancer patients and has the scale and expertise in global drug development and commercialization to advance fruquintinib globally outside of China,” said Weiguo Su, executive director, CEO, and chief scientific officer, Hutchmed, in the release. “We believe that fruquintinib has a very strong future and look forward to working with Takeda to unlock its potential.”

Fruquintinib received fast track approval for treatment of metastatic CRC from FDA in 2020. Hutchmed submitted a new drug application in December 2022, which is expected to be completed in the first half of 2023. Takeda plans to submit additional applications to the European Medicines Agency and the Japan Pharmaceuticals and Medical Devices Agency.

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Takeda will pay approximately $400 million upfront for the exclusive worldwide rights to Hutchmed’s fruquintinib, a tyrosine kinase inhibitor, outside of China.

Takeda Strikes $1.13 Billion Deal for Hutchmed Colorectal Cancer Treatment

Eli Lilly and Company (Lilly) announced that FDA had rejected its accelerated approval application for donanemab as a treatment for early symptomatic Alzheimer's disease on Jan. 19, 2022. In its response letter, FDA cited concerns surrounding an insufficient quantity of clinical trial data.

According to a company press release, FDA specifically requested that Lilly provide data from at least 100 patients who received a minimum of 12 months of continued treatment on donanemab. Lilly cited the experimental design of the study, which allowed patients to complete their treatment once targeted deposits of amyloid plaque were reduced to predefined endpoints, as causing the insufficient quantity of data. Lilly is currently in the middle of a confirmatory Phase III clinical trial (TRAILBLAZER-ALZ 2), with topline data expected in the second quarter of 2023.

"We anticipate [TRAILBLAZER-ALZ 2] will confirm the benefit and safety profile we observed in the TRAILBLAZER-ALZ Phase II study and believe that patients and physicians will be well served by having the full Phase III data available alongside our Phase II data when they need to make treatment decisions," said Anne White, executive vice-president and president, Lilly Neuroscience, Eli Lilly and Company, in the press release. "We are committed to working with the FDA to ensure the fastest possible path to bring this potential medicine to patients in need."

In a response letter, FDA stated that it could not grant Eli Lilly and Company accelerated approval due to concerns surrounding insufficient quantity of clinical trial data.

FDA Rejects Accelerated Approval for Lilly’s Alzheimer’s Drug

The FDA is issuing a new guidance to support sponsors in the development of drugs for monkeypox (mpox). The guidance is intended to provide nonclinical, virology, and clinical considerations for mpox drug development programs, targeting on recommendations to support initiation of clinical trials.

In the past, FDA has approved drugs for the treatment of smallpox under regulations commonly referred to as the Animal Rule, but this pathway is not applicable to drugs for mpox because the research team can design and implement clinical trials for mpox that are both ethical and feasible.

The FDA’s guidance documents do not establish legally enforceable responsibilities but instead describe the Agency’s current thinking on a topic and should only be viewed as recommendations.

The guidance is intended to provide nonclinical, virology, and clinical considerations for mpox drug development programs, targeting on recommendations to support initiation of clinical trials.

FDA Issues Guidance on Development of Drugs for Monkeypox

AmMax Bio announced on Jan. 19, 2023 that the European Medicines Agency (EMA) has granted AMB-05X Priority Medicines (PRIME) designation for the treatment of tenosynovial giant cell tumor (TGCT).

“The positive proof-of-concept data from our prior 12-week Phase II study of AMB-05X in TGCT provided the basis for our PRIME application,” said Larry Hsu, PhD, chief executive officer of AmMax Bio, in a press release. “We appreciate EMA’s decision to award AMB-05X its PRIME designation, which validates the high potential therapeutic value of the program and the significant unmet medical need for patients impacted by TGCT.”

AmMax Bio presented positive Phase II data for the novel local administration of AMB-05X in patients with TGCT at the Connective Tissue Oncology Society meeting in November 2022. The efficacy data across multiple clinical endpoints and a favorable safety profile support the potential of AMB-05X as a best-in-class therapy for the treatment of TGCT regardless of surgical resectability.

The PRIME designation was launched by EMA to boost support for the development of medicines that target an unmet medical need. EMA will continue to offer early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks through PRIME, according to the company press release.

The efficacy data across multiple clinical endpoints and a favorable safety profile support the potential of AMB-05X as a best-in-class therapy for the treatment of TGCT regardless of surgical resectability. 

AMB-05X Receives EMA PRIME Designation for Treatment of Tenosynovial Giant Cell Tumor

Asimov, a synthetic biology company advancing the design and manufacture of therapeutics, announced a strategic partnership with Center for Breakthrough Medicines (CBM), a contract development and manufacturing organization (CDMO) on Jan. 18, 2023.

The partnership led to Asimov licensing its clonal GMP HEK293 suspension cell line to CBM for pre-clinical and clinical production of viral vectors for its clients. According to the press release, HEK293 cell lines are the industry standard for producing therapeutic viral vectors, which are used mainly in gene therapies.

CBM will now be available for clients to get immediate access to a high-performance clonal good manufacturing practice qualified cell line as a measure of its comprehensive capabilities for vector manufacturing designed to deliver high yields and higher throughput without comprising quality.

“Our partnership with CBM is a major step forward in our goal to advance therapeutics manufacturing,” said Alec Nielsen, CEO of Asimov, in a press release. “This validates our efforts in the viral vector space, and supports other research directions in host cell optimization, genetic system engineering, and bioreactor process modeling and development. By licensing our HEK293 viral vector production platform, CBM will be able to provide their clients with immediate access to reduce the cost of manufacturing these complex therapeutics.”

The agreement finds CDM offering its clients access to the platform as a part of its end-to-end comprehensive capabilities for vector manufacturing designed to accelerate development and manufacturing timelines of vector-based advanced therapies.

“We believe this is the initial step of many with Asimov, an industry leader in mammalian synthetic biology and cell line development,” said Avi Nandi, chief technical officer at CBM, in a press release. “We continue to make significant investments into our technology platforms to ensure our clients have access to an end-to-end solution that allows them to develop and manufacture at lower costs. Our goal is for rare disease therapies to be accessible to as many patients around the world as possible and our relationship with Asimov removes a key barrier to therapy developers.”

The partnership led to Asimov licensing its clonal GMP HEK293 suspension cell line to CBM for pre-clinical and clinical production of viral vectors for its clients. 

Asimov and Center for Breakthrough Medicines Collaborate for Viral Vector Production

On Jan. 19, 2023, Advaxis, Inc., a biotechnology company focused on the discovery, development, and commercialization of immunotherapies, completed a previously announced merger with Ayala Pharmaceuticals, Inc., a clinical-stage oncology company that develops and commercializes small molecule therapeutics for patients who have rare tumors and aggressive cancers.

The merged company will operate under the name Ayala Pharmaceuticals and will target the development and commercialization of Ayala’s lead program, AL102, for the treatment of desmoid tumors. AL102 has received Fast Track Designation from FDA and is currently in phase 3 of a trial for patients with desmoid tumors.

The merged company will operate under the name Ayala Pharmaceuticals and will target the development and commercialization of Ayala’s lead program, AL102, for the treatment of desmoid tumors. 