Pharmaceutical Technology Editors

Cue Biopharma, a clinical-stage biopharmaceutical company focused on injectable biologics designed to selectively target tumor-specific T cells directly within a patient’s body, announced on Oct. 4, 2022 that FDA has granted Fast Track designation to CUE-101 for the treatment of patients with human papilloma virus (HPV16+) recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC) as a monotherapy and in combination with KEYTRUDA (pembrolizumab).

Fast Track is a process designed to facilitate the development and expedite the review of drug candidates to treat serious conditions and fulfill an unmet medical need. Therapeutic candidates with Fast Track designation may be eligible for accelerated approval and priority review if supported by clinical data.

CUE-101 is currently being evaluated in a Phase Ib trial as a monotherapy for the treatment of second line and beyond patients with HPV16+ R/M HNSCC and as a first-line treatment in a Phase I dose escalation and expansion trial in combination with pembrolizumab for the same patient population.

“We are very pleased to have received Fast Track designation from the FDA for CUE-101. This designation not only underscores the large unmet need for patients with R/M head and neck cancer who currently rely on available non-targeted therapies, but also highlights the potential of CUE-101 to provide a significant clinical benefit,” said Matteo Levisetti, senior vice-president, clinical development of Cue Biopharma, in a company press release. “To date in its Phase Ib clinical trials, CUE-101 has demonstrated a favorable tolerability profile and single-agent anti-tumor activity in monotherapy as well as encouraging anti-tumor clinical activity in combination with pembrolizumab, supporting the potential to improve overall survival for these patients. We look forward to providing periodic updates and remain committed to advancing the development of CUE-101 to provide patients with a potentially more effective and better tolerated treatment option. We anticipate initiating a registrational trial for CUE-101 monotherapy by mid-2023.”

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FDA has granted Fast Track designation for CUE-101, a treatment of recurrent/metastatic head and neck squamous cell carcinoma. 

FDA Grants Fast Track Designation to Cue Biopharma

Pfizer announced on Oct. 3, 2022 that it has completed its acquisition of Biohaven Pharmaceuticals, the maker of NURTEC ODT (rimegepant), a migraine therapy approved for both acute treatment and prevention of episodic migraine in adults.

The acquisition adds a calcitonin gene-related peptide (CGRP) portfolio to Pfizer, including rimegepant, zavegepant, and a portfolio of pre-clinical CGRP assets. Pfizer has acquired all outstanding shares of Biohaven not already owned by Pfizer for $149.50 per share, for a total cost of approximately $11.6 billion. Biohaven is now a wholly-owned subsidiary of Pfizer.

Immediately prior to the closing of the acquisition Biohaven completed the spin-off of a new Biohaven company that retained the company’s non-CGRP development stage pipeline compounds, Kv7 ion channel activators, glutamate modulation, myostatin inhibition platforms, preclinical product candidates, and certain corporate infrastructure assets excluded from the Pfizer acquisition. Pfizer owns approximately 3% of the new Biohaven company.

"We are proud to build on Pfizer’s legacy of delivering breakthrough medicines for patients living with complex pain disorders,” said Aamir Malik, executive vice-president, chief business innovation officer, Pfizer, in a company press release. “The success of NURTEC ODT coupled with Biohaven’s CGRP pipeline will strengthen Pfizer’s innovative Internal Medicine pipeline through 2030, and beyond. Combined with Pfizer’s global reach, this acquisition increases our potential to bring new treatment options to patients with migraine – a disease which affects over 1 billion people worldwide.”

Pfizer has completed its acquisition of Biohaven Pharmaceuticals for approximately $11.6 billion.

Pfizer Completes Acquisition of Biohaven Pharmaceuticals

ROSS Ribbon Blenders are designed to minimize risk and maximize productivity and meet Occupational Safety and Health Administration standards with safety shaft guards and limit switches that prevent the operation of the agitator when any cover or manway is open. Additional safety grating can be installed on dedicated charge ports or across the entire blender opening for extra protection and to make it easier for operators to dump bags of dry raw material into the blender. This optional safety grating is highly customizable to provide a tailored solution to the operator’s needs.

The 5 ft³ ROSS Ribbon Blender (pictured above) has a special three-piece grate designed with an angled and pitched profile in lieu of flat surfaces. This provides a more rigid structure and more open area in the grating to improve the rate of loading. Additionally, powders are less likely to accumulate on the pitched surfaces, reducing the amount of sweeping required by the operator. All three sections of the grating have position sensors that interlock with the control panel, ensuring operator safety while running.

ROSS Ribbon Blenders are a customizable solution designed to minimize risk and maximize productivity.

Ergonomic, Safety-Focused Blenders

Canvax’s new inorganic pyrophosphatase is a DNA replication enhancer with ≥ 95% purity and has an enhanced capacity for increasing RNA yield in

transcription (IVT) reactions. The enzyme, which is isolated from 

, has demonstrated stability below 80ºF, and is free of endonucleases, exonucleases, and RNAases.

The enzyme is intended for use in RNA IVT reactions, as it is designed to reduce the inhibitory effects of protein-protein interactions. In addition, it is suitable for use in messenger RNA-based vaccine manufacturing, RNA labeling, and enhancement of DNA replication.

The new inorganic pyrophosphate from Canvax is intended for use in RNA IVT reactions. 

Inorganic DNA Replication Enzyme

L.B. Bohle’s QbCon 1 is intended for truly continuous drying and granulation that can meet all quality requirements for long processing times, featuring very narrow and reproducible residence time distributions in the dryer. The product filter surface for the air is kept permanently by means of a novel cleaning process, which allows for continuous granulation without interrupting the process.

The system uses process analytical technology to monitor the production process in-line with the intent of increasing patient safety, reducing costs, creating better process understanding, and optimizing process control. This entire process is controlled and monitored via a touch panel.

The conveying of the granules and the airflow are independently adjustable, allowing the drying effect of the granules to be controlled via the airflow, the supply air temperature, and the conveying speed. Additionally, the granulator is designed to maintain a stable quality of the granules with regard to moisture and particle size.

L.B. Bohle’s QbCon 1 is intended to meet all quality requirements for long granulation processing time. 

Continuous Granulator with Integrated Dryer

Novo Nordisk and Ventus Therapeutics, a biotherapeutics company focusing on small-molecule formulations, announced an exclusive licensing agreement on Sept. 29, 2022. Novo Nordisk will pay up to approximately $700 million for exclusive worldwide rights to develop and commercialize candidates from Ventus’ portfolio of peripherally-restricted NOD-, LRR- and pyrin domain-containing protein 3 (NLRP3) inhibitors.

According to a company press release, Novo Nordisk will pay Ventus $70 million upfront, as well as supply funding for research and development. Ventus will also be eligible for an additional $633 million that is contingent on achieving various clinical, regulatory, and commercial milestones, as well as tiered royalty fees.

NLRP3 is integral in the formation of the NLRP3 inflammasome, a multiprotein complex that regulates the innate immune system. Inflammasomes are involved in intracellular surveillance of danger signals that trigger an intense inflammatory response; therapeutic inhibition of NLRP3 can, therefore, prevent the formation of this inflammasome, thus inhibiting the inflammatory response.

Ventus’ lead NLRP3 inhibitor program is indicated for various diseases, including nonalcoholic steatohepatitis (NASH), chronic kidney disease, and other cardiometabolic conditions. Per the terms of the agreement, Ventus retains the right to develop NLRP3 inhibitors for certain systemic diseases, including specific inflammatory and respiratory diseases, as well as worldwide rights to the company’s brain-penetrant NLRP3 inhibitor program.

“NLRP3 is a biologically relevant target with significant potential across a number of liver, kidney, and cardiometabolic diseases,” said Karin Conde-Knape, senior vice-president, Global Drug Discovery, Novo Nordisk, in the release. “Ventus has developed a highly differentiated NLRP3 inhibitor program with best-in-class properties and compelling pre-clinical results. We are excited to partner with Ventus to advance this program to provide meaningful clinical benefit to patients within a broad range of diseases.”

“This is an important collaboration for Ventus that validates our structural biology capabilities to discover and develop highly differentiated molecules with novel chemical structures,” said Marcelo Bigal, president and CEO, Ventus Therapeutics, in the release. “We believe Novo Nordisk is the ideal partner to advance our lead NLRP3 program in a broad range of systemic disease areas in which they have deep expertise, such as cardiometabolic diseases.”

Novo Nordisk will pay up to $700 million for the exclusive rights to develop and commercialize candidates from Ventus Therapeutics NLRP3 inhibitor portfolio.