Pharmaceutical Technology Editors

Acaragen, a biopharmaceutical company focused on developing therapies for rare and orphan diseases, announced the acquisition of Arrevus Inc., another biopharmaceutical company developing therapies for orphan infectious diseases, on Oct. 28, 2021. Following this acquisition, Acaragen will receive rights to Arrevus’ lead candidate, ARV-1801 (now ACG-721), an oral, small molecule treatment in late-stage development for indications including cystic fibrosis, as well as several other preclinical programs.

According to a company press release, Aceragen plans to launch clinical studies for their newly acquired ACG-721 and ACC-801 treatment candidates. ACG-801 is an investigational form of recombinant human acid ceramidase (rhAC) designed to address the enzyme deficiency that causes Farber disease. The company hopes that ACG-721 can create a foundation in the cystic fibrosis field from which it can investigate ACG-801’s complementary mechanism.

“The acquisition of Arrevus represents a major step forward for Acaragen in executing on its stated goal of building a multi-product rare disease company that aspires to commercialize its own products,” said John Taylor, president and CEO, Aceragen, in a company press release. “Adding Arrevus’ clinical and regulatory capabilities along with its lead program in late-stage development, advances our transition to a diversified, rare-disease-focused biotech company with an amazing growth trajectory. We don’t see many other companies in the rare and orphan disease space that have the same combination of superior talent and a late-stage portfolio of targeted, high-value assets.”

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Aceragen’s acquisition of Arrevus adds key treatment candidates to its rare disease pipeline.

Aceragen Acquires Arrevus, Expands Late-Stage Rare Disease Pipeline

Novartis announced on Oct. 29, 2021, that FDA has approved Scemblix (asciminib) for the treatment of chronic myeloid leukemia (CML) in two key indications. FDA granted asciminib accelerated approval for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs) and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

According to a company press release, many patients with CML experience resistance and/or intolerance to currently available TKI therapies, especially those who’ve undergone multiple therapies or who have the t3151 mutation. Asciminib is the first FDA approved CML treatment that binds to the ABL myristoyl pocket, a novel mechanism of action intended to help the set of patients who resist traditional TKI therapies.

Approval was made following the Phase III ASCEMBL trial, which looked at major molecular response (MMR) rates in patients treated with asciminib versus Bosulif (bosutinib). At week 24, asciminib (25%) nearly doubled the MMR rate of bosutinib (13%). Additionally, the proportion of patients who discontinued treatment due to adverse reactions was more than three times lower in the asciminib v (7%) patients than those given bosutinib (25%).

“CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both,” expressed Michael J. Mauro, hematologist and myeloproliferative neoplasms program leader at Memorial Sloan Kettering Cancer Center, in the press release. “The addition of Scemblix into the CML treatment landscape gives us a novel approach to combat this blood cancer, helping address clinical challenges in patients struggling after switching to a second treatment, as well as in patients who develop the T315I mutation and face significantly worse outcomes.”

Novartis’ scemblix (asciminib) is designed to provide a single treatment option to patients struggling with chronic myeloid leukemia.

FDA Approves Novartis’ Scemblix for Treatment of Chronic Myeloid Leukemia

Avantor announced on Nov. 1, 2021, that it had completed a previously announced acquisition of the Masterflex bioprocessing business and its related assets from Antylia Scientific, a portfolio company. Masterflex is an Illinois-based manufacturer of peristaltic pumps and aseptic single-use fluid transfer technologies.

According to a company press release, the Masterflex acquisition is intended to strengthen Avantor's offering in bioproduction platforms such as monoclonal antibodies, cell and gene therapy, and messenger-ribonucleic acid. Masterflex also supports both therapy and vaccine manufacturing, including those related to COVID-19.

"The acquisition of Masterflex extends Avantor's single-use offering and gives us a complete end-to-end solution for aseptic fluid transfer throughout the bioproduction workflow," said Michael Stubblefield, president and CEO of Avantor, in a company press release. "The acquisition demonstrates our commitment to the growing biopharma market and to providing more comprehensive solutions to some of the world's most complex challenges in therapy and vaccine development."

Following the acquisition, Avantor will have access to Masterflex’s various bioproduction operations.

Avantor Completes Acquisition of Masterflex

The WL-1-RT Wraparound Vial Labeling Machine

The WL-1-RT Wraparound Vial Labeling Machine, supplied by MG America via machinery manufacturer Packaging Efficiency Solutions, is capable of applying precisely placed labels on up to 600 vials per minute. This ability is important for high-speed glass vial lines, such as those producing COVID-19 vaccines. 

The WL-1-RT features positive vial control via a trunnion transportation system. The unit can accommodate both thermal transfer and laser coding printing operations, and it is preconfigured for the integration of an optical character recognition and optical character verification vision inspection system. Non-conforming labels are rejected prior to application, and infeed/outfeed by-flow tables provide optimal buffering. 

WL-1-RT Wraparound Vial Labeling Machine can label up to 600 vials per minute.

Wraparound Vial Labeling Machine

PerkinElmer’s antibody-dependent cellular cytotoxicity (ADCC)+ Cell Line is an expression cell line for the company’s Horizon Discovery CHOSOURCE platform, which is designed for rapid identification of clones expressing high levels of recombinant therapeutic proteins. 

The ADCC+ cell line eliminates the fucose gene to increase ADCC activity with the intent of improving the potency and efficacy of therapeutic antibodies and fusion protein biotherapeutics. At the same time, it aims to reduce dosage requirements and side effects for patients.

The ADCC+ Cell Line is designed for rapid identification of clones expressing high levels of recombinant therapeutic proteins.

ADCC Expression Cell Line

The Mettler-Toledo StarWeigh Checkweigher

The Mettler-Toledo StarWeigh checkweigher is designed to provide laboratory accuracy of fill levels at production throughputs for small, instable, or lightweight products such as glass and plastic bottles, vials, and ampules used in liquid pharmaceuticals. 

Up to four load cells can be fitted to the StarWeigh checkweigher, including Mettler-Toledo’s FlashCell, which is capable of delivering weighing results (+/- 5mg) at speeds of up to 300 products per minute. 

StarWeigh is a compact checkweigher that can be installed within existing production lines. It features customizable starwheels to fit different package shape requirements and enable quick product changeovers.

The Mettler-Toledo StarWeigh checkweigher provides fill level measurements of products like glass and plastic bottles, vials, and ampules.