Pharmaceutical Technology Editors

The National Institute of Health (NIH) recently unveiled a new process for revealing key details of choroideremia, a rare genetic disorder that leads to blindness. Found through a study by the National Eye Institute (NEI), the approach combines traditional eye imaging techniques with adaptive optics, a type of technology that enhances imaging resolution.

According to an agency press release from Sept. 13, 2022, this approach was developed by Johnny Tam, head of the Clinical and Translational Imaging Unit at NEI. By combining adaptive optics with indocyanine green dye, the team was able to view live cells in the retina, including light-sensing photoreceptors, retinal pigment epithelium (RPE), and choroidal blood vessels. This allowed the researchers to see the extent to which choroideremia disrupts these tissues, information which can be used to design effective treatments for the condition.

The major findings of the study concerned the retinal pigment epithelium (RPE), a layer of pigmented cells needed for the nourishment and survival of photoreceptors. Patients with choroideremia had RPE cells that were significantly enlarged, with some enlarged as much as five-fold; by contrast, photoreceptor and blood vessel layers had less disruptions. According to the release, this suggested to the researchers that RPE disruptions may play a significant role in the disease.

“It’s not obvious at first, but using an existing tool in the clinic, we can monitor and track the cellular status of the RPE layer,” said Tam in the release. “This could prove valuable in identifying which patients would benefit the most from therapeutic interventions.”

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A combination of eye imaging techniques and adaptive optics has revealed key details of choroideremia.

Novel Imaging Approach Reveals Key Details About Rare Eye Disease

Actylis, a global manufacturer and sourcing expert of raw materials and performance ingredients, announced its debut on Sept. 26, 2022. The new company combines Aceto and 10 industry specialists into an integrated global ingredient powerhouse; A&C, A&C Bio Buffer, Biotron Laboratories, Cascade Chemistry, Finar, Inter-Actifs, IsleChem, Pharma Waldhof, Syntor Fine Chemicals, and Talus.

According to a company press release, Actylis aims to address the major unmet need for better and more dependable access to critical raw materials and performance ingredients needed for the manufacture of highly regulated products in key industries. The combination of the many specialists will enable Actylis’ hybrid manufacturing and sourcing model, which is designed to provide various benefits to its customers in high-growth end markets.

“Actylis unites multiple industry specialists with a wide range of capabilities into a new, global enterprise with a unique hybrid approach that is greater than the sum of its parts,” said Gilles Cottier, CEO of Actylis, in the release. “This consolidation enables us to offer customers across diverse locations and industries highly flexible, customized solutions addressing their specific needs, while assuring reliable on-time delivery of the high-quality ingredients essential to their success.”

Actylis combines Aceto and its 10 acquired specialists into a singular global brand.

Actylis Debuts as an Integrated Global Specialty Ingredients Manufacturing and Sourcing Provider

Octagon Therapeutics, a preclinical-stage biotechnology company, announced a partnership with Novo Nordisk on Sept. 20, 2022. The research collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.

The program selectively targets a population of pathologically activated B cells that contribute to autoimmune diseases like systemic lupus and vasculitis. According to a company press release, this approach can be applied in other disease areas to target disease-driving processes while retaining healthy immune functioning. A recent pilot project, initiated through Novo Nordisk’s Co-creation Greenhouse Accelerator program, demonstrated proof-of-concept in identifying atypical characteristics of other immune cells during disease; this ultimately led to the discovery of new therapeutic targets.

"Octagon has a unique platform technology to look into functional biology in a novel way.It makes use of clinical samples and primary immune cells to identify new drug targets,” said Uli Stilz, vice-president, Bio Innovation Hub, Novo Nordisk, in the press release. “Combined with our disease understanding in the cardiometabolic space and Octagon´s approach in targeting specific lymphocyte populations that drive disease progression, it will be exciting to see what therapeutic discoveries the collaboration can lead to."

"Novo Nordisk has a strong expertise in diabetes and related cardiometabolic conditions like [nonalcoholic fatty liver disease] and kidney disease." said Isaac Stoner, CEO, Octagon Therapeutics, in the press release."This collaboration will enable Octagon to expand our technology into these disease areas and to further leverage the impressive clinical sample collection that Novo Nordisk has available."

The collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.

Novo Nordisk and Octagon Therapeutics Enter Inflammatory Disease Research Collaboration

Alcami, a US-based pharmaceutical contract development and manufacturing organization (CDMO), announced the addition of a 12,000 ft² solid-state characterization laboratory to its Morrisville, NC laboratory on Sept. 22, 2022. According to a company press release, the expanded laboratory is designed to increase the company’s NC footprint and complement the company’s existing sterile and oral solid drug product development, manufacturing, and analytical capabilities for products in all phases of development.

"We look forward to supporting our clients in the discovery of novel polymorph, salt, and cocrystal forms and in the development of robust crystallization and formulation processes," said Elliott Franco, vice-president, Laboratory Operations, Alcami, in the press release. "We have the technical expertise and technologies to provide our clients with a seamless transition from initial characterization through [good manufacturing practice] manufacturing and analytical release.”

The dedicated solid-state research and development suite is designed to promote discovery of novel polymorph, salt, and cocrystal forms.

Alcami Adds Solid-State Characterization Capabilities to North Carolina Laboratory

Pfizer announced on Sept. 22, 2022 an agreement with Global Fund to supply up to 6 million treatment courses of its COVID-19 oral treatment, PAXLOVID as part of its COVID-19 Response Mechanism (C19RM). The C19RM has been the primary vessel for providing grant support to low-and-middle-income countries to purchase COVID-19 tests, treatments, and personal protective equipment.

PAXLOVID treatment courses will be available through C19RM, subject to local regulatory approval by the 132 grant-eligible countries determined by Global Fund based on income classification and disease burden. According to a company press release, Pfizer anticipates supply to be available beginning in 2022, pending regulatory authorization and based on country demand. All low and lower-middle-income countries will pay a not-for-profit price, while upper-middle-income countries will pay the price defined in Pfizer’s tiered pricing approach.

“After so much disruption and loss due to COVID-19, we must continue to accelerate access to PAXLOVID as a treatment option for high-risk patients in all regions of the world along with test and treat programs that help get treatment quickly to those in need”, said Albert Bourla, chairman and CEO, Pfizer, in the press release. “This agreement with Global Fund is a critical step that will boost equitable access for high-risk patients in low-and-middle-income countries.”

Pfizer is set to supply global fund up to 6 million treatment courses of its COVID-19 oral treatment, PAXLOVID, for low-and-middle-income countries.

Pfizer to Supply up to 6 Million PAXLOVID Treatment Courses for Low-and-Middle-Income Countries

Roche announced on Sept. 19, 2022 that the European Commission (EC) has approved Vabysmo (faricimab) for the treatment of neovascular or “wet” age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME). These conditions are two of the leading causes of vision loss worldwide, affecting more than 40 million people, according to a company press release.

Faricimab is currently the only injectable eye medicine approved in Europe. The treatment will begin at intervals of up to four months for people with nAMD and DME, with the potential to require fewer eye injections over time. The treatment is a bispecific antibody designed to target and inhibit two disease pathways to restore vascular stability.

“Many people with nAMD and DME struggle to keep up with the monthly eye injections and physician visits, often associated with current standards of care, and unfortunately their vision may suffer as a result of undertreatment,” said Ramin Tadayoni, head of the ophthalmology department, Lariboisière, Saint-Louis and Rothschild Hospitals, Paris, France, and European Society of Retina Specialists (EURETINA) president elect, in the release. “For people in Europe living with these conditions, today’s approval offers the first new mechanism of action in over a decade; one which could improve and protect their vision with fewer injections over time.”

EC has approved Roche’s Vabysmo, the first bispecific antibody for the eye, for two leading causes of vision loss.