Pharmaceutical Technology Editors

Polaryx Therapeutics, a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced that FDA granted orphan drug designation for PLX-200 to treat Krabbe disease on Sept. 2, 2021.

Krabbe Disease is a rare genetic disorder caused by a deficiency in a lysosomal enzyme called galactocerebrosidase (GALC). When GALC is dysfunctional or has a reduced presence in the body, it causes galactosylsphingosine to accumulate in the central and peripheral nervous systems. This results in demyelination and can lead to death in affected children within the first two years of life.

PLX-200 is a drug that binds to the retinoid X-receptor-α, which binds to PPARα. It activates PPARα, which results in enhanced production of transcription factor EB (TFEB). TFEB then binds to a gene promoter involved in lysosome biogenesis, facilitating their production.

“Because supportive care is the only available treatment option for most cases of Krabbe disease, this designation validates our scientific rationale and strongly motivates us to expedite the clinical development of PLX-200 in Krabbe disease.” said an-Jun Lee, president and CEO of Polaryx, in a company press release. “We are moving forward with the necessary development steps to move into the clinical study as soon as possible.”

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Orphan drug designation for Polaryx’s PLX-200 will facilitate research into its use treating Krabbe Disease, a rare lysosomal genetic disorder.

Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 for Treatment of Krabbe Disease

Thermo Fisher Scientific announced on Sept. 2, 2021 that it was awarded a $192.5 million contract by the US Department of Defense to expand domestic manufacturing of pipette tips to ensure reliable supply. There have been reports of 

 hindering bio/pharmaceutical activities. The award was issued on behalf of and in coordination with the US Department of Health and Human Services.

Under the contract, Thermo Fisher will co-invest with the US government to build a new energy-efficient manufacturing facility for pipette tips used in disease research and in diagnostic tests, including COVID-19. The new center will be housed in North Carolina and will be designed in line with Thermo Fisher's carbon-neutral goals. Construction is expected to be completed by the third quarter of 2024, but pipette tip manufacturing is expected to start as early as the third quarter of 2023.

The company had also announced its own investments in mid-2020 to increase pipette tip production capacity. That expansion was to support COVID-19 testing. Since the start of the pandemic, Thermo Fisher has invested more than $180 million in expanding its laboratory plastics consumables production as well as another $600 million to increase global bioprocessing capacity. The company has also opened new sites dedicated to manufacturing viral transport media used in COVID-19 sample collection and current good manufacturing practice plasmid DNA used in messenger RNA-based vaccines.

"This award and the resulting capacity ensure that future demand surges in the [United States], from the current COVID-19 pandemic to the next crisis, will be met with greater supply assurance," said Mark Stevenson, executive vice-president and chief operating officer of Thermo Fisher, in a company press release. "Supply chain agility is critical to our customers, our government, and to the health and safety of our citizens, and we're excited to bring this state-of-the-art capacity online, especially since its efficient design will also showcase our commitment to achieve net-zero carbon emissions by 2050."

The US government has granted Thermo Fisher Scientific a $192.5 million contract that the company will use to expand domestic manufacturing for pipette tips.

Thermo Fisher Scientific to Expand Pipette Tip Manufacturing Under $192.5 Million Government Contract

Diffusion Pharmaceuticals, a biopharmaceutical company developing novel oxygenation therapies, announced on Aug. 30, 2021 that they had obtained investigational new drug (IND) clearance from FDA’s Pulmonology, Allergy, and Critical Care division on their lead product candidate, trans sodium crocetinate (TSC).

Diffusion now has four open INDs related to TSC among select FDA divisions. In addition to the new clearance, there are existing ones from the Cardiology and Nephrology, Neurology, and Oncology divisions.

The trial that will be conducted under the new IND will be a Phase II, double-blind, randomized, placebo-controlled study that will evaluate the effects of TSC on the diffusion of carbon monoxide through the lungs (DLCO) in patients with Interstitial Lung Disease (ILD).

The ILD-DLCO trial will be the third of Diffusion’s three oxygenation trials, which were designed to assess the ability of TSC to enhance oxygen uptake, peripheral oxygenation, and consumption. Their results will inform the selection of TSC’s dosing regimen and initial lead indication, which Diffusion expects will be selected by the end of 2021 and initiated in the first half of 2022, according to a company press release.

“The clearance of our new IND within the Pulmonary Division is a great milestone for Diffusion and TSC,” said Chris Galloway, chief medical officer of Diffusion, in the press release. “Not only does it give us the ability to conduct the ILD-DLCO trial in the US, but also expands our potential regulatory opportunities across multiple divisions at the FDA encompassing numerous therapeutic areas.”

FDA clearance of trans sodium crocetinate allows Diffusion to proceed with their latest Phase II oxygenation trial.

Diffusion Pharmaceuticals Announces FDA Clearance of IND Application for TSC

Element Material Technology announced on Sept. 2, 2021, that it has acquired Impact Analytical. This move is the latest in a series of acquisitions made by Element to expand their life sciences footprint in North America, according to a company press release.

Based in Midland, Michigan, Impact operates in the pharmaceutical, medical device, consumer product, specialty chemicals, and most recently bio-analytics segments. In particular, the company specializes in extractables and leachables.

Element’s acquisition of Impact will add various testing services to their pharmaceutical, medical device, environmental, and food segments. This is intended to complement the life science capabilities the company added with the acquisitions of ALG and Avomeen in the last twelve months, according to the press release.

“As our eighth acquisition this year and our fourth in the life sciences market, we are pleased to welcome Impact Analytical to Element,” said Rick Sluiters, executive vice-president of Americas at Element, in the press release. “With ALG and Avomeen in America and the recent announcement of our acquisition of Arch Sciences Group in Europe, Element has significantly strengthened its position in the life sciences sector.

Element’s acquisition of Impact Analytical expands their life sciences footprint in North America.

Element Acquires Impact Analytical

Immutep, an Australian-based biotechnology company specializing in immunotherapy, announced receipt of a patent from the Chinese Patent Office on Aug. 27, 2021 for LAG525, a lymphocyte-activation gene 3 (LAG-3) antagonist antibody. The Chinese patent follows corresponding patents in Australia, the United States, Europe, and Japan, which were granted in 2018 through 2020.

The patent claims are specifically directed to LAG525, pharmaceutical compositions comprising LAG525, nucleic acid molecules that code for the LAG525 antibody, an expression vector or host cell that comprises the nucleic acid molecules, and to the use of LAG525 in the manufacture of a preparation for the treatment of cancer or infectious disease.

LAG525 (leramilimab) is a humanized form of Immutep’s IMP701 antibody, which has been out-licensed to Novartis. Novartis and Immutep co-own the patent, which will expire on March 13, 2035.

The antibody plays a role in controlling signaling pathways in both effector T cells and regulatory T cells (Treg). The antibody activates effector T cells by blocking inhibitory signals that would otherwise switch them off. The antibody also inhibits Treg function that normally prevents T cells from responding to antigen stimulation. Both mechanisms of action prevent the immune system from responding to and killing cancer cells. Other antagonist LAG-3 antibodies in development target only the effector T-cell pathway but don’t address the Treg pathway.

LAG525 is being evaluated in several Phase I and/or Phase II clinical trials in combination with spartalizumab, Novartis’ programmed cell death 1 (PD1) inhibitor to treat various cancers. Under an agreement between the companies, Novartis has full responsibility for the continued development of the antibody program, and Immutep is eligible to receive development-based milestone payments and royalties on sales following commercialization of the antibody.

Immutep was granted a Chinese patent for LAG525, a LAG-3 antagonist antibody, under evaluation for cancer treatment.

Immutep Granted a Chinese Patent for LAG-3 Antagonist Antibody

PharmaceuticsInternational, Inc. (Pii) announced in an Aug. 31, 2021 press release that Roberto Almodóvar Febles has been hired as their Head of Aseptic Operations. In this role, Febles will oversee and manage day-to-day aseptic manufacturing. He will work closely with Sam Chia, the head of aseptic manufacturing science and technology, as well as the operations leadership team members.

Prior to joining Pii, Febles worked with Baxter Healthcare, Amgen, iPR Pharmaceuticals (the CMO for AstraZeneca), and Mylan. He earned a Bachelors of Science in both Chemistry and Chemical Engineering at the University of Puerto Rico, Mayagüez Campus, and an Executive Master degree in Business Administration from the Interamerican University of Puerto Rico, Cupey Campus.

“Roberto is an experienced manufacturing leader with more than 15 years of progressive experience in aseptic manufacturing as an operator, supervisor, and manager,” said Kurt Nielsen, CEO of Pii, in a company press release. “I am confident Roberto and the other teams will work to successfully deliver on-time, and right first time, for our clients."

Febles will oversee day-to-day aseptic manufacturing at Pharmaceutics International, Inc.