Pharmaceutical Technology Editors

Avance Clinical, an Australia-based contract research organization (CRO) for biotechs, announced on Sept. 6, 2022 that it has expanded its services into North America with the acquisition of C3 Research Associates, a US-based CRO partner company. The acquisition will enable Avance to offer biotech clients an easier transition from early phase to later phase studies.

With the acquisition, Avance will have the capability to progress early phase studies in Australia and New Zealand into the United States, allowing biotech clients to continue their clinical development programs while retaining the same CRO services. This efficiency would reduce time and cost and allow for biotech companies to deliver quality data that will be acceptable to FDA.

The company intends to make significant investments in its US operations, including growing its team to more than 120 people and leveraging deep industry experience across a broad range of therapeutic areas, specifically oncology, according to a company press release.

“Avance Clinical has been working with C3 Research Associates in recent years, and they share a similar culture and focus on customer service and clinical excellence. This strong relationship made C3 Research Associates a natural acquisition choice for Avance Clinical’s expansion plans into North America. The due diligence process has taken more than [six] months, and we are excited to now welcome our US team and offer the GlobalReady model for our clients,” said

Yvonne Lungershausen, CEO of Avance Clinical, in the press release.

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Avance Clinical’s acquisition of C3 Research Associates expands its CRO services to North America.

Avance Clinical Acquires C3 Research Associates

ElevateBio, a biotechnology company specializing in cell and gene therapies, recently announced a long-term strategic partnership with the University of Pittsburgh to accelerate the development of innovative cell and gene therapies. Under the agreement, ElevateBio will locate one of its next BaseCamp process development and good manufacturing practice manufacturing facilities, the Pitt BioForge at Hazelwood Green, in Pittsburgh.

According to an Aug. 25, 2022 company press release, the new facility will be equipped with gene editing, induced pluripotent stem cell, cell, vector, and protein engineering capabilities. The university was awarded a $100 million grant by the Richard King Mellon Foundation in November 2021 to support creation of the facility. The facility is expected to generate 170 full-time jobs, 900 construction jobs, and 360 off-site support jobs.

“This announcement supports the region's rise as a leader in cell and gene therapy and advances our vision of bringing an entirely new commercial manufacturing sector to the area," said Patrick Gallagher, chancellor, University of Pittsburgh, in the press release. "The University of Pittsburgh is proud to partner with ElevateBio in this work, which will see us leveraging lessons from the lab—in new and exciting ways—for the benefit of human health.”

“To realize our vision of transforming the cell and gene therapy field for decades to come, broadening our footprint across metropolitan areas is a key priority for us, and we are thrilled that the University of Pittsburgh will be home to one of our BaseCamp facilities,” said David Hallal, chairman and CEO, ElevateBio, in the press release. “We’ve identified Pittsburgh as an ideal location to extend our BaseCamp presence as it sits at the intersection of science, technology, and talent. We are grateful for the support of the Governor and County Executive as we bring the first-of-its-kind offering we have built at ElevateBio BaseCamp to advance the work of the entire biopharmaceutical industry.”

In partnership with the University of Pittsburgh, ElevateBio will open its next manufacturing center in the city.

ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center

Good Therapeutics, a privately held biopharmaceutical company, announced on Sept. 7, 2022 that it has entered into a definitive merger to be acquired by Roche for $250 million, with additional funding contingent on predetermined development, regulatory, and commercial milestones. The deal is expected to close in the third quarter of 2022.

This acquisition grants Roche the rights to Good Therapeutic’s conditionally activated programmed cell death-1 regulated interleukin-2 (PD-1-regulated IL-2) program as well as the exclusive right to the platform technology for the development of PD-1-regulated IL-2 receptor agonist therapeutics. According to a company press release, the current team at Good Therapeutics plans to form a new company, Bonum Therapeutics, to design further conditionally active therapeutics in immuno-oncology and other fields.

“Good Therapeutics was founded to create a new class of conditionally active therapeutics that will be more effective and avoid the problem of systemic immune activation seen with previous versions of such drugs. We have focused on PD-1-IL-2 as a biology that has great potential for benefiting patients,” said John Mulligan, founder and CEO, Good Therapeutics, in the press release. “Roche is a leader in immuno-oncology and has pioneered the field of engineered PD-1-targeted IL-2 therapeutics. Given their expertise in this field and broad capabilities in oncology, we believe they are a perfect choice for taking this important program forward.”

“We are excited to bring Good’s innovative PD-1-regulated IL-2 program into our existing oncology pipeline, which nicely complements our efforts on next-generation PD-1-targeted IL-2 therapeutics and our broader oncology strategy of providing cancer patients with innovative solutions and improving health outcomes,” said James Sabry, global head of pharma partnering, Roche, in the press release.

Roche’s acquisition of Good Therapeutics grants them rights to the company's conditionally activated PD-1-regulated IL-2 program.

Roche to Acquire Good Therapeutics for $250 Million

SIRIO Pharma (SIRIO), a global nutraceutical contract development and manufacturing organization (CDMO), announced on Sept. 9, 2022 that it has appointed Sara Lesina as a new general manager in Europe. Lesina will seek to drive forward a strategy of targeting small and medium brands launching novel nutraceutical formulations.

Lesina previously worked at Lonza as executive director. According to a company press release, Lonza appointed Lesina to further its goal of nurturing new partnerships with large nutrition brands across the major European markets.

“I joined SIRIO because I see the tremendous resources it has – SIRIO has over 200 R&D scientists across its sites – and there are clear synergies with like-minded innovation focused customers,” commented Lesina in the press release. “We anticipate significant growth in the nutra market in next few years as customers see the benefits of working with an agile CDMO partner to help launch products to market faster. The SIRIO value proposition anchored on R&D and formulation capabilities, combined with our global manufacturing footprint, positions us well to tap into this growth by launching new products for customers.”

“Sara brings with her a tremendous global network and what impressed us is the clarity of purpose to establish SIRIO as the contract research and development hub for the most ambitious global brands,” added Rui Yang, vice-president at SIRIO. “Across Europe there is an increasing number of companies launching new products that could reduce their time to market and/or acquisition by working with SIRIO. Sara’s remit will be to help develop locally relevant formulatory options, working as the European conduit to access SIRIO’s global R&D resources.”

SIRIO is enhancing its strategy for small and medium nutrition brands as Lonza’s Sara Lesina joins as GM in Europe.

SIRIO Enhances Company Strategy with Addition of Lonza’s Sara Lesina

AstraZeneca announced on Sept. 6, 2022 that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Lynparza (Olaparib) in Great Britain for the adjuvant treatment for adult patients with germline BRCA-mutated HER2-negative high-risk early breast cancer. The treatment may be used as monotherapy or in combination with endocrine therapy and is for use in patients who have been previously treated with neoadjuvant or adjuvant chemotherapy.

According to a company press release, an estimated 2.3 million patients are diagnosed with breast cancer each year, making it the most diagnosed cancer worldwide. Almost 56,000 people are diagnosed with breast cancer per year in the United Kingdom, and roughly 90% of all breast cancer patients are diagnosed in an early stage of disease. BRCA mutations are found in approximately 5% of patients. One in three women may still experience a recurrence, and if the cancer recurs, it is incurable.

The decision from the MHRA was based on results from the OlympiA Phase III trial. In the primary endpoint of the trial, there was a statistically significant and clinically meaningful improvement in three-year invasive disease-free survival (iDFS), reducing the risk of invasive breast cancer recurrences, second primary invasive cancers, or death.

“Today’s approval marks a new era of care in the UK for patients with an inherited form of breast cancer,” said Andrew Tutt, chair of the OlympiA Phase III trial and professor of oncology at The Institute of Cancer Research, London and King’s College London, in the press release. “For patients with high-risk early-stage breast cancer, including those with germline BRCA mutations, recurrence rates remain unacceptably high, with more than one in four of these patients seeing their cancer return following surgery and systemic treatment. Olaparib is a targeted treatment option that exploits the specific biology of this inherited type of breast cancer and was shown to improve survival and help prevent cancer recurrence. I am hopeful it will become a new standard of care.”

Lynparza has been approved in Great Britain as an adjuvant treatment for patients with germline BRCA-mutated HER2-negative high-risk early breast cancer.

Lynparza Approved in Great Britain

Sanofi announced on Aug. 31, 2022 that FDA approved its drug, Xenpozyme (olipudase alfa-rpcp), for treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. According to a company press release, olipudase alfa-rpcp is the first therapy indicated specifically for the treatment of ASMD and the only approved treatment for the disease.

ASMD, which represents a spectrum of disease, is classified into three categories: type A, type B, and type A/B. Type A and type B sit on opposite ends of the spectrum, whereas A/B is an intermediate form. The condition can result in an enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other symptoms. According to the release, fewer than 120 patients are diagnosed with ASMD in the United States; approximately 66% of these patients are pediatric.

Xenpozyme is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy that is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme allowing for the breakdown of the lipid sphingomyelin. In individuals with ASMD, the deficiency in the ASM enzyme leads to sphingomyelin accumulation in various tissues. The drug is administered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase.

Prior to its approval, Xenpozyme received breakthrough therapy and priority review designations. According to the release, FDA based its approval on results from two clinical trials (ASCEND and ASCEND-Ped), which demonstrated clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide) and platelet count as well as reduced liver and spleen volumes. Japan and the European Union also approved the treatment in March and June of 2022, respectively.

“Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families,” said Bill Sibold, vice-president and head of Specialty Care, Sanofi, in the press release. “This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and our unwavering commitment to this historically overlooked community.”

Sanofi’s Xenpozyme (olipudase alfa-rpcp) is now the only approved treatment for acid sphingomyelinase deficiency.