On May 31, 2022 ORPHELIA Pharma, the French biopharmaceutical company focused on developing and commercializing orphan medicines in oncology and neurology, reported the successful completion of the phase 1 study of KIMOZO, novel ready-to-use oral suspension of temozolomide, aimed at determining bioequivalence between KIMOZO suspension and temozolomide capsules (Press release, ORPHELIA Pharma, MAY 31, 2022, View Source;utm_medium=rss&utm_campaign=orphelia-reports-positive-results-of-the-bioequivalence-study-of-kimozo-oral-suspension-of-temozolomide [SID1234615249]).

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The abstract #368704 entitled "bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary central nervous system malignancies" by Ducray et al., was selected by the Scientific Program Committee of the 2022 ASCO (Free ASCO Whitepaper) annual meeting. The data released online demonstrate the equivalence between KIMOZO oral suspension and temozolomide capsules.

"The positive results of the bioequivalence study are an important milestone in the development of KIMOZO", comments Caroline Lemarchand, Chief Pharmaceutical Development Officer at ORPHELIA Pharma. "KIMOZO is the first drinkable form of temozolomide that has been specifically developed to address the needs of young children with cancer", she adds.

"Temozolomide is part of the standard of care for the treatment of relapsed or refractory neuroblastoma, a devastating cancer that affects young children. KIMOZO is currently undergoing further clinical evaluation (TEMOkids trial, NCT04610736) and our goal is to make this pediatric formulation of temozolomide available to children in the shortest timeframe possible"; concludes Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma.

About the bioequivalence study (NCT04467346)

The NCT04467346 bioequivalence study was an open label, randomized, crossover, 2-period study in 30 adult patients with primary CNS malignancies. The study objective was to evaluate primarily the bioequivalence between KIMOZO oral suspension and TEMODAL capsules, to define KIMOZO pharmacokinetic parameters and to assess the safety and tolerability including buccal tolerance of KIMOZO.

On May 31, 2022 GSK plc (LSE/NYSE: GSK) reported that it has entered into a definitive agreement to acquire Affinivax, Inc. (Affinivax), a clinical-stage biopharmaceutical company based in Cambridge, Boston, Massachusetts, for a $2.1 billion upfront payment and up to $1.2 billion in potential development milestones (Press release, GlaxoSmithKline, MAY 31, 2022, View Source [SID1234615247]). Affinivax is pioneering the development of a novel class of vaccines, the most advanced of which are next-generation pneumococcal vaccines.

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Pneumococcal disease includes pneumonia, meningitis, bloodstream infections, and milder diseases such as sinusitis and otitis media and remains a significant unmet medical need despite the availability of current pneumococcal vaccines. There are many different pneumococcal serotypes. The number of serotypes in current vaccines is limited due to the degree of immunological interference observed when using existing conjugation technologies.

Affinivax has developed the Multiple Antigen Presenting System (MAPS), a novel technology that supports higher valency than conventional conjugation technologies, enabling broader coverage against prevalent pneumococcal serotypes and potentially creating higher immunogenicity than current vaccines. Affinivax’s most advanced vaccine candidate (AFX3772) includes 24 pneumococcal polysaccharides plus two conserved pneumococcal proteins (compared to up to 20 serotypes in currently approved vaccines). A 30-plus valent pneumococcal candidate vaccine is also in pre-clinical development.

Dr Hal Barron, Chief Scientific Officer and President R&D, GSK, said: "The proposed acquisition further strengthens our vaccines R&D pipeline, provides access to a new, potentially disruptive technology, and broadens GSK’s existing scientific footprint in the Boston area. We look forward to working with the many talented people at Affinivax to combine our industry-leading development, manufacturing, and commercialisation capabilities to make this exciting new technology available to those in need."

In the adult phase I/II clinical trials, AFX3772 was well tolerated in participants and demonstrated good immune responses compared to the current standard of care. In July 2021, the US Food and Drug Administration granted Breakthrough Therapy designation for AFX3772 to prevent S. pneumoniae invasive disease and pneumonia in adults 50 years and above. Phase III is expected to start in the short term. Phase I/II clinical trials to assess the use of the vaccine in paediatrics are planned to begin later this year.

"Affinivax grew out of our founders’ scientific and personal vision to drive vaccine innovation to make a meaningful impact on people’s lives, in both developed and developing countries," said Steven Brugger, CEO of Affinivax, Inc. "Over the past eight years, we have taken that vision from the initial development of our MAPS vaccine platform at Boston Children’s Hospital to a pipeline of novel vaccines with our lead vaccine candidate in late-stage clinical studies. We are proud that GSK has recognized our team’s accomplishments and are confident that GSK is an ideal new home for our MAPS platform and the team behind its success. GSK’s significant capabilities will enable continued advances with MAPS to improve existing vaccines – as is the case with our lead Streptococcus pneumoniae MAPS vaccine program – and develop vaccines that combat novel and resistant infectious diseases for which there are no effective immunization strategies available today."

Financial considerations

Under the terms of the agreement, GSK will acquire 100% of the outstanding shares of Affinivax. The consideration for the acquisition comprises an upfront payment of $2.1 billion to be paid upon closing and two potential milestone payments of $0.6 billion to be paid upon the achievement of certain paediatric clinical development milestones. The transaction is subject to customary closing conditions, including the expiration or early termination of the waiting period under the Hart-Scott- Rodino Anti-Trust Improvements Act of 1976. The transaction is expected to close in the third quarter of 2022.

GSK will account for the transaction as a business combination.

New GSK reaffirms its full-year 2022 guidance and the medium-term outlook for 2021-2026 of more than 5% sales and 10% adjusted operating profit CAGR* at CER**.

* CAGR: Compound Annual Growth Rate; **CER: Constant Exchange Rate

Pneumococcal disease

The bacterium Streptococcus pneumoniae causes pneumococcal disease. There are multiple types of pneumococcal disease, including bloodstream infections (sepsis), pneumonia, meningitis, and other milder diseases such as sinusitis and otitis media. A significant unmet medical need remains despite the availability of current vaccines. Pneumococcal pneumonia causes an estimated 150,000 hospitalisations each year in the United States; pneumococcal meningitis and bacteremia killed approximately 3,250 people in the United States in 2019[i].

MAPSTM technology platform

Multiple Antigen Presenting System (MAPS) is a novel and highly efficient vaccine technology platform that enables the precise, high-affinity binding of disease-relevant polysaccharides to disease-relevant protein antigens in a single vaccine. Immunisation with the resulting polysaccharide-protein complexes induces a broad and potentially protective B-cell (antibody) response to the polysaccharides and a separate B-cell and T-cell immune response to the proteins. The distinctive plug-and-play nature of MAPS enables the targeting of a broad range of infectious diseases.

The initial use of the technology has been directed primarily toward preventing pneumococcal disease. Applicability of the technology has also been demonstrated for additional infectious disease pathogens, including those that cause hospital-acquired infections.

On May 31, 2022 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that it it will present a poster on the 4-1BB conditional agonist antibody ATOR-1017 at the 2022 ASCO (Free ASCO Whitepaper) (American Society of Clinical Oncology) Annual Meeting, being held in Chicago June 3-7 (Press release, Alligator Bioscience, MAY 31, 2022, View Source [SID1234615246]).

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The poster, entitled "Initial findings from a first-in-human, multicenter, open-label study of ATOR-1017, a 4-1BB antibody, in patients with advanced solid malignancies", outlines results from Alligator’s first-in-human clinical trial with ATOR-1017, which is being developed as a tumor-directed therapy for advanced/metastatic solid cancers.

The results, to be presented at ASCO (Free ASCO Whitepaper), demonstrate an excellent safety profile. Five (22.7%) of the 22 patients treated with ATOR-1017 experienced grade 3-4 treatment-related adverse events (TRAEs). None of the TRAEs resulted in treatment discontinuation. No dose-limiting toxicity was observed, and thus the maximum tolerated dose (MTD) of ATOR-1017 has not been reached. ATOR-1017 exhibits a dose dependent and favorable pharmacokinetic profile. Activation of peripheral T cells and increased levels of soluble 4-1BB was observed across active dose levels of ATOR-1017, demonstrating biological activity and proof of mechanism.

Stable disease was achieved as best objective response in 10 (45%) of patients, with the longest treatment duration being 16 months.

Overall, the data showed that ATOR-1017 is safe and well-tolerated at doses up to 600 mg and has shown signs of clinical benefit. Dose escalation continues at the 900 mg dose and data from this cohort is expected to be reported in 2022.

"We are excited to be able to present these very promising data at ASCO (Free ASCO Whitepaper), outlining the strong safety profile and signs of efficacy of our ATOR-1017 drug candidate," said Søren Bregenholt, PhD, CEO of Alligator Bioscience. "4-1BB antibodies have been plagued with poor efficacy or unacceptable safety profile, but ATOR-1017 is distinct from other 4-1BB antibodies, partly because of its unique binding profile but also because its immunostimulating function is dependent on cross-linking to Fc-gamma receptors on immune cells. This localizes the immunostimulation to the tumor region, where both 4-1BB and Fc-gamma receptors are expressed at high levels. This means that ATOR-1017 has the potential to address a significant unmet medical need, and we look forward to finalizing this study and selecting a recommended dose for the upcoming Phase 2 study."

The Phase I study with ATOR-1017 is an open-label, dose-escalation study in patients with histologically confirmed, advanced, and/or refractory solid cancer (NCT04144842). The primary objective of the study is to investigate the safety and tolerability of ATOR-1017 and to determine the recommended dose for subsequent Phase 2 studies.

On May 31, 2022 Selvita S.A. – [ticker: WSE: SLV] – one of the largest preclinical contract research organizations in Europe, reported its development in all business segments in the first quarter of 2022 (Press release, Selvita, MAY 31, 2022, View Source [SID1234615241]).

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The increase in the scale of operations along with high margins

In the first quarter of 2022, Selvita Group reported revenues in the amount of EUR 20.5 million, indicating an increase of 35% y/y. EBITDA and net profit (excluding the impact of the non-cash incentive program) amounted to EUR 6.0 million and EUR 3.6 million, respectively, which translates into an increase of 56% and 119%. Margins grew in line with the expanding scale of the business. The EBITDA margin increased from 25.2% in the previous year to 29.1% in 2022, while the net profit margin increased from 10.9% to 17.7%.

Services provided in Poland closed the first quarter of 2022 with revenues of EUR 9.8 million, an increase of 43% y/y. EBITDA in the reporting period amounted to EUR 2.9 million, achieving the annual growth dynamics of 110%. The segment also significantly improved the EBITDA margin, which increased from 19.0% in the previous year to 28.1% in 2022. A significant increase in the revenues in the area of ​​regulatory research was noted, from EUR 1.5 million achieved in the first quarter of 2021, to EUR 2.7 million in 2022 (+ 83% y/y).

The segment of services provided in Croatia increased the commercial revenues generated by 22% y/y, reaching EUR 7.8 million. EBITDA in the analyzed period amounted to EUR 2.4 million, showing an increase of 21% y/y. The margin on the services remained at a similar level, 31.3% in Q1 2021 vs. 31.1% in Q1 2022.

In the reporting period, Ardigen generated EUR 2.2 million in commercial revenues, compared to EUR 1.4 million last year, which translated into an improvement of 60% y / y. EBITDA increased from EUR 0.4 million to EUR 0.6 million (+44% y/y), and the EBITDA margin was 24.3%, which means a minimal decrease compared to Q1 2021 (-2 pp).

– I am very pleased with the results achieved. The high pace of growth in all our business segments, as well as the improvement in EBITDA profitability and net profit, show that dynamic development can go hand in hand with good margins. This is the result of the work of our scientists who strive to provide our clients with high quality services every day. At Selvita, we believe that people are the most important asset, and our financial results reflect this – comments Bogusław Sieczkowski, Chief Executive Officer at Selvita S.A.

The backlog of Selvita Group also grew dynamically in the reported period, and currently amounts to EUR 58.8 million, indicating an increase of 38% y/y. In the area of ​​drug discovery, backlog reached EUR 44.6 million, increasing by 30% as compared to the same period previous year. The regulatory research segment has been growing even faster, with backlog amounting to EUR 6.2 million (+ 111% y/y). Ardigen reported EUR 6.5 million of backlog, almost EUR 2.0 million more than in 2021.

– The high value of contracted orders allows us to be optimistic about the entire year 2022 – adds Sieczkowski.

New Development Strategy 2022 – 2025

– During the first quarter of this year, we worked intensively on our new strategy. Rapid development of the Group over the last years meant that our previous, four-year strategy, was implemented in a little over two years. As part of the assumptions of the new development plan, we plan to grow three times by 2025, and achieve annual revenues of EUR 200 million, while maintaining a stable, high margin. We are convinced that the implementation of these goals will allow us to become a global, preclinical CRO, offering clients an increasingly comprehensive range of services – said Sieczkowski.

In the first months of 2022, Selvita made several operational steps supporting further development of the Group. Integration of services in the area of ​​drug discovery, integration of sales and business development, as well as creation of a department supporting the management of operational activities, investments, and infrastructure, constitute a strong foundation for the implementation of the assumptions of the new strategy for 2022-2025.

On May 31, 2022 Immedica Pharma AB ("Immedica") and Japanese company OrphanPacific, Inc. ("OrphanPacific") reported that they on May 2, 2022, entered an agreement under which OrphanPacific gains the exclusive rights to Ravicti in Japan (Press release, Immedica Pharma, MAY 31, 2022, View Source [SID1234615240]).

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The pharmaceutical drug product Ravicti is approved in Europe and North America for treatment of urea cycle disorders (UCD).

Under the announced partnership, OrphanPacific is granted a license to develop, register and commercialize the product in UCD in Japan. OrphanPacific will initiate a clinical trial for obtaining approval for glycerol phenylbutyrate in Japan. OrphanPacific is already Immedica’s commercial partner in Japan for the commercialization of the drug product Buphenyl.

"It is with great pleasure that we announce this expanded partnership with OrphanPacific, under which we will be able to make Ravicti available to UCD patients also in Japan. The entered agreement also further strengthens Immedica’s geographical footprint, by introducing yet another product collaboration in Japan," says Anders Edvell, CEO of Immedica.

About Urea Cycle Disorders (UCD)

Urea cycle disorders are a group of metabolic diseases that affect a specific enzyme or transporter in the urea cycle leading to elevated ammonia or glutamine levels in the circulation. Symptoms of the disorder can begin at any age, with more severe defects beginning early in life. UCD patients may experience episodes, called hyperammonemic crises, when ammonia levels in the blood become excessively high, which can result in irreversible brain damage, coma or death. In Japan, UCDs occurs in 1 in 8,000 to 44,000 people and is one of the designated intractable diseases.