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.

On May 31, 2022 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company that aims to develop the world’s most potent vaccines, reported updated overall survival (OS) results from its Phase 1/2 study evaluating GRANITE, an individualized vaccine-based immunotherapy, to treat advanced solid tumors (Press release, Gritstone Oncology, MAY 31, 2022, View Source [SID1234615261]). Additionally, the company announced it is presenting a "Trial in Progress" poster summarizing the Phase 2/3 GRANITE-CRC-1L- study (randomized study for first-line maintenance treatment of metastatic, microsatellite stable colorectal cancer) at the 2022 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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"The updated OS data from our Phase 1/2 study continue to demonstrate a correlation between molecular response and overall survival, and points to the potentially significant impact GRANITE is having on lives of patients with advanced stage cancer," said Andrew Allen, M.D., Ph.D., Co-founder, President and Chief Executive Officer of Gritstone. "The median overall survival observed in multiple trials of various therapies in the third-line MSS-CRC setting has been around 6-7 months, which is consistent with what we observed in our study in patients who did not have molecular response (median overall survival of 7.8 months). The very different outcome observed in the molecular responder subset, with median overall survival of over 18 months, is all the more striking given how rarely long-term survival is observed in MSS-CRC patients who have been treated with and progressed on two prior therapies. These maturing data, demonstrating long-term clinical benefit, further support moving GRANITE upstream in the treatment of patients with this grim cancer in the ongoing randomized Phase 2/3 study, which has registrational intent."

The Phase 1/2 study is evaluating the safety, immunogenicity, and clinical activity of GRANITE in combination with PD-1 checkpoint inhibitor, Opdivo (nivolumab) and subcutaneous anti-CTLA-4 antibody, Yervoy (ipilimumab) in advanced solid tumors. This study enrolled and treated 26 patients as of ESMO (Free ESMO Whitepaper) 2021 presentation with previously treated, metastatic solid tumors including patients with colorectal cancer, gastroesophageal adenocarcinoma, and non-small cell lung cancer. As presented at ESMO (Free ESMO Whitepaper) 2021, of 9 patients with MSS-CRC who were treated and evaluable for molecular response, 4 experienced a molecular response (as evidenced by a reduction in circulating tumor DNA [ctDNA]) and continue to have an OS advantage compared to those patients who did not have a molecular response.

Opdivo and Yervoy are trademarks of Bristol-Myers Squibb Company.

Updated overall survival data from GRANITE Phase 1/2:

4 of 9 treated patients with MSS-CRC had a molecular response (as reported during the ESMO (Free ESMO Whitepaper) 2021 data presentation) and the observed median overall survival in this group will now exceed 18 months (median OS not yet reached versus 7.8 months in those who did not have a molecular response).
All patients with MSS-CRC assessed for molecular response and alive at the time of the ESMO (Free ESMO Whitepaper) 2021 data presentation remain alive after an additional 35 weeks of follow-up.
ASCO presentation details are as follows:

Abstract TPS3635: Phase 2/3, randomized, open-label study of an individualized neoantigen vaccine (self-amplifying mRNA and adenoviral vectors) plus immune checkpoint blockade as maintenance for patients with newly diagnosed metastatic colorectal cancer (GRANITE)
Date/Time: Saturday 04 June 2022, 8:00am CST
Session: Gastrointestinal Cancer – Colorectal and Anal
Location: Poster 425b

On May 31, 2022 Shasqi, a clinical-stage biotechnology company developing precision activated oncology therapeutics with its proprietary Click Activated Protodrugs Against Cancer (CAPAC) platform, reported that it will present interim data from its Phase 1 clinical study of SQ3370 in advanced solid tumors, at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held at McCormick Place in Chicago, IL, on June 3-7, 2022 (Press release, Shasqi, MAY 31, 2022, View Source [SID1234615278]).

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Presentation details:

Abstract Title: Interim Phase 1 results for SQ3370 in advanced solid tumors
Abstract Number: 3085
Poster Number: 77
Poster Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Session Date and Time: Sunday, June 5, 2022, 8:00 AM-11:00 AM CDT
Key highlights include:

26 patients received treatment with SQ3370 and as of the data cutoff date, 21 patients were evaluable
77% of patients had metastases with a median number of 2 metastatic sites (1-5); most frequently lung (50%) and more than 50% being previously treated with doxorubicin
Escalating doses of SQP33 protodrug ranging from 0.38x to 12x the molar equivalent of conventional doxorubicin per cycle along with a dose of 10 or 20 mL of SQL70 were administered
SQ3370 was well tolerated with 62% of patients receiving more than 500 mg/m2 cumulative doxorubicin equivalents given as SQP33
The most common treatment emergent adverse events reported were nausea, fatigue, anemia, and constipation
At a median follow-up of 9.2 weeks, of the 21 evaluable patients, 71% had stable disease, with a median duration of 80 days
Dose escalation continues as the maximum tolerable dose has not yet been reached
SQ3370 demonstrates proof of concept for the CAPAC platform
About CAPAC and SQ3370

SQ3370 is the first click chemistry-based treatment to be tested in humans. It utilizes Shasqi’s proprietary CAPAC platform, an approach that activates cancer drugs at a tumor with decreased systemic toxicity. Shasqi is validating its platform with SQ3370, which is designed to activate a powerful chemotherapeutic, doxorubicin, at the tumor site. The investigational product is based on the chemical reaction between a drug protected through a trans-cyclooctene modification (a protodrug) and a tetrazine-modified biopolymer. The biopolymer is injected into the target tumor lesion, where it precisely activates an intravenously infused protodrug. Shasqi believes its click-chemistry approach can improve the efficacy and safety of many existing therapeutics across various modalities with a limited therapeutic window.

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 InveniAI LLC, a global leader in applying Artificial Intelligence (AI) and Machine Learning (ML) to transform drug and target discovery and development, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151), a global specialty pharmaceutical company creating innovative medical solutions utilizing the latest biotechnology, reported the achievement of the first milestone in disease model systems that triggered a milestone payment that has been received by InveniAI under the research collaboration between the companies (Press release, Kyowa Hakko Kirin, MAY 31, 2022, View Source [SID1234615262]).

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Yoshifumi Torii, Ph.D., Executive Officer, Vice President, and Head of R&D Division of Kyowa Kirin, added, "We are excited to achieve a successful outcome by leveraging InveniAI’s platform AlphaMeld that detected very early signals of association between targets/mechanism of action and clinical utility based on the platform’s analytics. We have since validated this program which, as predicted, has shown signals of efficacy in preclinical proof-of-concept studies for the treatment of a disease indication where current therapeutic options lack efficacy and have adverse effects. We are eager to test this very promising candidate for further studies. We also look forward to the selection of additional novel programs identified by InveniAI’s truly game-changing platform and believe that our collaboration will contribute to addressing enduring unmet medical needs."

"We are delighted with the progress of our synergistic collaboration with Kyowa Kirin and the advancement of an AI-identified target and associated clinical utility at unprecedented speed," said InveniAI’s President and Chief Executive Officer, Krishnan Nandabalan, Ph.D., "Our AI-powered drug and target discovery platform, AlphaMeld, has repeatedly demonstrated the ability to generate concepts that have been validated by our partners from preclinical to late-stage clinical success. The ability to translate data into meaningful efficacy, safety, and clinical endpoints is pushing the current probability of clinical success from 1 in 10 to 3 in 10 – a significant impact in today’s drug discovery and development paradigm," he added.

InveniAI and Kyowa Kirin have been collaborating since 2018 and have expanded the framework of their collaboration twice so far (2020 and 2021)*. This milestone achievement is the result of the first joint research to maximize the portfolio value that began in 2018. In addition to the above-mentioned projects, there are ongoing projects aimed at discovering new drug candidates in several disease areas.

About AlphaMeld
AlphaMeld is an Artificial Intelligence (AI) and Machine Learning (ML) powered platform that accelerates innovation for the discovery of targets, drugs, and healthcare products and technologies. The platform generates testable hypotheses by taking into account the ideal mode of pharmacotherapy (antibody, protein replacement, siRNA, mRNA, small molecule, cell and gene therapy, and gene-editing modalities), disease severity, gene ontology, disease pathways, proteinopathies, standard of care, emerging innovation, and enabling technologies while factoring in medical, scientific, strategic, and commercial considerations.