On October 27, 2022 Orion Research Foundation reported that it is awarding EUR 1 million in grants and two EUR 100,000 special grants (Press release, Orion , OCT 27, 2022, View Source [SID1234622511])

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The Board of Directors of the Orion Research Foundation will today award grants on the basis of applications amounting to a total of EUR 1 million for 2023. This year, the Board of the Research Foundation will also award two special grants of EUR 100,000 to mid-career researchers. The grants will be awarded to Associate Professor Tiina Sikanen from the Division of Pharmaceutical Chemistry and Technology at the University of Helsinki, and to Professor of Cancer Biology Jukka Westermarck from the Institute of Biomedicine and
Research Director at the Turku Bioscience Centre at the University of Turku.

Each year, the Orion Research Foundation distributes its investment income and the donation it receives from Orion Corporation as grants to young researchers and researchers who have recently gained doctorates to enable them to continue their research. In recent years, the Foundation has awarded grants by application amounting to a total of approximately EUR 1 million annually to researchers in the fields of medicine, veterinary medicine, pharmacy and related natural sciences such as chemistry and physics. This year, two special awards, which could not be applied for, will also be awarded. The two special grants will be awarded to mid-career researchers both for their valuable research work and for speaking publicly in support of research funding, thus promoting the cause of all researchers.

In the past, the Research Foundation has granted three special awards on the basis of various award criteria to Hannes Lohi, research group leader, in 2012, and to Academy Professor Johanna Ivaska and Professor of Pharmacogenetics Mikko Niemi in 2017.

Seeking a drug that is harmless for humans and the environment

Tiina Sikanen is Associate Professor at the Division of Pharmaceutical Chemistry and Technology at the University of Helsinki. She earned a PhD in pharmacy from the University of Helsinki in 2007 and a Master of Science in Technology degree in chemical engineering from Aalto University in 2010.

Associate Professor Sikanen’s research group develops microchip-based methods for studying the metabolism of pharmaceutical ingredients, in other words, their transformation, in the body using the enzymes of various animal species and humans. Sikanen’s aim is to develop methods that enable rapid and patient-specific metabolite analytics to support personalised treatments. Patient-specific metabolite analytics can reduce the side effects of medicines as well as drug residues that burden the environment.

"I want to form an overall idea of how research can provide a better understanding in the future of the environmental impacts and risks associated with pharmaceuticals ending up in the environment," Sikanen says. In her appearances and publications, she has improved the public understanding of the environmental risks posed by active pharmaceutical ingredients and the need to reduce these risks among decision-makers, pharmaceutical and healthcare professionals and consumers.

Sikanen has received a number of major awards for her research, including the Award for Scientific Courage from the Academy of Finland in 2019 and the L’Orèal For Women in Science award in 2020. She has published more than 60 scientific articles.

Identifying the signalling mechanisms of cancer cells

Jukka Westermarck is Professor of Cancer Biology at the Department of Medical Biochemistry in the Faculty of Medicine and Research Director at the Turku Bioscience Centre at the University of Turku. He earned the degree of licentiate of medicine in 1996 and a doctorate in medicine with distinction in 1998 from the University of Turku.

His research team has endeavoured to identify the signalling mechanisms in cancer cells that promote malignancy in cancer. The group’s most significant achievement has been the identification of the CIP2A oncoprotein. This has provided new outlooks regarding the future opportunities of cancer treatment. CIP2A overproduction is one of the changes most commonly seen in cancer. "In about 70 per cent of all cancer samples, in almost all cancers, this mechanism is in operation.
Over the last 4 to 5 years, the biology associated with phosphatases has also received attention in drug development. We are now starting to get closer to being able to bring new treatment to patients," says Westermarck.

Westermarck has received many awards for his successful scientific work, the most significant of which are the Young Investigator Award from the Finnish Medical Society Duodecim in 2007 and the Anders Jahre Young Scientist Award in 2009. He has published 116 original papers and 14 review articles.

On October 27, 2022 Neuren Pharmaceuticals (ASX: NEU) reported its quarterly activity and cash flow report for Q3 2022 (Press release, Neuren, OCT 27, 2022, View Source;[email protected] [SID1234622510]).

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Neuren CEO Jon Pilcher commented: "Neuren continued to achieve the planned milestones during the quarter, remaining on track for the transforming catalysts still to come, notably the FDA target action date for trofinetide in Rett syndrome on 12 March 2023, followed by Phase 2 results for NNZ-2591 in multiple indications.

"Commentary on events since 30 June and outlook
Trofinetide for Rett syndrome In September 2022 the US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for trofinetide to treat Rett syndrome in adults and pediatric patients two years of age and older, that was submitted in July by Neuren’s North America partner Acadia Pharmaceuticals (Nasdaq: ACAD). The FDA granted a Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of 12 March 2023. The FDA also informed Acadia that at that time they were not planning to hold an Advisory Committee meeting. The trofinetide program has Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA. If approved, trofinetide will be the first drug for the treatment of Rett syndrome.

Acadia has exclusive rights to develop and commercialize trofinetide in North America, which is fully funded by Acadia.

Acceptance of the NDA earned a milestone payment of US$10 million from Acadia, which Neuren received in October. In 2023, if the NDA is approved by the FDA, Neuren expects to earn revenue for Rett syndrome in the US alone of A$112 million plus royalties. The next potential milestone payment to Neuren would be US$40 million (A$61 million at an assumed exchange rate of 0.65), payable following the first commercial sale of trofinetide in the United States. Subsequently, Neuren is eligible to receive double-digit percentage royalties on net sales of trofinetide in North America, plus milestone payments of up to US$350 million (A$538 million) on achievement of a series of four thresholds of total annual net sales, plus one third of the market value of a Rare Pediatric Disease Priority Review Voucher if awarded by the FDA upon approval of the NDA, with the one third share estimated by Neuren as US$33 million (A$51 million). No royalties or similar costs are payable by Neuren to third parties, which means that Neuren’s revenue from Acadia will flow through to pre-tax profit.

Neuren retains all rights to trofinetide for all countries outside North America and has a fully paid-up, irrevocable licence for use in those countries to all data generated by Acadia. Rett syndrome is a devastating condition with no approved therapies and there is urgent unmet need around the world for a treatment. Neuren has received strong interest for potential commercial partnerships and discussions are continuing under a process to secure the optimum outcome for shareholders and for patients. NNZ-2591 for multiple neurodevelopmental disorders During Q3 Neuren commenced its Phase 2 clinical trials of NNZ-2591 in each of Angelman syndrome (AS), Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS).

The open label Phase 2 trials will each enrol up to 20 children to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591. All subjects will receive NNZ-2591 as an oral liquid dose twice daily, with titration up to the target mg/kg dose during the first 6 weeks of treatment, subject to safety and tolerability. The treatment period is preceded by 4 weeks of observation to thoroughly examine the baseline characteristics prior to treatment, against which safety and efficacy will be assessed for each child. A follow-up assessment will be made 2 weeks after end of treatment. For each trial there are three age cohorts. Safety and tolerability is assessed in the oldest cohort before proceeding with the middle cohort and then safety and tolerability is assessed in the middle cohort before proceeding with the youngest cohort.

Neuren is also planning a Phase 2 trial in a fourth disorder, Prader-Willi syndrome, with an Investigational New Drug (IND) Application to be submitted to the FDA in Q4 2022. Neuren has Orphan Drug designation from the FDA for NNZ-2591 in all four syndromes, which are serious neurodevelopmental disorders with no approved medicines. The estimated number of potential patients being targeted across these four disorders is more than five times larger than Rett syndrome. Neuren retains all global rights to NNZ-2591. The overall aim of these first trials in patients is to expedite the generation of data that will enable the subsequent trials to be designed as registration trials. Prioritising fast enrolment of subjects, the AS trial is being conducted in Australia, whilst the PMS and PTHS trials are being conducted in the US.

In order to expedite the overall development plan, in parallel with conducting the Phase 2 trials Neuren is executing the additional development work required to be ready for Phase 3 development. This includes non-clinical toxicity studies to support longer clinical trials and commercial use of the product, as well as optimisation of the drug product and drug substance manufacturing arrangements. Neuren is well funded from current cash reserves to execute the Phase 2 trials and Phase 3 preparation, notwithstanding the anticipated material cash flows from trofinetide. Investor relations In September, Neuren was added into the S&P/ASX 300 index. Since 30 June, Neuren has presented at the healthcare conferences of Evans & Partners, Euroz Hartleys and Goldman Sachs, as well as at the ASX Small and Mid-Cap conference. Presentations are scheduled at the Wilsons and Bell Potter healthcare conferences. Neuren will also present at the Jefferies London Healthcare Conference in November.

Human resources In July, Neuren’s skills and experience in pediatric neurology and orphan drug development were further enhanced by the appointment of Liza Squires M.D. to the new position of Chief Medical Officer, based in the United States. Dr Squires is a board-certified physician in General Pediatrics and Neurology with Special Competence in Child Neurology. Over the past 20 years, she has held positions of increasing responsibilities in both early and late-stage development at Johnson and Johnson, Shire Pharmaceuticals, Lumos Pharma, Aevi Genomic Medicine and Origin Biosciences. She has led and contributed to multiple New Drug Applications resulting in global regulatory approvals and has extensive experience in orphan drug development.Financials Cash reserves at 30 September 2022 were $27.3 million, compared with $31.1 million at 30 June 2022. In Q3 net cash of $4.0 million was used in operating activities, with R&D payments of $2.8 million mainly relating to the NNZ-2591 Phase 2 clinical trials and the foundational work to prepare for Phase 3 development of NNZ-2591 across multiple indications.

The carrying value in AUD of USD cash held to mitigate exchange rate risk for USD expenditure increased by $0.2m for the quarter, due to the strengthening of the USD against the AUD. Payments to related parties of approximately $194,000 comprised the Managing Director’s executive remuneration and non-executive directors’ fees.

On October 27, 2022 Kiromic BioPharma, Inc. (NASDAQ: KRBP) ("Kiromic" or the "Company"), a clinical-stage fully-integrated biotherapeutics company using its proprietary DIAMOND artificial intelligence and data mining platform to develop cell and gene therapies with a focus on immuno-oncology, reported it entered into a Standby Equity Purchase Agreement (the "SEPA") with YA II PN, LTD, a partnership managed by Yorkville Advisors Global LP, a global investment manager, to sell up to $8 million of the Company’s stock at any time during the 24 months following the date of the SEPA, subject to the effectiveness of a registration statement with the Securities and Exchange Commission and other conditions (Press release, Kiromic, OCT 27, 2022, View Source [SID1234622509]).

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The SEPA gives Kiromic the right, but not the obligation, to sell up to $5 million of shares of common stock, which may be increased by an additional $3 million of shares of common stock, at the Company’s request, subject to terms and limitations set forth in the agreement. The number of shares issuable under the SEPA is limited to a number equivalent to 19.99 % of the outstanding common stock of the Company as of the date of the SEPA agreement unless certain pricing conditions are met, which could have the effect of limiting the total proceeds under the SEPA.

Kiromic separately also secured $2 million in gross proceeds from a new investor through the issuance of a secured convertible promissory note. The convertible note converts into the Company’s common stock at a conversion price equal to $0.35 per share.

Following the close of the two financings, Kiromic also announced the completed settlement of its prior litigation with Sabby Capital Management, another institutional shareholder and certain affiliates.

"This equity facility is an important piece of our financing strategy that not only provides flexibility and additional runway towards achieving our clinical milestones, but also enables us to settle a key prior legal issue," stated Pietro Bersani, Chief Executive Officer of Kiromic. "We believe Yorkville Advisors will be a strong financial partner for us. With the combination of both financings, we have the facilities in place whereby we have the potential to raise up to $10 million of financing, subject to certain conditions, of which $2 million has already been secured, and we look forward to ongoing discussions with additional potential investors," continued Mr. Bersani.

For further information on the SEPA or convertible note, including important terms and conditions, please see Company’s filings with the Securities and Exchange Commission, including the Company’s Current Reports on Form 8-K filed with the Securities and Exchange Commission.

On October 27, 2022 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported that it will report its third quarter 2022 financial results after market close on Thursday, November 3, 2022 (Press release, Rigel, OCT 27, 2022, View Source [SID1234622508]). Rigel senior management will follow the announcement with a live conference call and webcast at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss the financial results and give an update on the business.

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Participants can access the live conference call by dialing 877-407-3088 (domestic) or 201-389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay for 90 days after the call via the Rigel website.

On October 27, 2022 Biomea Fusion, Inc. ("Biomea")(Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, reported the dosing of the first patient in the CLL cohort of COVALENT-101, the company’s ongoing Phase I clinical trial evaluating BMF-219, Biomea’s investigational covalent menin inhibitor, in patients with R/R AML, ALL, DLBCL, MM, and now CLL (Press release, Biomea Fusion, OCT 27, 2022, View Source [SID1234622507]).

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"Despite the advances in the treatment of CLL, we know that the majority of patients relapse and are in need of a new, novel therapy," stated Thomas Butler, Biomea’s Chief Executive Officer and Chairman of the Board. "Based on the remarkable preclinical data we presented at ASCO (Free ASCO Whitepaper) of BMF-219’s effect in CLL models, including comparisons to currently available treatments, we believe BMF-219 could represent a transformative treatment option for CLL patients. With many of the Biomea Fusion team members having a long history and involvement with the successful development of CLL agents, and ibrutinib in particular, it’s quite special to be able to now evaluate the clinical potential of BMF-219 for those CLL patients that are still in need of a therapy."

At ASCO (Free ASCO Whitepaper), Biomea presented data demonstrating BMF-219’s powerful cell-killing activity as a novel, first-in-class single agent against CLL patient samples, representing a broad spectrum of mutational profiles, including those with poor prognostic mutations, such as TP53 and NOTCH1, chromosomal aberrations such as del(13q), trisomy 12 and complex karyotype. BMF-219 demonstrated near 100% response even in samples resistant to multiple standard-of-care agents.

"I am very proud of our research and translational team’s ability to further elucidate the central role of the scaffold protein menin and identify a host of specific subsets across various cancers where BMF-219 is achieving robust preclinical results. We are very excited to continue to advance this new therapeutic approach for patients with multiple liquid and solid tumors, many of whom have very little remaining alternatives," concluded Thomas Butler.

Biomea’s preclinical presentations on CLL can be accessed at the following link: View Source

About COVALENT-101

COVALENT-101 is a Phase I, open-label, multi-center, dose escalation and dose expansion study originally designed to assess the safety, tolerability, and pharmacokinetics/pharmacodynamics of oral dosing of BMF-219 in patients with R/R acute leukemias —including subpopulations where menin inhibition is expected to provide maximal therapeutic benefit (e.g., patients with MLL1/KMT2A gene rearrangements or NPM1 mutations), multiple myeloma (MM) and diffuse large B-cell lymphoma (DLBCL). The study design has now been expanded to include a cohort for patients with R/R CLL. Additional information about the Phase I clinical trial of BMF-219 can be found at ClinicalTrials.gov using the identifier NCT05153330.

About Chronic Lymphocytic Leukemia (CLL)

CLL is a chronic leukemia that progresses relatively slowly and typically impacts older adults. In the United States, approximately 20,000 patients are diagnosed with CLL each year. While the existing treatment options produce 5-year survival outcomes greater than 87%, there is an unmet need for patients that have high- or medium-risk cytogenetic profiles and those who have relapsed or were refractory to existing treatments.