On October 31, 2023 iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways, reported that the US Food and Drug Administration (FDA) has granted permission to proceed with clinical investigations of roginolisib in the United States of America (Press release, iOnctura, OCT 31, 2023, View Source [SID1234640231]).

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Roginolisib (IOA-244) is in development for solid and hematologic malignancies including uveal melanoma, a rare cancer in which malignant (cancer) cells form in the uveal tract of the eye. When the cancer metastases, which it does in approximately 50% of patients, there are limited treatment options and projected overall survival is only a year.

As the first novel allosteric modulator of PI3Kδ, roginolisib marks a new era in drug development within the class. The unique binding mode, combined with high selectivity for PI3Kδ, is expected to translate to an improved safety and tolerability profile relative to that of earlier generation inhibitors.

"As we remain on track to deliver final clinical data from the initial patients treated with roginolisib in 2024, the clearance of our IND application demonstrates our commitment to driving roginolisib through the clinic" said Catherine Pickering, Chief Executive Officer of iOnctura. "We believe roginolisib has the potential to slow or halt the progression of uveal melanoma, so providing an important treatment option for patients who currently have no approved therapeutic options after they progress on their first line therapy."

Roginolisib is being investigated in the DIONE-01 trial, a two-part, first-in-human Phase I study (ClinicalTrials.gov, identifier NCT04328844). The study is fully enrolled with final data expected Q1 2024. Across all patients treated to date, roginolisib monotherapy has shown 7% Grade 3/4 toxicities, with no dose limiting toxicities, drug-related serious adverse event (SAE) or drug related adverse event (AE) leading to dose interruption or discontinuation. Whilst median Overall Survival has not yet been reached, 62% of patients were alive at 12 months, which compares favourably to historical controls of 34% in the same setting[1].

Long-term administration of roginolisib is well tolerated and patients have been treated for up to 40 months in the study. Promising clinical activity has been observed across patients with both solid and hematological cancers.

[1] Rantala et al., Melanoma Res. 2019 Dec;29(6):561-568

On October 31, 2023 Molecure S.A. ("Molecure": WSE: MOC) a clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases reported third quarter results for the period ended 30 September 2023 (Press release, Molecure, OCT 31, 2023, View Source [SID1234640059]). The full report in Polish can be found here View Source

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Marcin Szumowski, CEO and President of the Management Board of Molecure said: "We have made substantial financial and operational progress during 2023 including the completion of a significantly oversubscribed secondary public offering. This will enable the company to deliver meaningful additional data which will be important in fulfilling the longer-term potential of our pipeline.

We have also achieved key milestones in clinical development with the dosing of the first cancer patient in our Phase I study with OATD-02 with anticipated initial results in the next few months. Additionally, we expect to dose the first pulmonary sarcoidosis patient in the Phase II study of OATD-01 before the end of the year.

Molecure is entering a very exciting stage in its development with a number of near-term value-creating milestones set to be achieved in our pipeline and we look forward to providing meaningful updates on our progress over the next year. "

Commercial & Operational Highlights in the third quarter

Successful Public Offering

Molecure successfully raised, through a Secondary Public Offering, gross proceeds of approximately PLN50m (USD12m) from existing and new shareholders,
Proceeds and expected grant awards will be used to fund and build Molecure’s first in class sustainable pipeline of breakthrough therapies through significant value inflection points including completion of the Phase II study for OATD-01 in sarcoidosis and completion of the Phase I clinical trial for OATD-02 in oncology patients, with the possibility of expansion into additional indications and combination therapies.
US FDA Clearance to conduct Phase II clinical trial – OATD-01

Molecure received US FDA Investigational New Drug (IND) approval for OATD-01 which will allow the company to conduct Phase II clinical trials in the US. The planned Phase II proof-of-concept study will be the first to treat patients suffering from pulmonary sarcoidosis and is expected to start in the fourth quarter 2023.
Molecure has submitted applications to the EMA and UK MHRA to initiate a Phase II clinical trial in the European Union and Norway and the UK respectively.
Molecure signed an agreement with Simbec-Orion, a leading global Clinical Research Organization which will conduct the clinical trial on behalf of Molecure. The Phase II trial will be conducted in the US and several European Union Countries and enroll 90+ patients with active pulmonary sarcoidosis.
Nine Months Financial Highlights

Operating income of PLN1.3 million, in line with the same period in 2022.
Operating expenses totaled PLN16.3 million, an increase of PLN4.3 million vs the same period last year. This was mainly due to higher research costs as the company’s pipeline advances, higher salaries and costs of external services.
Net loss for the first nine months of the year totaled PLN11.5 million vs net loss of PLN9.4 million in the first nine months of the year in 2022.
As of September 30, 2023, Molecure had cash of nearly PLN85 million (US$20 million).
US$/PLN exchange rate 4.2 as of 30 September 2023.

On October 31, 2023 Incyte reported its 2023 third quarter financial and corporate update (Presentation, Incyte, OCT 31, 2023, View Source [SID1234639530]).

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On October 31, 2023 Leidos Holdings, Inc. (NYSE: LDOS), a FORTUNE 500 science and technology leader, reported financial results for the third quarter of fiscal year 2023 (Press release, Leidos, OCT 31, 2023, View Source [SID1234639224]).

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Thomas Bell, Leidos Chief Executive Officer, commented, "Our third quarter financial results build on the momentum from last quarter and are the direct result of our entire team being aligned on our direction and intent on delivering against our commitments. With record revenues, non-GAAP earnings, cash flow, bookings, and backlog, our growth outlook is improving, and we are raising our guidance for all 2023 financial metrics. As we sharpen our strategy and better align our organization, our focus on differentiating technology will enable us to unlock our full potential and heighten our value proposition to our customers, our nation, and its allies."

On October 31, 2023 Nimbus Therapeutics, LLC ("Nimbus Therapeutics" or "Nimbus"), a clinical-stage company that designs and develops breakthrough medicines through its powerful computational drug discovery engine, reported initial data from the company’s ongoing Phase 1/2 study of NDI-101150, a small-molecule inhibitor of HPK1, which are being presented in a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 38th Annual Meeting in San Diego, CA (Press release, Nimbus Therapeutics, OCT 31, 2023, View Source [SID1234638826]).

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The data presented at SITC (Free SITC Whitepaper) include initial safety and efficacy results from single agent therapy (n=13) in the dose escalation portion of the trial. Monotherapy treatment with NDI-101150 resulted in clinical benefit in 3 patients: One patient with renal cell carcinoma exhibited a complete response (CR) — the absence of all detectable cancer following treatment — while two patients with pancreatic cancer and renal cell carcinoma, respectively, exhibited prolonged (> 6 months) stable disease. Notably, the renal cell carcinoma patient who experienced a CR and clinical benefit with monotherapy NDI-101150 had been previously treated with and progressed on a regimen of nivolumab, an immune checkpoint inhibitor antibody. Furthermore, NDI-101150 demonstrated an acceptable safety profile below 200 mg/day, the identified non-tolerated dose.

"We’re pleased to share these first clinical data from our clinical trial of NDI-101150. We are encouraged by the preliminary efficacy we have seen thus far, which supports the potential of NDI-101150 to provide a meaningful therapeutic option for patients with solid tumors," said Nathalie Franchimont, M.D., Ph.D., Chief Medical Officer of Nimbus. "HPK1 inhibition is an exciting approach because of its potential to activate not just T cells, as checkpoint inhibitors do, but also B cells and dendritic cells. We look forward to sharing future updates on NDI-101150, including data from ongoing combination cohorts and dose expansion cohorts."

The Phase 1/2 trial (NCT05128487) is a multicenter, open-label study to assess the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of NDI-101150 given as monotherapy or in combination with pembrolizumab in adults with advanced or metastatic solid tumors.

In a second poster at SITC (Free SITC Whitepaper), Nimbus is presenting new preclinical data which support the potential for both broad immunotherapeutic potential and best-in-class selectivity of NDI-101150 among publicly disclosed HPK1 inhibitor programs to date. NDI-101150 was found to be over 300 times more selective for HPK1 than related proteins in the MAP4K family — potentially reducing off-target effects.

"Our driving purpose at Nimbus is to leverage our expertise and technology to design breakthrough medicines for patients," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "The data presented at SITC (Free SITC Whitepaper) reinforce the potential first-in-class and best-in-class profile of our HPK1 inhibitor, and we will continue to work to realize its possible benefits to patients with cancer."