On October 12, 2023 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported an update on its Phase 3 open-label registrational clinical trial (the REGAL study) for galinpepimut-S (GPS) in patients with acute myeloid leukemia (AML) who have achieved complete remission following second-line salvage therapy (CR2 patients) (Press release, Sellas Life Sciences, OCT 12, 2023, View Source [SID1234635891]).

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The Company expects to complete enrollment in the REGAL study, other than the 20-25 patients anticipated to be enrolled in China, in November 2023. The number of patients needed for the pre-specified interim and final analyses have already been enrolled. The interim analysis (after 60 events) is on track to occur in late 2023 or early 2024 and the final analysis (after 80 events) is on track to occur by the end of 2024. Because these analyses are event driven, they may occur at a different time than currently expected.

The Company anticipates that 3D Medicines Inc. (3D Medicines), its commercialization partner for GPS in Greater China, will begin enrolling patients in China in the REGAL study in the fourth quarter of 2023 which will trigger two development milestone payments totaling $13.0 million. The Company and 3D Medicines had previously projected that enrollment in China would commence in the third quarter of 2023, however, unforeseen supply-chain delays in China impacted the projected timeline. 3D Medicines has successfully obtained all regulatory permits, recruited investigators, and set up an expansive network of 11 treatment centers across China for participation in the REGAL study.

The next meeting of the Independent Data Monitoring Committee for the REGAL study is scheduled for the end of November 2023.

"We are pleased to be so close to achieving this most important milestone – the completion of enrollment in the REGAL study – and, at the same time, it is important to note that we already have a sufficient number of patients enrolled for the pre-specified analyses," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Furthermore, 3D Medicines’ enthusiasm for the REGAL study remains high. We expect the first patient dosing in China to take place this quarter which will trigger the milestone payment, and we are exploring options to potentially expedite the payment. Importantly, adding approximately 25 patients from China could potentially facilitate drug approval in this significant market, assuming positive data, further yielding additional milestone payments and royalties for SELLAS."

On October 12, 2023 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported initial clinical data for RLY-4008 (lirafugratinib) in patients with FGFR2-altered solid tumors (Press release, Relay Therapeutics, OCT 12, 2023, View Source [SID1234635890]). The data demonstrate activity across several sub-groups, including patients with FGFR2-fusion tumors and patients with FGFR2-altered HR+/HER2- breast cancer. These data are being presented today at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper).

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"These data provide important early evidence that RLY-4008, or lirafugratinib, has the potential to help both patients with FGFR2-fusion cholangiocarcinoma as previously reported, as well as those with multiple other types of FGFR2-altered tumors," said Don Bergstrom, M.D., Ph.D., President of R&D at Relay Therapeutics. "We are excited by the potential for lirafugratinib to help many more patients and are focused on advancing this opportunity as well as our PI3Kα programs, with the initiation of a RLY-2608 triplet combination trial this year."

ReFocus Trial

Lirafugratinib is currently being evaluated in the two-part global Phase 1/2 ReFocus trial in patients with FGFR2-altered tumors. The first part of the study (dose escalation) is complete, and the second part of the study (dose expansion) is ongoing at the 70mg QD recommended Phase 2 dose. The dose expansion portion of the study includes four cholangiocarcinoma (CCA) arms and three (non-CCA) tumor agnostic arms (1: FGFR2 fusions, 2: FGFR2 amplifications and 3: FGFR2 mutations).

As of the August 23, 2023 cut-off date, the three tumor agnostic arms of the study had enrolled 84 FGFR inhibitor-naïve patients who were efficacy evaluable across 18 tumor types, including 26 patients with FGFR2 fusions, 34 patients with FGFR2 amplifications and 24 patients with FGFR2 mutations. Across these arms of the study, enrolled patients had received a median of approximately three prior lines of therapy, with the vast majority (94%) having received prior chemotherapy/ADC and nearly half (45%) having received prior targeted therapies.

Encouraging Initial FGFR2-Fusion Tumor-Agnostic Signal with Promising Durability

In patients with FGFR2 fusions, there was consistent activity across a range of tumor types.

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Nine of 26 patients experienced a partial response (PR) (35% overall response rate (ORR))
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Sixty-three percent of confirmed responders experienced a duration of response of at least 6 months as of the data cut-off date
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There were 11 tumor types represented amongst enrolled patients with FGFR2 fusions, including pancreatic (n=6), ovarian (n=3), gastric (n=3), non-small-cell lung (NSCLC, n=2), and breast (n=2)
Compelling Response Rate with Multiple Long-Term Responses in Heavily Pre-Treated Patients with HR+/HER2- Breast Cancer

The study enrolled 14 patients with breast cancer across all FGFR2 alterations, 10 of whom had HR+/HER2- breast cancer.

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Four of the 10 HR+/HER2- patients achieved PRs (40% ORR)
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Three of the four responders remain on treatment, with the longest duration of response 72 weeks and ongoing as of the data cut-off date
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All responders had a duration of response of at least 6 months
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All 14 patients were very heavily pre-treated, with a median of six prior lines of therapy
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All patients had received prior targeted therapies
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Nearly all patients had received prior chemotherapy/ADC (93%)
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The vast majority of patients had received prior endocrine therapy (79%) and prior CDK4/6 (71%)
Early Tumor-Agnostic Signal in FGFR2-Amplifications

There were signals of activity in patients with a range of FGFR2-amplified tumor types.

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Eight of 34 patients experienced a PR (24% ORR)
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PRs seen across tumor types, including gastric, breast, colorectal, and esophageal
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Six patients remain on treatment as of data cut-off, including four responders, one patient with stable disease and one patient who continued treatment beyond disease progression
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Forty-three percent of confirmed responders experienced a duration of response of at least 6 months
Additional Signals

Early, promising efficacy signals were seen in patients with FGFR2-fusions and amplifications across eight tumor types, including gastric, breast, pancreatic, NSCLC, ovarian, colorectal, esophageal, and carcinoma of unknown primary origin. In addition, three of the 24 patients with FGFR2 mutations achieved a PR (breast, gastric and ameloblastic tumors).

Safety Data Remain Generally Consistent with Previously Reported Profile

The safety analysis from the tumor agnostic cohorts, as of the data cut-off date, was generally consistent with the analysis from the 2022 ESMO (Free ESMO Whitepaper) data disclosure.

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Most treatment-related adverse events were expected FGFR2 on-target, low-grade, monitorable, generally manageable and largely reversible
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There were no observed Grade 4 or 5 adverse events
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Off-target toxicities of hyperphosphatemia and diarrhea continued to be clinically insignificant
Lirafugratinib Next Steps

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Continue enrollment in the three tumor agnostic cohorts
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The company expects to report additional clinical data and a regulatory update in 2024
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Enrollment is complete in the pivotal expansion cohort in patients with FGFR2-fusion CCA who have not previously received an FGFR inhibitor
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Near-term commercial readiness activities for CCA will be paused and aligned with the broader tumor agnostic opportunity
The AACR (Free AACR Whitepaper)-NCI-EORTC presentation and poster are available on the Relay Therapeutics website under Publications: View Source

Pipeline Updates

The company will continue to prioritize and expand further PI3Kα mutant selective development, including:

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RLY-2608: continue ongoing ReDiscover trial with focus on RLY-2608 + fulvestrant cohorts
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Initiate triplet combination with RLY-2608 + fulvestrant + CDK4/6 by the end of 2023
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Next PI3Kα clinical data update expected in 2024
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Additionally, Relay Therapeutics has decided to pause further development efforts on RLY-2139 (CDK2 inhibitor)
Cash Runway Extended

With the decision to pause CCA commercial readiness and RLY-2139 development, Relay Therapeutics expects its cash, cash equivalents and investments will be sufficient to fund its current operating plan into the second half of 2026.

Conference Call Information

Relay Therapeutics will host a conference call and live webcast today, Thursday, October 12, 2023, at 5:30 p.m. ET. Registration and dial-in for the conference call may be accessed on Relay Therapeutics’ website under Events in the News & Events section through the following link: View Source An archived replay of the webcast will be available following the event.

About RLY-4008 (lirafugratinib)

RLY-4008 (lirafugratinib) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models, while minimal inhibition of other targets was observed, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in cellular and in vivo preclinical models. Lirafugratinib is currently being evaluated in a clinical trial in patients with advanced or metastatic FGFR2-altered solid tumors with a single arm, potentially registration-enabling cohort for FGFRi-naïve FGFR2-fusion CCA. To learn more about the clinical trial of lirafugratinib, please visit here.

ReFocus Trial Background

RLY-4008 (lirafugratinib) is currently being evaluated in a global Phase 1/2 clinical trial (ReFocus) in patients with FGFR2-altered CCA and multiple other solid tumors including a single-arm, potentially registration-enabling cohort for FGFRi-naïve FGFR2-fusion CCA. The Phase 1 dose escalation has been completed, and 70 mg QD has been selected as the registrational dose. The expansion cohorts were initiated in December 2021 and now consist of seven different cohorts based on FGFR2 alteration and tumor type. Of the seven cohorts, the potential pivotal cohort consists of approximately 100 previously treated, FGFRi-naïve FGFR2-fusion CCA patients.

On October 12, 2023 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, excision, and elimination, reported that two abstracts have been accepted by the European Society of Gene & Cell Therapy (ESGCT) for poster presentations at the upcoming Congress on October 24-27, 2023, in Brussels, Belgium (Press release, Precision Biosciences, OCT 12, 2023, View Source [SID1234635889]).

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Presentation Details:

Title: Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo
Poster Number: #P641
Presenter: Cassie Gorsuch, VP of Gene Therapy, Precision Biosciences
Date and Time: Wednesday, October 25, 2023, 5:00 PM – 6:15 PM CEST and Thursday, October 26, 2023, 8:30 PM – 9:30 PM CEST
Location: Gare Maritime

Title: ARCUS-mediated excision of the "hot spot" region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)
Poster Number: Poster #P653
Presenter: Cassie Gorsuch, VP of Gene Therapy, Precision Biosciences
Date and Time: Wednesday, October 25, 2023, 5:00 PM – 6:15 PM CEST and Thursday, October 26, 2023, 8:30 PM – 9:30 PM CEST
Location: Gare Maritime

On October 12, 2023 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with hematologic malignancies and solid tumors, reported financial results for the fiscal quarter ended August 31, 2023, and provided a corporate update (Press release, Nurix Therapeutics, OCT 12, 2023, View Source [SID1234635888]).

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"As our drug development pipeline continues to advance in the clinic, we are excited to add to our pipeline with Seagen to advance a portfolio of Degrader-Antibody Conjugates, or DACs, a new class of highly selective cancer therapeutics," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "Nurix enters the fourth quarter of 2023 in a strong financial position due to our ability to access significant revenue from corporate partners including our recently announced agreement with Seagen and ongoing strategic collaborations with Gilead and Sanofi."

Recent Business Highlights

•Nurix announced a first of its kind strategic collaboration with Seagen: In September, Nurix entered a collaboration with Seagen to develop a portfolio of Degrader-Antibody Conjugates (DACs): antibodies that deliver a targeted protein degrader payload to selectively kill cancer cells. Nurix received a $60 million upfront payment and has the potential to receive approximately $3.4 billion in milestone payments plus future royalties. Nurix also retains an option for U.S. profit sharing and co-promotion on two products arising from the collaboration. In addition, Nurix announced that with the receipt of the $60 million upfront payment, Nurix expects that its existing cash, cash equivalents and marketable securities, excluding any future potential milestones from collaborations, will be sufficient to fund its operating activities into the second quarter of 2025.

•Nurix expanded its Phase 1a trial of NX-1607 to include a combination with paclitaxel: In August, Nurix treated the first patient with NX-1607 in combination with paclitaxel. The decision to initiate combination trials was informed by the evolving safety and activity data from Phase 1a.

Upcoming Program Highlights*

•NX-5948: NX-5948 is an orally bioavailable degrader of Bruton’s tyrosine kinase (BTK) designed without immunomodulatory activity. Nurix is evaluating NX-5948 in a Phase 1 clinical trial in adults with relapsed or refractory B-cell malignancies and expects to present initial clinical data from the Phase 1a portion of the study in the second half of 2023. In addition, Nurix expects to define a dose for the Phase 1b cohort expansion in the second half of 2023. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT05131022).

•NX-2127: NX-2127 is an orally bioavailable degrader of BTK with immunomodulatory activity for the treatment of patients with relapsed or refractory B-cell malignancies. Nurix is conducting a Phase 1 clinical trial of NX-2127, which includes three Phase 1b expansion cohorts in patients with diffuse large B cell lymphoma (DLBCL), mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL). Nurix anticipates presenting additional clinical results from this ongoing trial in the second half of 2023. Nurix also anticipates defining a regulatory strategy for NX-2127 in the second half of 2023 based on emerging clinical data and feedback from the FDA. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT04830137).

•NX-1607: Nurix’s lead drug candidate from its targeted protein elevation portfolio, NX-1607, is an orally bioavailable inhibitor of the E3 ligase Casitas B-lineage lymphoma proto-oncogene B (CBL-B) for immuno-oncology indications including a range of solid tumor types and lymphoma. Nurix is evaluating NX-1607 in an ongoing, Phase 1a dose escalation trial in monotherapy and in a combination cohort utilizing paclitaxel in adults in a range of oncology indications. Nurix expects to present clinical data from the Phase 1a portion of the study and to define a dose for Phase 1b cohort expansion in the second half of 2023. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT05107674).

•NX-0479/GS-6791: GS-6791 (previously NX-0479) is a potent, selective, oral IRAK4 degrader. Degradation of IRAK4 by GS-6791 has potential applications in the treatment of rheumatoid arthritis and other inflammatory diseases. Nurix’s partner, Gilead Sciences, is responsible for conducting IND-enabling studies and advancing this program to clinical development.

•Continued advancement of strategic collaborations with Gilead and Sanofi: Nurix expects to continue to achieve substantial research collaboration milestones throughout the terms of its collaborations with Gilead and Sanofi.
*Expected timing of events throughout this press release is based on calendar year quarters.

Fiscal Third Quarter 2023 Financial Results

Revenue for the three months ended August 31, 2023, was $18.5 million compared to $10.8 million for the three months ended August 31, 2022. The increase was primarily due to a higher percentage of completion of performance obligations and an increase in the value of milestones achieved in the current period. During the three months ended August 31, 2023, Nurix achieved research milestones under its collaborations with Gilead and Sanofi totaling $6.0 million and $2.0 million, respectively.

Research and development expenses for the three months ended August 31, 2023, were $47.9 million compared to $47.8 million for the three months ended August 31, 2022. There was an increase in clinical costs as Nurix continued its clinical trial programs and ongoing patient enrollment, primarily offset by a decrease in research related costs and in contract manufacturing. There was also an increase in facility and other costs primarily driven by additional investments in information technology and lease related expenses.

General and administrative expenses for the three months ended August 31, 2023, were $10.6 million compared to $9.7 million for the three months ended August 31, 2022. The increase was primarily related to an increase in non-cash stock-based compensation expense and an increase in professional service costs related to the Seagen collaboration agreement, offset by a decrease in outside consulting costs.

Net loss for the three months ended August 31, 2023, was $37.0 million, or ($0.68) per share, compared to a net loss of $45.7 million for the three months ended August 31, 2022, or ($0.90) per share.
Cash, cash equivalents and marketable securities was $268.7 million as of August 31, 2023, compared to $308.6 million as of May 31, 2023.

On October 12, 2023 Morphic therapeutics presented tis corporate presentation (Presentation, Morphic Therapeutic, OCT 12, 2023, View Source [SID1234635887]).

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