On May 2, 2024 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a clinical-stage pharmaceutical company dedicated to developing personalized cancer treatments, reported the early discontinuation of its Phase 2 clinical trial of stenoparib, a novel PARP inhibitor, for the treatment of advanced recurrent ovarian cancer (Press release, Allarity Therapeutics, MAY 2, 2024, View Source [SID1234642603]). The patients enrolled in the trial had been pre-screened by Allarity’s unique Drug Response predictor (DRP) companion diagnostic (CDx) in order to treat only patients with the highest likelihood of deriving clinical benefit.

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The trial, evaluating stenoparib given twice daily, has shown clear clinical benefit, including tumor shrinkage and long-term disease stability, in heavily pre-treated ovarian cancer patients who otherwise have limited life expectancy. These results have provided sufficient clinical proof of concept for stenoparib as monotherapy, prompting Allarity to halt further enrollment in this trial to enable and accelerate the development of a follow-on trial with FDA regulatory intent.

"Based on the substantial clinical benefit observed in the early stages of the trial, we have achieved proof of concept results that surpassed our initial expectations and provided the critical insights we were seeking," stated Thomas Jensen, CEO of Allarity Therapeutics. "Concluding the trial now is the most effective way to re-allocate our financial resources to develop a follow-on trial with the fastest route to regulatory submission for stenoparib. The patients enrolled in this trial are heavily pretreated, having undergone multiple prior treatments, often including PARP inhibitors. It is highly noteworthy that stenoparib, used in patients selected with the DRP CDx, has delivered sustained clinical benefit for such very heavily pre-treated patients in the trial."

This Company’s decision will not affect the ongoing treatment of current patients, as described in greater detail in Allarity’s March 27, 2024, press release.

Allarity is committed to rapidly analyzing the trial data and plans to present more comprehensive data as early as possible in a clinical update. This early trial conclusion marks a significant milestone in developing stenoparib, reflecting Allarity’s dedication to advancing stenoparib to address the urgent needs of advanced ovarian cancer patients.

About Advanced, Recurrent Ovarian Cancer
Advanced, recurrent ovarian cancer refers to the return of ovarian cancer after it has been treated. This condition typically occurs in the later stages of the disease, which are classified as stage III or IV at diagnosis. It is characterized by the cancer’s resistance to treatment, making its management particularly challenging. The initial symptoms of ovarian cancer can be very subtle, leading to a late diagnosis in many cases.

The recurrence of ovarian cancer is not uncommon, and it significantly complicates the therapeutic landscape. The current main goals of treating advanced, recurrent ovarian cancer are to extend the patient’s life while maintaining or improving their quality of life, as curative options are limited. Treatment strategies may involve a combination of surgery, chemotherapy, targeted therapy, and, in some cases, radiation therapy. However, the effectiveness of these treatments diminishes with each recurrence, highlighting the need for innovative approaches to manage the disease.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has proven its ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients dozens of clinical studies (both retrospective and prospective). The DRP platform, which can be used in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature.

On May 2, 2024 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported a $50 million milestone payment was received from Royalty Pharma plc (NASDAQ: RPRX) (Press release, Arrowhead Pharmaceuticals, MAY 2, 2024, View Source [SID1234642602]). This milestone was triggered after the completion of enrollment of the Phase 3 OCEAN(a) – Outcomes Trial of olpasiran, being conducted by Amgen (NASDAQ: AMGN). Pursuant to its 2016 agreement with Amgen and 2022 agreement with Royalty Pharma, Arrowhead is further eligible to receive up to an additional $375 million from Amgen and $110 million from Royalty Pharma in aggregate development, regulatory, and sales milestone payments associated with olpasiran.

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"The rapid enrollment of the OCEAN(a) – Outcomes Trial demonstrates the strong interest in olpasiran, developed using Arrowhead’s proprietary TRiMTM technology and licensed to Amgen in 2016. Partnering is an important part of our strategy and we are pleased with all the care and work undertaken to bring this potentially important new therapy closer to patients," said Christopher Anzalone, Ph.D., Arrowhead’s president and CEO. "Our pipeline of wholly owned or partnered TRiMTM-enabled candidates now includes three programs in Phase 3 – olpasiran, fazirsiran, and plozasiran. Importantly, our lead wholly owned candidate plozasiran, a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3), is on schedule to complete its first pivotal Phase 3 study this quarter, with a topline readout soon after."

Olpasiran is a small interfering RNA (siRNA) originally developed by Arrowhead using its proprietary Targeted RNAi Molecule (TRiMTM) platform. It is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease. The primary objective of the Phase 3 OCEAN(a) – Outcomes Trial is to compare the effect of treatment with olpasiran, to placebo, on the risk for coronary heart disease death, myocardial infarction, or urgent coronary revascularization in participants with atherosclerotic cardiovascular disease and elevated lipoprotein(a).

About Lp(a)

Lp(a) is primarily genetically determined1-3 and a presumed independent risk factor for cardiovascular disease (CVD). Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).1 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease.

On May 2, 2024 Theratechnologies Inc. ("Theratechnologies" or the "Company") (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported that it will present long-term efficacy, safety and pharmacokinetic (PK) data on the use of its lead investigational peptide-drug conjugate (PDC) candidate, TH1902 (sudocetaxel zendusortide), in patients with solid tumors (Press release, Theratechnologies, MAY 2, 2024, View Source [SID1234642599]). The Company will present the long-term data in a poster session at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which takes place May 31-June 4, 2024, in Chicago, IL.

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The ASCO (Free ASCO Whitepaper) poster represents the first presentation of long-term data from Part 1 (dose escalation) and Part 2 (dose expansion) of Theratechnologies’ Phase 1 clinical trial of sudocetaxel zendusortide in individuals with solid tumors, following preliminary evidence of antitumor activity presented at the 2023 ASCO (Free ASCO Whitepaper) annual meeting. This updated analysis will present further data on long-term efficacy, safety and PK from Parts 1 and 2, focusing specifically on patients receiving sudocetaxel zendusortide at a dose of 300 mg/m2 every three weeks. Patients in this dosing group have cancers with known high expression of sortilin (SORT1), including ovarian cancer, endometrial cancer, triple-negative breast cancer (TNBC) and melanoma. Part 3 (dose optimization) of the Phase 1 trial, in patients with advanced ovarian cancer, is ongoing.

"We have eagerly awaited the updated analysis from Parts 1 and 2 of the Phase 1 trial, as it will provide our first evidence of the long-term effects of sudocetaxel zendusortide in patients with solid tumors," said Christian Marsolais, Ph.D., Senior Vice President and Chief Medical Officer at Theratechnologies. "These safety, efficacy and pharmacokinetics data are particularly timely, in that they will provide valuable context as we continue to evaluate this novel peptide-drug conjugate in Part 3 of this ongoing trial."

Details of the poster presentation are as follows:

June 1, 2024, 9:00 AM-12:00 PM CDT

Presenting Author: Ira Winer, MD, Karmanos Cancer Institute, Detroit, MI

Session Category: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Session Title: Long-term efficacy, safety and PK data of TH1902 (sudocetaxel zendusortide) in solid tumors: A novel SORT1-targeting peptide-drug-conjugate (PDC)

Location: Hall A, McCormick Place Congress Center, Chicago

Poster Board Number: 226

Abstract Presentation Number: 3081

About Sudocetaxel Zendusortide (TH1902) and SORT1+ Technology

Sudocetaxel zendusortide is a first-of-its-kind sortilin receptor (SORT1)-targeting PDC, and the first compound to emerge from the Company’s broader licensed oncology platform. A new chemical entity, sudocetaxel zendusortide employs a cleavable linker to conjugate (attach) a proprietary peptide to docetaxel, a well-established cytotoxic chemotherapeutic agent used to treat many cancers. The FDA granted Fast Track designation to sudocetaxel zendusortide as a single agent for the treatment of all sortilin-positive recurrent advanced solid tumors that are refractory to standard therapy. Sudocetaxel zendusortide is currently being evaluated in a Phase 1 clinical trial.

Theratechnologies has established the SORT1+ Technology platform as an engine for the development of PDCs that target SORT1, which is expressed in multiple tumor types. SORT1 is a "scavenger" receptor that plays a significant role in rapid protein internalization, sorting, and trafficking. Expression of SORT1 is associated with aggressive disease, poor prognosis, and decreased survival. It is estimated that SORT1 is expressed in 40% to 90% of endometrial, ovarian, colorectal, triple-negative breast (TNBC), and pancreatic cancers, making this receptor an attractive target for anticancer drug development.

On May 2, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported first quarter financial results for the period ended March 31, 2024 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, MAY 2, 2024, View Source [SID1234642598]).

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"We are very encouraged by the strong start to the OGSIVEO launch and are focused on continuing our momentum towards establishing OGSIVEO as the standard of care treatment for adults with desmoid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are also pleased to have initiated the rolling NDA submission for mirdametinib in children and adults with NF1-PN, which would be our second marketed product serving another group of patients who are currently suffering with a high unmet need, if approved. We are making significant progress across our commercial, development and corporate objectives and look forward to providing updates throughout the year."
Recent Business Highlights and Upcoming Milestones
OGSIVEO (Nirogacestat)
•Strong execution in the first full quarter of launch, with net product revenue of $21.0 million in the first quarter of 2024.
•Received U.S. Food and Drug Administration (FDA) approval of a Supplemental New Drug Application (NDA) for OGSIVEO 150 mg and 100 mg tablets in new blister packaging, which have been developed to enhance patient convenience with OGSIVEO. The blister packs are expected to be on the market in mid-May 2024.
•Received validation from the European Medicines Agency (EMA) on a Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors in February 2024.
•Additional data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors were accepted for presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. An abstract describing the onset and resolution of ovarian toxicity for desmoid tumor patients treated with nirogacestat was selected as an oral presentation to be delivered on May 31, 2024. Two additional sub-group analyses evaluating nirogacestat in desmoid tumor patients with poor prognostic factors and in those with adenomatous polyposis (APC) mutations will also be presented on June 1, 2024.
•On track to report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors in the second half of 2024.

•Continuing to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
Mirdametinib
•Initiated rolling submission of an NDA to the FDA for mirdametinib for the treatment of children and adults with NF1-PN in March 2024. SpringWorks expects to complete the NDA submission in the second quarter of 2024 and expects to file a MAA with the EMA for mirdametinib for the treatment of children and adults with NF1-PN in the European Union the second half of 2024.
•Data from the pediatric and adult cohorts of the Phase 2b ReNeu trial were accepted for oral presentation as a rapid oral abstract at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on June 3, 2024. SpringWorks also expects to publish the ReNeu trial results in a peer-reviewed journal in 2024.
Emerging Pipeline
•On track to present additional data from the dose expansion portion of the Phase 1b trial evaluating brimarafenib (BGB-3245) in adult patients with RAF mutant solid tumors in the second half of 2024. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.
•Patients continue to be enrolled in the dose escalation phase of the SpringWorks-sponsored Phase 1/2a combination study of brimarafenib and mirdametinib.
•MapKure initiated a Phase 1b combination trial of brimarafenib with panitumumab, a monoclonal antibody targeting Epidermal Growth Factor Receptor in colorectal and pancreatic cancer patients with known MAPK pathway mutations; patient dosing is currently underway.
•Dose expansion in adult patients with NRAS mutant solid tumors is ongoing in the BeiGene-sponsored Phase 1b trial evaluating mirdametinib in combination with BeiGene’s RAF dimer inhibitor, lifirafenib.
•On track to initiate a Phase 1a trial of SW-682, an investigational novel, oral, potent, and selective pan-TEAD inhibitor, in Hippo-mutant solid tumors in the second quarter of 2024.

General Corporate

•The U.S. Patent and Trademark Office has recently issued five new patents for OGSIVEO. The U.S. patent portfolio for OGSIVEO now includes 21 Orange Book listed patents, providing protection into 2043.

First Quarter 2024 Financial Results

•Revenues: OGSIVEO net product revenues were $21.0 million in the first quarter of 2024, the first full quarter of the U.S. launch.
•Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $60.1 million for the first quarter of 2024, compared to $44.2 million for the comparable period of 2023. The increase in SG&A expense was primarily attributable to commercial activities supporting the U.S. launch of OGSIVEO.
•Research and Development (R&D) Expenses: R&D expenses were $53.6 million for the first quarter of 2024, compared to $33.5 million for the comparable period of 2023. The increase in R&D expenses was primarily attributable to an increase in costs related to drug manufacturing, clinical trials, other research, consulting and professional services, and an increase in employee costs associated with headcount growth.
•Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $87.4 million, or $1.18 per share, for the first quarter of 2024. This compares to a net loss of $73.4 million, or $1.18 per share, for the comparable period of 2023.
•Cash, Cash Equivalents, and Marketable Securities: Cash, cash equivalents and marketable securities were $573.0 million as of March 31, 2024.

Conference Call Information

SpringWorks will host a conference call and webcast today, Thursday, May 2, at 8:30 a.m. ET to review its first quarter 2024 financial results and discuss recent business updates. To join the live webcast and view the corresponding slides, please click here. To access the live call by phone, please pre-register for the call by clicking here. Once registration is complete, participants will be provided with a dial-in number and conference code to access the call. A replay of the webcast will be available for a limited time following the event on the Investors and Media section of the Company’s website at View Source

On May 2, 2024 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 first quarter 2024 financial results and corporate highlights (Press release, Relay Therapeutics, MAY 2, 2024, View Source [SID1234642597]).

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"We have started 2024 with a focus on maintaining strong execution across our portfolio," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "We continue to progress a robust RLY-2608 development program and expect to share additional data in the second half of the year. In parallel, we have continued to advance our pre-clinical pipeline and look forward to disclosing at least one new program this year, which is being designed to have first-in-class potential."

Recent Corporate Highlights

RLY-2608 (ReDiscover study)

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RLY-2608 doublet: continued to enroll patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer in two additional dose expansion cohorts of RLY-2608 in combination with fulvestrant – a second 600mg BID cohort as well as one at 400mg BID
•
RLY-2608 triplet: continued enrollment of RLY-2608 + fulvestrant + ribociclib triplet combination in patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer
Lirafugratinib (RLY-4008; ReFocus study)

•
As previously disclosed, the company will minimize resource allocation in 2024 to allow data to mature and inform future clinical development decisions
Anticipated 2024 Milestones

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RLY-2608
o
RLY-2608 + fulvestrant data update in the second half of 2024
o
RLY-2608 + fulvestrant + ribociclib initial safety data in the second half of 2024
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Lirafugratinib: tumor agnostic data and regulatory update in the second half of 2024
•
Pre-clinical: disclose new program(s) in 2024

First Quarter 2024 Financial Results

Cash, Cash Equivalents and Investments: As of March 31, 2024, cash, cash equivalents and investments totaled $749.6 million compared to $750.1 million as of December 31, 2023. The company expects its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into the second half of 2026.

Revenue: Revenue was $10.0 million for the first quarter of 2024, as compared to $0.2 million for the first quarter of 2023. The increase was primarily due to a payment of $10.0 million in connection with a milestone achieved under the company’s Collaboration and License Agreement with Genentech, Inc. during the first quarter of 2024.

R&D Expenses: Research and development expenses were $82.4 million for the first quarter of 2024, as compared to $82.8 million for the first quarter of 2023. The decrease was primarily due to the impact of prioritization of certain programs in our pipeline, as previously disclosed in 2023 and earlier in 2024.

G&A Expenses: General and administrative expenses were $19.8 million for the first quarter of 2024, as compared to $19.6 million for the first quarter of 2023. The increase was primarily due to additional stock compensation expense.

Net Loss: Net loss was $81.4 million for the first quarter of 2024, or a net loss per share of $0.62, as compared to a net loss of $94.2 million for the first quarter of 2023, or a net loss per share of $0.78.