On February 27, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported financial results for the fourth quarter and full year periods ended December 31, 2023 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, FEB 27, 2024, View Source [SID1234640531]).

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"In 2023, OGSIVEO became the first FDA-approved therapy for adults with desmoid tumors with our approval in November and we are very pleased with the initial progress of our launch. We are also very excited by our opportunity in NF1-PN based on the positive topline data from our pivotal Phase 2b ReNeu trial, which demonstrated mirdametinib’s best-in-class potential for both children and adults with these devastating tumors. In addition, we made strong progress across our broader pipeline and continued to strengthen our financial position and our intellectual property protection for our lead assets," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for 2024 is to continue delivering a successful U.S. launch for OGSIVEO in desmoid tumors, to file our NDA for mirdametinib with the goal of having our second approval by 2025, to make progress towards expanding the reach of OGSIVEO into additional geographies outside of the U.S., and to unlock additional opportunities across our emerging portfolio."

Recent Business Highlights and Upcoming Milestones

OGSIVEO (Nirogacestat)

Received U.S. Food and Drug Administration (FDA) approval for OGSIVEO, an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment on November 27, 2023.
Launched OGSIVEO in the U.S. and achieved net product revenue of $5.4 million in the first partial quarter of the launch. To date, OGSIVEO has been reimbursed by payers representing over 98% of commercial lives, as well as by Medicare and Medicaid, with coverage aligned to the FDA-approved label.
Highlighted the update of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) in December 2023 which recommend nirogacestat as an NCCN Category 1, Preferred treatment option for desmoid tumors.
Submitted a Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors to the European Medicines Agency (EMA) in February 2024.
Presented additional patient-reported outcome data from the Phase 3 DeFi trial at the 2023 Connective Tissue Oncology Society Annual Meeting.
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

Presented positive topline data from the pediatric and adult cohorts of the Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in NF1-associated plexiform neurofibromas (NF1-PN) in November 2023. The confirmed objective response rate was 52% in pediatric patients and 41% in adult patients, as assessed by Blinded Independent Central Review, which was the primary endpoint of the study. Mirdametinib treatment also showed deep and durable responses and demonstrated significant improvements in key secondary patient-reported outcome measures. Mirdametinib was generally well tolerated in the trial, with the majority of adverse events (AEs) being Grade 1 or Grade 2. The most frequently reported AEs were rash, diarrhea, and vomiting in the pediatric cohort and rash, diarrhea, and nausea in the adult cohort.
On track to submit an New Drug Application (NDA) to the FDA for mirdametinib for the treatment of children and adults with NF1-PN in the first half of 2024.
Expect to present data from the pediatric and adult cohorts of the ReNeu trial at a medical congress in the first half of 2024 and to submit the trial results for publication 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.
The FDA cleared the Investigational New Drug (IND) application submitted by MapKure for a combination study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations. MapKure expects to initiate a Phase 1b trial in the first quarter of 2024.
Dose expansion cohort is ongoing in the BeiGene-sponsored Phase 1b/2 trial evaluating mirdametinib in combination with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with NRAS mutant solid tumors.
The FDA cleared the IND application for SW-682, a novel, oral, potent, and selective TEA Domain inhibitor designed to treat tumors driven by Hippo pathway mutations. SpringWorks plans to initiate a Phase 1a trial of SW-682 in Hippo mutant solid tumors in the first half of 2024.
General Corporate

Strengthened balance sheet with upsized public offering in December 2023; gross proceeds from the offering, before deducting underwriting discounts and commissions and offering expenses, were approximately $316.2 million.
The United States Patent and Trademark Office has recently issued four new patents for OGSIVEO and one new patent for mirdametinib, extending protection for both products into 2043. The U.S. patent portfolio for OGSIVEO includes 16 Orange Book listed patents; the U.S. patent portfolio for mirdametinib includes 10 patents that are expected to be Orange Book listed.
Fourth Quarter and Full Year 2023 Financial Results

Revenues: OGSIVEO net product revenues were $5.4 million in the fourth quarter of 2023, the first partial quarter of the U.S. launch.
Research and Development (R&D) Expenses: R&D expenses were $43.7 million and $150.5 million for the fourth quarter and full year 2023, respectively, compared to $37.9 million and $146.1 million for the comparable periods of 2022. The increase in R&D expense for the fourth quarter and year ended 2023 was primarily attributable to an increase in employee costs associated with head count growth, partially offset by a decrease in costs related to drug manufacturing, clinical trials and other research.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $59.8 million and $197.6 million for the fourth quarter and full year 2023, respectively, compared to $40.5 million and $134.6 million for the comparable periods of 2022. The increase in SG&A expense for the fourth quarter and the full year 2023 were largely attributable to commercial readiness activities to support the U.S. launch of OGSIVEO.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $94.3 million, or $1.44 per share, for the fourth quarter of 2023 and a net loss of $325.1 million, or $5.15 loss per share, for the year ended December 31, 2023. This compares to a net loss of $74.2 million, or $1.19 per share, for the fourth quarter of 2022 and a net loss of $277.4 million, or $5.21 per share for the year ended December 31, 2022.
Cash Position: Cash, cash equivalents and marketable securities were $662.6 million as of December 31, 2023.

On February 27, 2024 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, reported clinical results from its Phase 1/2 dose escalation study of NT219 in combination with cetuximab in the treatment of patients with recurrent/metastatic head and neck cancer (R/M SCCHN) (Press release, Purple Biotech, FEB 27, 2024, View Source [SID1234640530]).

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The data were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Targeted Anticancer Therapies (ESMO TAT) Congress 2024 in Paris on Monday, February 26, 2024 by Dr. Ari Rosenberg, Assistant Professor of Medicine at the University of Chicago, clinical investigator in the study, and member of Purple Biotech’s Head & Neck Cancer Scientific Advisory Board, in an oral presentation titled "Interim results of a Phase 1/2 trial of NT219 in combination with cetuximab in patients with advanced/metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN)".

The Phase 1/2 dose escalation study (NCT04474470) evaluated NT219 as a monotherapy in various indications and in combination with cetuximab in the treatment of R/M SCCHN and colorectal cancer.

As of cut-off date of January 25, 2024:

● Seventeen patients with R/M SCCHN were enrolled in the combination arm of NT219 + cetuximab. The median number of prior lines of therapy was 2 and 94% of the patients received prior immunotherapy.

● Safety profile was well tolerated and manageable including at 100 mg/kg. Most frequent treatment emergent adverse events (AEs) were infusion related reactions and nausea, and no treatment-related Grade 4/5 AEs were observed.

● Pharmacokinetic analysis demonstrated dose dependent increase in NT219 plasma concentrations.

● Fifteen patients were evaluable for efficacy, 7 of whom were at the relevant highest dose levels of 50 and 100 mg/kg in which anti-tumor activity was observed. Out of these 7 patients, 2 had confirmed partial responses and 3 stable diseases (all patients with partial response and stable disease have HPV negative disease), representing a 29% ORR and 71% DCR. Median follow-up across all dose levels is 9.4 months (95% CI: 3.4-10.0, 8 out of 15 patients remaining in follow up).

The Company recently reported NT219’s recommended Phase 2 dose of 100 mg/kg.

"We were encouraged to see anti-tumor activity in HPV negative patients," said Dr. Michael Schickler, Purple Biotech’s Head of Clinical and Regulatory Affairs. "There is an unmet medical need for patients in second- and third-line R/M SCCHN, most of them having HPV negative disease, with relatively short survival of less than nine months. NT219 should continue to be tested to establish better treatment options for this patient population."

"These positive data support our path forward for NT219 as we plan to initiate a Phase 2 study in combination with cetuximab in head and neck cancer in the first half of 2024," stated Gil Efron, Chief Executive Officer of Purple Biotech. "We thank the study participants, their families, and clinical researchers for participating in this important study."

About NT219

NT219 is a first-in-class, small molecule that promotes Insulin Receptor Substrates 1/2 (IRS) degradation and inhibits Signal Transducer and Activator of Transcription 3 (STAT3) phosphorylation, two major complementary signaling pathways that play a key role in the tumor and its microenvironment. IRS1/2 acts as scaffolds, organizing signaling complexes that mediate mitogenic, metastatic, angiogenic, and anti-apoptotic signals from IGF1R and other oncogenes, consisting of an important driver in multiple cancers and is highly involved in triggering drug resistance. STAT3 is a transcription factor that is broadly hyperactivated in many cancers, promoting proliferation, survival, angiogenesis, metastasis, and tumor immune evasion. Feedback activation of STAT3 plays a prominent role in mediating drug resistance to various anti-cancer therapies. As an inhibitor of both IRS1/2 and STAT3, NT219 has the potential to prevent the development of resistance to multiple approved therapies.

On February 27, 2024 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, reported that members of the management team will participate at two upcoming investor conferences (Press release, PureTech Health, FEB 27, 2024, View Source [SID1234640529]). Webcasts of the presentations will be available at View Source

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TD Cowen 44th Annual Health Care Conference

Presenters: Daphne Zohar, Founder and Chief Executive Officer, and Eric Elenko, Ph.D., Chief Innovation Officer

Date: Tuesday, March 5, 2024

Time: 1:30 p.m. EST

Leerink Partners Global Biopharma Conference

Presenters: Bharatt Chowrira, Ph.D., J.D., President, and Eric Elenko, Ph.D., Chief Innovation Officer

Date: Wednesday, March 13, 2024

Time: 10:40 a.m. EDT

On February 27, 2024 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the company will present a company overview at the following conferences (Press release, PTC Therapeutics, FEB 27, 2024, View Source [SID1234640528]):

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TD Cowen 44th Annual Health Care Conference
Monday, March 4, at 1:30 p.m. EST

Leerink Partners Global Biopharma Conference 2024
Monday, March 11, at 2:40 p.m. EDT

Barclays 26th Annual Healthcare Conference
Tuesday, March 12, at 4:05 p.m. EDT

The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On February 27, 2024 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical-stage biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, reported a corporate update and provided fourth quarter 2023 financial results (Press release, Pliant Therapeutics, FEB 27, 2024, View Source [SID1234640527]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"The progress made by our teams during the fourth quarter of 2023 position us well for executing upon our 2024 milestones including the recent positive data from the INTEGRIS-PSC trial that support the late-stage development of bexotegrast in this important indication," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "In 2024, our focus will be on the advancement the BEACON-IPF late-stage program, gathering clarity on next steps for a late-stage PSC program and providing updates to our early-stage portfolio including from our oncology and muscular dystrophy programs."

Fourth Quarter and Recent Highlights
Bexotegrast Highlights
•Positive safety and efficacy data from 320 mg dose cohort of INTEGRIS-PSC Phase 2a trial in patients with primary sclerosing cholangitis (PSC). At a once-daily dose of 320 mg, bexotegrast was well tolerated over 12 weeks of treatment with no drug-related severe or serious adverse events. At the 320 mg dose, bexotegrast reduced both Enhanced Liver Fibrosis (ELF) scores and PRO-C3 levels and showed improvements in hepatocyte function and bile flow by contrast MRI imaging relative to placebo at Week 12. Twenty-four-week data from the 320 mg dose group is expected in mid-2024.
•Enrollment continues in BEACON-IPF, a Phase 2b trial of bexotegrast in patients with idiopathic pulmonary fibrosis (IPF). BEACON-IPF is a 52-week, multinational, randomized, dose-ranging, double-blind, placebo-controlled trial evaluating bexotegrast at once-daily doses of 160 mg or 320 mg. BEACON-IPF is expected to enroll approximately 270 patients with IPF.
Pipeline Programs
•Phase 1 trial of PLN-101095 in solid tumors is enrolling. This is a Phase 1 open label trial of PLN-101095, an oral, small molecule, dual selective inhibitor of αvβ8 and αvβ1 integrins designed to block TGF-β activation in the tumor microenvironment. The trial is currently dosing the third of five planned dose cohorts in a Phase 1 open label dose-escalation trial of PLN-101095 as monotherapy and in combination with pembrolizumab in patients with solid tumors that are resistant to immune checkpoint inhibitors. Preliminary data is expected in late 2024.
•Muscular dystrophy program on track for regulatory filing in the first quarter of 2024. PLN-101325 is a monoclonal antibody designed to act as an allosteric agonist of integrin α7β1. Filing for first-in-human clinical studies in Duchenne muscular dystrophy (DMD) is expected in the first quarter of 2024.

Fourth Quarter 2023 Financial Results
•Research and development expenses were $33.2 million, as compared to $25.1 million for the prior-year quarter. The increase was due primarily to higher employee-related expenses and increased clinical and manufacturing-related costs associated with our lead program, bexotegrast.
•General and administrative expenses were $13.9 million, as compared to $14.3 million for the prior-year quarter. The decrease was primarily due to a decrease in stock-based compensation as we achieved a clinical development milestone in the fourth quarter of 2022 that triggered the vesting of certain performance based awards.
•Net loss was $41.1 million as compared to $35.1 million for the prior-year quarter. The increase was due to higher operating expenses coupled with a decrease in collaboration revenues under the Novartis collaboration during the quarter.
•As of December 31, 2023, the Company had cash, cash equivalents and short-term investments of $495.7 million which the Company expects, together with funds available under the Oxford Loan Agreement, to be sufficient to fund operations into the second half of 2026.