On May 6, 2021 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Crinetics Pharmaceuticals, MAY 6, 2021, View Source [SID1234579457]).

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"In the first quarter we achieved key clinical and regulatory milestones across our pipeline," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Based on our interactions with the FDA and other regulators, we developed a robust Phase 3 program for paltusotine that is designed to support its approval for all acromegaly patients who require pharmacotherapy, including both untreated patients and those switching from injected standard of care. This progress was complemented by the advancement of CRN04894 and CRN04777 into Phase 1 trials in healthy volunteers, which are designed to generate safety, pharmacokinetic, and biomarker data that could establish clinical proof of concept and provide important dose guidance information for subsequent trials in patients. Looking ahead, we are on track for a steady cadence of catalysts through the remainder of the year, including data from both of our Phase 1 trials and the initiation of a clinical study in patients with neuroendocrine tumors complicated by carcinoid syndrome."

First Quarter and Subsequent Highlights

Announced the design of the Phase 3 program for paltusotine in acromegaly. In March 2021, Crinetics announced plans to initiate two Phase 3 studies based on interactions with the U.S. Food and Drug Administration (FDA) and other regulators. The first of these studies, PATHFNDR-1, is a double-blind, placebo-controlled, nine-month clinical trial evaluating the safety and efficacy of paltusotine in acromegaly patients who are biochemically controlled (IGF-1 ≤ 1.0 × upper limit of normal [ULN]) and who are on stable doses of somatostatin receptor ligand monotherapy (octreotide LAR or lanreotide depot). The second study, PATHFNDR-2, is a double-blind, placebo-controlled, twelve-week trial in acromegaly patients with elevated IGF-1 levels who are medication naïve or who are not being treated with pharmacotherapy (untreated patients). If successful, Crinetics believes these trials could support registration of paltusotine in the United States and Europe for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care. The company initiated PATHFNDR-1 in April and expects to initiate PATHFNDR-2 in 2H 2021.
Showcased broad clinical-stage pipeline at ENDO 2021. In March 2021, Crinetics gave presentations on its three clinical programs at the Endocrine Society’s annual ENDO 2021 congress. Posters on the company’s acromegaly program included a summary of the previously announced ACROBAT Edge Phase 2 results, as well as details of the new tablet formulation of paltusotine. Presentations related to the company’s earlier stage clinical programs included a poster with preclinical data supporting the development of CRN04777 as a treatment for congenital hyperinsulinism (HI) and a live oral presentation with preclinical evidence supporting the further evaluation of CRN04894 in Cushing’s disease and congenital adrenal hyperplasia (CAH).
Advanced ACTH antagonist CRN04894 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04894, an investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of diseases associated with excess ACTH such as Cushing’s disease and congenital adrenal hyperplasia (CAH). This study is designed to evaluate the safety and tolerability of CRN04894 in healthy volunteers, and to provide clinical proof-of-concept data by measuring the effect of CRN04894 on the suppression of endocrine biomarkers that are used as key endpoints in patient studies and reflect the ability of CRN04894 to block ACTH-stimulated adrenal function. This healthy volunteer trial is also expected to provide important information for dose selection and be predictive of efficacy in Cushing’s disease and CAH patients. Crinetics expects preliminary safety, pharmacokinetic, and endocrine biomarker data in mid-2021.
Advanced SST5 agonist CRN04777 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist. CRN04777 is being developed as a treatment for congenital HI, a rare genetic disease in which excess insulin secretion causes life-threatening hypoglycemia (low blood glucose). This study is designed to evaluate the safety and tolerability of CRN04777 in healthy volunteers and provide clinical proof-of-concept data by measuring both the ability of CRN04777 to inhibit glucose- and sulfonylurea-induced insulin secretion and the corresponding effects on blood glucose levels. These endocrine biomarkers are indicative of the ability of CRN04777 to prevent hypoglycemia and are expected to provide information for dose selection and be predictive of efficacy in patients with hyperinsulinism. Crinetics expects preliminary safety and pharmacological effect data in mid-2021.
Strengthened balance sheet with successful common stock offering. In April 2021, Crinetics completed an underwritten follow-on offering and raised net proceeds of approximately $72.5 million.
First Quarter 2021 Financial Results

Research and development expenses were $17.6 million for the three months ended March 31, 2021, compared to $13.9 million for the same period in 2020. The increase was primarily attributable to additional personnel costs and clinical development and manufacturing activities for paltusotine, CRN04894, and CRN04777 as well as the company’s other preclinical programs.
General and administrative expenses were $5.3 million for the three months ended March 31, 2021, compared to $4.0 million for the same period in 2020. The increase was primarily due to additional personnel costs to support the company’s growth.
Net loss for the three months ended March 31, 2021 was $22.9 million, compared to a net loss of $17.4 million for the three months ended March 31, 2020.
Unrestricted cash, cash equivalents and investments totaled $150.7 million as of March 31, 2021, compared to $170.9 million as of December 31, 2020. The cash balance at the end of March does not include the $72.5 million of net proceeds from the follow-on offering completed in April.
As of April 30, 2021, the company had 37,593,371 common shares outstanding.

On May 6, 2021 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the first quarter ended March 31, 2021 (Press release, X4 Pharmaceuticals, MAY 6, 2021, View Source [SID1234579444]). The company also provided an update on its lead product candidate, mavorixafor, a novel small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials for patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN), respectively.

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"We are excited by the strong progress we have achieved to date in 2021 in the development of mavorixafor across multiple rare disease indications," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "In addition, we recently completed an at-the-market PIPE financing that provided $55 million in gross proceeds to the company. We believe that the significant level of new and existing investor interest demonstrates confidence in the clinical and commercial potential of mavorixafor while also extending our expected cash runway into late 2022, as well as supporting our additional pipeline programs. We look forward to continuing to report both clinical enrollment and data milestones throughout the rest of 2021 and beyond."

Recent Highlights
•Mavorixafor Phase 1b Data in Waldenström’s to be Presented at EHA (Free EHA Whitepaper) 2021. X4 has received notification of acceptance of an abstract for a poster presentation at this year’s annual congress of the European Hematology Association (EHA) (Free EHA Whitepaper), taking place virtually from June 9-17, 2021. The abstract and poster will include initial clinical safety and efficacy data from the ongoing Phase 1b clinical trial of mavorixafor in combination with ibrutinib in the treatment of a subset of Waldenström’s macroglobulinemia patients with mutations to both the MYD88 and CXCR4 genes.
•Enrollment Update in 4WHIM Clinical Trial Expected to be Announced in Mid-2021. The 4WHIM Phase 3 trial is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in approximately 18-28 genetically confirmed WHIM patients over the course of a 52-week study. The primary endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold in response to treatment with mavorixafor versus placebo. Secondary endpoints will assess infection rates, wart burden, and markers of immune system function and quality of life among others. Based on the current rate of enrollment, X4 expects to provide an important enrollment update on the trial in the second or third quarter of 2021.
•Raised $55.0 Million in an At-the-Market PIPE Financing. In March 2021, X4 priced a $55 million at-the-market private placement financing at $8.70 per common share. The financing transaction included participation from leading biotechnology investors that are both new and existing X4 shareholders. The financing extended the company’s guidance on its expected cash runway into the fourth quarter of 2022.

First Quarter 2021 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $116.7 million in cash, cash equivalents and restricted cash as of March 31, 2021. X4 expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
•Research and Development Expenses were $12.1 million for the first quarter ended March 31, 2021, as compared to $8.9 million for the comparable period in 2020. R&D expenses include $0.6 million and $0.2 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.
•General and Administrative Expenses were $5.8 million for the first quarter ended March 31, 2021, as compared to $4.7 million for the comparable period in 2020. G&A expenses include $0.7 million and $0.4 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.
•Net Loss: X4 reported a net loss of $18.7 million for the quarter ended March 31, 2021, as compared to a net loss of $11.1 million for the comparable period in 2020. Net losses include $1.3 million and $0.6 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.

Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 5658628. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On May 6, 2021 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) reported its first quarter 2021 financial results and highlights (Press release, Synta Pharmaceuticals, MAY 6, 2021, View Source [SID1234579443]).

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"We expect several important events for Madrigal to occur in 2021," stated Paul Friedman, M.D., Chief Executive Officer of Madrigal. "These include completing enrolling a sufficient number of subjects into the MAESTRO-NASH 52-week, serial liver biopsy population, which we expect to accomplish by the end of the second quarter, to support a future application for accelerated approval to FDA; we also expect to release topline 52 week data from the blinded arms of MAESTRO-NAFLD-1 by the end of the year."

Dr. Friedman continued, "As the competitive landscape has evolved in the sector, we believe Madrigal has emerged as a leader in the race to develop and commercialize a drug to treat NASH."

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, "We expect the open label arms of MAESTRO-NAFLD-1 will continue to generate compelling results, including data from open label patients treated with resmetirom for 52 weeks that we hope to present at major medical conferences this year."

Dr. Taub continued, "We are enthusiastic about the growing data set from leading researchers and clinical investigators to support generally accepted, noninvasive diagnostic approaches to identify and monitor patients with NASH and significant liver fibrosis."

Financial Results for the Three Months Ended March 31, 2021
As of March 31, 2021, Madrigal had cash, cash equivalents and marketable securities of $307.2 million, compared to $284.1 million at December 31, 2020. The increase in cash and marketable securities resulted primarily from net proceeds of $66.6 million from sales of common stock via the Company’s at-the-market sales agreement, which were partially offset by cash used in operations of $43.4 million.

Operating expenses were $53.0 million for the three month period ended March 31, 2021, compared to $38.0 million in the comparable prior year period.

Research and development expenses for the three month period ended March 31, 2021 were $45.8 million, compared to $33.4 million in the comparable prior year period. The increase is attributable primarily to additional activities related to the Phase 3 clinical trials, and an increase in head count.

General and administrative expenses for the three month period ended March 31, 2021 were $7.2 million, compared to $4.6 million in the comparable prior year period. The increase in general and administrative expenses for the latest three month period is due primarily to increases in commercial preparation activities, including an increase in headcount.

Interest income for the three month period ended March 31, 2021 was $0.2 million, compared to $1.9 million in the comparable prior year period. The decrease in interest income for the latest three month period was due primarily to lower average principal balances in our investment accounts in 2021, and decreased interest rates.

About Resmetirom (MGL-3196)
Thyroid hormone, through activation of its β-receptor in hepatocytes, plays a central role in liver function impacting a range of health parameters from levels of serum cholesterol and triglycerides to the pathological buildup of fat in the liver. Thyroid hormone receptor (THR)-β action in the liver is key to proper function of the liver, including regulation of mitochondrial activity such as breakdown of liver fat and control of the level of normal, healthy mitochondria. Patients with NASH have reduced levels of thyroid hormone activity in the liver with resultant impaired hepatic function, in part due to the inflamed state of the liver that causes degradation of thyroid hormone.

To exploit the thyroid hormone receptor (THR)-β pathway for therapeutic purposes in cardio-metabolic and liver diseases, it is important to avoid activity at the THR-α receptor, the predominant systemic receptor for thyroid hormone that is responsible for activity outside the liver including in heart and bone. The lack of selectivity of older thyromimetic compounds, chemically-related toxicities and undesirable distribution in the body led to safety concerns. Madrigal recognized that greater selectivity for thyroid hormone receptor (THR)-β and liver targeting might overcome these challenges and deliver the full therapeutic potential of THR-β agonism. Resmetirom has been shown to be highly selective based on 1) THR-β receptor functional selectivity based on both in vitro and in vivo assays and 2) specific uptake into the liver, its site of action, virtually avoiding any uptake into tissues outside the liver. In short and long term human and animal studies, resmetirom has been confirmed to be safe and devoid of activity at the THR-α receptor and without impact on bone or cardiac parameters. Resmetirom does not impact the thyroid axis hormones, including the central thyroid axis. Madrigal believes that resmetirom is the first orally administered, small-molecule, liver-directed, truly β-selective THR agonist.

About the Phase 3 Registration Program for the Treatment of NASH (Non-alcoholic steatohepatitis)
Analyses from the resmetirom Phase 2 NASH study demonstrate that the magnitude of liver fat reduction accurately predicts NASH resolution and liver fibrosis reduction and, specifically, that the resmetirom doses being used in Madrigal’s Phase 3 MAESTRO-NASH trial could achieve the level of fat reduction predictive of NASH resolution and fibrosis reduction [Madrigal COVID and ABSTRACT Press Release_20200414].

The Phase 3 MAESTRO-NASH trial is initially expected to enroll 900 patients with biopsy-proven NASH (fibrosis stage 2 or 3), randomized 1:1:1 to receive resmetirom 80 mg once a day, 100 mg once a day, or placebo. After 52 weeks of treatment a second biopsy is performed. The primary surrogate endpoint on biopsy will be NASH resolution, with at least a 2-point reduction in NAS (NASH Activity Score), and with no worsening of fibrosis. Two key secondary endpoints are liver fibrosis improvement of at least one stage, with no worsening of NASH, and lowering of LDL-cholesterol [ClinicalTrials.gov/NCT03900429].

A second 52-week Phase 3 multi-center, double-blind, randomized, placebo-controlled study of resmetirom, MAESTRO-NAFLD-1, was initiated in December 2019 in 700 patients with non-alcoholic fatty liver disease (NAFLD), presumed NASH, randomized 1:1:1 to receive resmetirom 80 mg once a day, 100 mg once a day, or placebo. MAESTRO-NAFLD-1 also includes a 100 mg resmetirom open label arm in up to 100 patients. The trial was expanded to include more than 1,200 patients, in order to significantly enhance resmetirom’s safety database and provide further opportunity to study selected patient subgroups. Unlike MAESTRO-NASH, MAESTRO-NAFLD-1 is a non-biopsy study and represents a "real-life" NASH study. NASH or presumed NASH is documented using historical liver biopsy or non-invasive techniques including fibroscan and MRI-PDFF. Using non-invasive measures, MAESTRO-NAFLD-1 is designed to provide incremental safety information to support the NASH indication as well as provide additional data regarding clinically relevant key secondary efficacy endpoints to better characterize the potential clinical benefits of resmetirom on cardiovascular and liver related endpoints. These key secondary endpoints include LDL-cholesterol, apolipoprotein B and triglyceride (TG) lowering; reduction of liver fat as determined by magnetic resonance imaging, proton density fat fraction (MRI-PDFF); and reduction of PRO-C3, a NASH fibrosis biomarker. [ClinicalTrials.gov/NCT04197479] Additional secondary and exploratory endpoints will be assessed including reduction in liver enzymes, fibroscan scores and other fibrosis and inflammatory biomarkers.

These and other data, including safety parameters, form the basis for potential subpart H submission to FDA for accelerated approval for the treatment of NASH. The original 900 patients in the MAESTRO-NASH study will continue on therapy after the initial 52-week treatment period; up to another 1,100 patients are to be added using the same randomization plan and the study is expected to continue for up to 54 months to accrue and measure clinical events, most relevantly progression to cirrhosis.

About Resmetirom’s Potential to Confer Cardiovascular Risk Reduction in NASH patients
Additionally, resmetirom lowers multiple atherogenic lipids, including LDL cholesterol, apolipoprotein B, triglycerides, and lipoprotein (a), as demonstrated in Phase 2, a key differentiating factor compared with other NASH therapeutics. The magnitude of reduction of these lipids support a potential indication for treatment of hyperlipidemia in NASH patients and predicts a potential for benefit on cardiovascular (CV) events in NASH patients who die most frequently of CV, not liver disease.

Because of their diabetes, dyslipidemia, hypertension, obesity in concert with an inflamed, fatty liver, NASH patients, particularly those with advanced fibrosis, are at a substantially increased CV risk compared to the general population. Resmetirom’s ability to decrease liver fat, which is an independent risk factor for CV events, and resmetirom’s effect to reduce atherogenic lipids are being further evaluated in several key secondary endpoints in both MAESTRO Phase 3 clinical studies.

On May 6, 2021 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported first quarter financial results for the period ended March 31, 2021 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, MAY 6, 2021, View Source [SID1234579442]).

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"In the first quarter of 2021 we continued to execute on our 10 clinical programs and grew our portfolio of opportunities aimed at serving a broad group of patients across our three distinct oncology segments: late-stage rare oncology, hematological cancers and biomarker-defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are poised for multiple important data readouts this year, including topline data from our Phase 3 DeFi trial, initial clinical data from our BCMA combination trial of nirogacestat with GSK’s BLENREP in multiple myeloma and initial clinical data from our metastatic solid tumor trials being conducted in collaboration with BeiGene. I look forward to reporting on our continued progress throughout 2021."

Recent Business Highlights and Upcoming Milestones

Late-Stage Rare Oncology

In February 2021, SpringWorks reported interim data from the adult stratum of the ongoing potentially registrational Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas. Of the first 20 adult patients enrolled, 50% had achieved an objective response, the primary endpoint of the study, as assessed by blinded independent central review, and 16 of these 20 patients (80%) remained on study as of the January 22, 2021 data cutoff. In addition, mirdametinib was generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2 and only one Grade 3 TRAE reported; there have been no Grade 4 or 5 TRAEs reported. The Company expects to report additional clinical data from the ReNeu trial at a medical conference this year and to complete enrollment of the trial in the second half of 2021.
SpringWorks expects to report topline data from the Phase 3 DeFi trial in the second half of 2021, as previously disclosed.
Recruitment is ongoing in a Phase 2 study sponsored by the Children’s Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.
B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

Enrollment is ongoing in three Phase 1 studies evaluating nirogacestat in combination with anti-B-cell maturation antigen (BCMA) therapies in adult patients with relapsed or refractory multiple myeloma: a Phase 1b trial sponsored by GSK evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), a Phase 1 study sponsored by Allogene evaluating nirogacestat in combination with ALLO-715, and a Phase 1 study sponsored by Janssen Research & Development, LLC (Janssen) evaluating nirogacestat in combination with teclistamab. Initial data from the GSK-sponsored study are expected in 2021. SpringWorks also expects that two additional collaborator-sponsored trials will initiate in the first half of 2021, as previously disclosed: nirogacestat + Pfizer’s elranatamab and nirogacestat + Precision Biosciences’ PBCAR269A.
In March 2021, SpringWorks entered into a services agreement with ONCOtracker, Inc., a leading innovation center for novel cancer treatment and testing with a focus on the diagnosis and treatment of multiple myeloma, other B-cell malignancies, and related disorders, to explore the effect of nirogacestat on soluble BCMA release and membrane-bound BCMA receptor density in primary bone marrow samples collected from multiple myeloma patients. This research is intended to further confirm nirogacestat’s ability to potentiate BCMA by using ex vivo patient-derived models with associated longitudinal clinical characterization for each sample.
Biomarker-Defined Metastatic Solid Tumors

In May 2021, SpringWorks entered into an exclusive worldwide license agreement with Katholieke Universiteit Leuven (KU Leuven) and the Flanders Institute for Biotechnology (VIB) for the in-license of a portfolio of novel small molecule inhibitors of the TEA Domain (TEAD) family of transcription factors, designed for the potential treatment of biomarker-defined solid tumors driven by aberrant Hippo pathway signaling. The licensed portfolio includes advanced lead compounds and multiple backup compounds from diverse chemical series, which were discovered at KU Leuven’s Center for Drug Design and Discovery (CD3) in collaboration with Professor Georg Halder of the VIB-KU Leuven Center for Cancer Biology. SpringWorks expects to nominate a development candidate from this portfolio and move into IND-enabling studies in 2022.
Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. BeiGene is sponsoring this trial and SpringWorks and BeiGene expect to report initial clinical data in 2021, as previously disclosed.
Enrollment is ongoing in a Phase 1 trial of BGB-3245 in adult patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene. Initial clinical data from the MapKure-sponsored Phase 1 trial are expected in 2021, as previously disclosed.
In March 2021, SpringWorks entered into a research collaboration agreement with Inserm Transfert, the private subsidiary of Inserm, acting as a delegatee of the French National Institute of Health and Medical Research (Inserm) to explore the ability of nirogacestat to delay the development of resistance to the EGFR inhibitor osimertinib in preclinical models of non-small cell lung cancer (NSCLC) driven by the EGFR C797S gatekeeper mutation. This research, which will be led by Antonio Maraver, Ph.D. at Montpellier Cancer Research Institute (IRCM U1194), is intended to build on previous in vivo work from Dr. Maraver’s lab demonstrating the potential for gamma secretase inhibition to enhance the activity of osimertinib in EGFR-driven models of NSCLC.1
General Corporate

In March 2021, Bhavesh Ashar was appointed Chief Commercial Officer of SpringWorks. Mr. Ashar has more than 20 years of global pharmaceutical and biotechnology experience, most recently having served as Senior Vice President, General Manager of U.S. Oncology at Bayer Healthcare where he was responsible for a broad portfolio in prostate, liver, colorectal, GIST, hematologic and tumor-agnostic biomarker driven cancers.
First Quarter 2021 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $17.4 million for the first quarter, compared to $9.7 million for the comparable period of 2020. The increases in R&D expenses were primarily attributable to an increase in external costs related to drug manufacturing and trial costs, and an increase in internal costs driven by the growth in employee costs associated with increases in the number of R&D personnel and an increase in stock-based compensation expense.
General and Administrative (G&A) Expenses: G&A expenses were $12.4 million for the first quarter, compared to $6.4 million for the comparable period of 2020. The increases in G&A expenses were primarily attributable to the hiring of additional personnel in G&A functions supporting the growth of the organization, as well as an increase in stock-based compensation expense.
Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $29.8 million, or $0.62 per share, for the first quarter of 2021. This compares to a net loss of $15.3 million, or $0.37 per share, for the comparable period of 2020.
Cash Position: Cash, cash equivalents and marketable securities were $541.0 million as of March 31, 2021.
COVID-19 Update

To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks’ business operations, in particular on SpringWorks’ clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks’ periodic filings with the Securities and Exchange Commission and refer to the Forward-Looking Statements section in this press release.

On May 6, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the first quarter of 2021 (Press release, Kadmon, MAY 6, 2021, View Source [SID1234579440]).

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"We continue to ramp up commercial launch preparation activities for belumosudil in anticipation of the PDUFA goal date of August 30, 2021. Labeling discussions with the FDA are progressing and we believe that belumosudil, if approved, will serve a critical, unmet need for patients with cGVHD," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "In addition, we are exploring the therapeutic potential of belumosudil in systemic sclerosis, a disease with similar manifestations to cGVHD. We continue to enroll patients in two separate Phase 2 studies in this indication and expect to present initial data from the open-label study by year-end 2021. In parallel, we are advancing the ongoing Phase 1 clinical trial of KD033, our anti-PD-L1/IL-15 fusion protein. We look forward to sharing initial safety data from this trial at ASCO (Free ASCO Whitepaper) in June 2021 and additional clinical data in the fourth quarter of 2021."

2021 Anticipated Key Clinical Milestones:

Belumosudil in chronic graft-versus-host disease (cGVHD)

Continue commercial launch readiness activities in anticipation of the Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2021
Belumosudil in diffuse subcutaneous systemic sclerosis (dcSSc)

Continue enrollment in the open-label Phase 2 clinical trial of belumosudil in patients with dcSSc (KD025-215); the Company plans to present initial data from the trial by year-end 2021
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in dcSSc (KD025-209)
KD033

Present initial safety data from the ongoing Phase 1 clinical trial of KD033, the Company’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held June 4-8, 2021; additional clinical data from the trial are expected to be available in Q4 2021
Enrollment in the first two dose cohorts of the KD033 Phase 1 trial (KD033-101) was successfully completed; enrollment is ongoing in the next dose level (cohort 3)
Financial Results

First Quarter 2021 Results

Loss from operations for the three months ended March 31, 2021 was $27.4 million compared to $16.1 million for the same period in 2020, which included $6.0 million in one-time license revenues related to the Meiji strategic partnership.

The $5.6 million increase in operating expenses for the three months ended March 31, 2021 as compared to 2020 was primarily related to belumosudil commercial launch readiness activities, as well as research and development costs for KD033 and our preclinical product candidates.

Liquidity and Capital Resources

At March 31, 2021, the Company’s cash, cash equivalents and marketable debt securities totaled $295.9 million, compared to $123.9 million at December 31, 2020.

About Belumosudil

Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. The FDA granted Priority Review for the New Drug Application (NDA) for belumosudil for the treatment of cGVHD and has assigned a PDUFA goal date of August 30, 2021. The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) and Project Orbis pilot programs. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy. The FDA has also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD and for the treatment of systemic sclerosis.