On February 25, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2020 (Press release, Agios Pharmaceuticals, FEB 25, 2021, View Source [SID1234575673]).

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"This past year was a transformative one for Agios," said Jackie Fouse, Ph.D., chief executive officer at Agios. "Despite the extraordinary challenges brought on by the global COVID-19 pandemic and civil and political unrest in the U.S., we remain hopeful for the future of our country and the promise of our industry and more confident than ever in our ability to execute on our plans on behalf of patients. The sale of our oncology business to Servier on attractive terms both allows our oncology portfolio to grow and flourish and facilitates our new laser focus on genetically defined diseases, where we anticipate a catalyst-rich year ahead for mitapivat across our three initial disease indications. In addition to our plans to file for approval for mitapivat in adults with PK deficiency in the U.S. and EU and launching our Phase 3 thalassemia trials later this year, we’re pleased to share our pivotal strategy for mitapivat in sickle cell disease which we believe will enable us to pursue a broad label for patients who desperately need new treatment options."

FOURTH QUARTER 2020 & RECENT HIGHLIGHTS

Entered into definitive agreement to sell commercial, clinical and research-stage oncology portfolio to Servier, an independent global pharmaceutical company, in December.
Achieved TIBSOVO (ivosidenib tablets) net sales of $39.1 million for the quarter and $121.1 million for the year, exceeding the $115 million updated net revenue target and representing a 102% increase in net sales year-over-year.
Announced topline results from two Phase 3 studies of mitapivat in adults with pyruvate kinase (PK) deficiency who were not regularly transfused (ACTIVATE) and who were regularly transfused (ACTIVATE-T ). Both studies achieved statistical significance for their primary endpoint with ACTIVATE demonstrating a clinically meaningful sustained increase in hemoglobin compared to placebo and ACTIVATE-T demonstrating a clinically meaningful reduction in transfusion burden. Statistical significance was also achieved for all pre-specified key secondary endpoints for ACTIVATE demonstrating an improvement compared to placebo, including in patient-reported outcomes (PRO) based on changes from baseline in pyruvate kinase deficiency diary (PKDD) score and pyruvate kinase deficiency impact assessment (PKDIA) score at Week 24. The safety profile was consistent with results from prior studies.
Completed U.S. and EU regulatory interactions on the pivotal development plan for mitapivat in sickle cell disease, resulting in the Phase 2/3 trial design being announced today.
The Phase 2 will randomize 69 patients 1:1:1 to 50 mg mitapivat BID, 100 mg mitapivat BID or matched placebo. The primary endpoint is hemoglobin response defined as ≥1 g/dL change from baseline to Week 12.
The Phase 3 will randomize 198 patients 2:1 to the selected Phase 2 dose of mitapivat or matched placebo. The study includes two primary endpoints: hemoglobin response defined as ≥1 g/dL change from baseline to Week 52 and annualized rate of sickle cell pain crises.
Potential regulatory approval with a broad label based on the operationally seamless Phase 2/3 trial expected by the end of 2026.
Appointed Darrin Miles, previously senior vice president, U.S. commercial and global marketing, to role of chief commercial officer.
Presented final data, including mature overall survival (OS) results, from the Phase 3 ClarIDHy study of TIBSOVO in patients with previously treated isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI) in January.
Presented updated Phase 1 data for mitapivat in sickle cell disease at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December.
Launched Anemia ID, a program providing no-cost genetic testing for patients with suspected hereditary anemias, including PK deficiency in November.
ANTICIPATED 2021 KEY MILESTONES

Corporate

Complete sale of oncology portfolio to Servier in a transaction worth up to $2 billion plus royalties, following a shareholder vote on March 25, and execute a meaningful portion of the planned $1.2 billion capital return by year-end.
Genetically Defined Diseases

File for regulatory approval for mitapivat in adults with PK deficiency: submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021.
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end.
Genetically Defined Disease Data Presentations

Submit data from the following clinical studies for presentation at the EHA (Free EHA Whitepaper) Virtual Congress, hosted June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings throughout 2021.
Present data from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end.
Oncology

Submit supplemental new drug application (sNDA) in the U.S. for TIBSOVO in patients with previously treated IDH1-mutant cholangiocarcinoma in Q1 2021.
Enrollment in the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1-mutant acute myeloid leukemia is expected to complete by year-end.
Enrollment in the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1-mutant advanced hematologic malignancies is expected to complete by year-end.
Full-year 2021 net product revenue for TIBSOVO is expected to be $160-170 million.
FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS

Revenue: Total revenue for the fourth quarter of 2020 was $44.0 million, which includes $2.0 million in collaboration revenue, $39.1 million of net product revenue from sales of TIBSOVO and $2.9 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to $35.4 million for the fourth quarter of 2019, which included $12.9 million in collaboration revenue, $19.6 million of net product revenue from U.S. sales of TIBSOVO and $3.0 million in royalty revenue from net global sales of IDHIFA. Total revenue for the year ended December 31, 2020 was $203.2 million compared to $117.9 million for the year ended December 31, 2019. The increase in 2020 revenue was primarily driven by a 102% increase in TIBSOVO net product revenue and higher collaboration revenue due to recognition of the remainder of the deferred revenue balance related to the completion of the metabolic immuno-oncology collaboration with Celgene Corporation, a wholly owned subsidiary of Bristol Myers Squibb Company.

Cost of Sales: Cost of sales were $1.0 million for the fourth quarter of 2020 compared to $0.3 million for the fourth quarter of 2019, and $2.8 million for the year ended December 31, 2020 compared to $1.3 million for the year ended December 31, 2019.

Research and Development (R&D) Expenses: R&D expenses were $95.7 million for the fourth quarter of 2020 compared to $106.2 million for the fourth quarter of 2019 and $367.5 million for the year ended December 31, 2020 compared to $410.9 million for the year ended December 31, 2019. The decrease in R&D expense was primarily attributable to a decrease in TIBSOVO clinical development costs, including winding down the ClarIDHy Phase 3 study.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $39.8 million for the fourth quarter of 2020 compared to $34.8 million for the fourth quarter of 2019, and $149.1 million for the year ended December 31, 2020 compared to $132.0 million for the year ended December 31, 2019. The increase in SG&A expense was primarily attributable to increased workforce expenses and professional fees related to the Servier transaction, partially offset by a decrease in external spending due to cost savings initiatives and reduced employee travel related expenses due to restrictions.

Net Loss: Net loss was $97.7 million for the fourth quarter of 2020 compared to $102.4 million for the fourth quarter of 2019, and $327.4 million for the year ended December 31, 2020 compared to a net loss of $411.5 million for the year ended December 31, 2019.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2020 were $670.5 million compared to $717.8 million as of December 31, 2019. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020, together with anticipated product and royalty revenue, interest income and expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its planned operating expenses and capital expenditure requirements to the end of 2022. Following the closing of the Servier transaction and net of the planned capital return, Agios expects to be able to fund its operation through major catalysts and to cash-flow positivity without the need to raise additional equity.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2020 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 8442238. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On February 25, 2021 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year of 2020 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 25, 2021, View Source [SID1234575672]).

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"As we reflect on 2020, we are proud of how we have progressed our immuno-inflammatory development programs," said Dr. Neal Walker, President & CEO of Aclaris. "We are very excited about the platform potential of ATI-450, an oral MK2 inhibitor which we have shown to inhibit TNFα, IL1β, and IL6. Given its novel mechanism, there are several immuno-inflammatory indications that ATI-450 may potentially address. We are also excited about the potential of our pipeline which is internally generated from KINect, our proprietary drug discovery platform, for various immuno-inflammatory indications. We look forward to building on this momentum in 2021."

Research and Development Highlights:

The global COVID-19 pandemic continues to rapidly evolve and has caused and may continue to cause Aclaris to experience disruptions that could impact the timing of its research and development and regulatory activities listed below.

ATI-450, an investigational oral small molecule MK2 inhibitor compound:

ATI-450-RA-201: A Phase 2a, multicenter, randomized, investigator and patient-blind, sponsor-unblinded, parallel group, placebo-controlled clinical trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-450 in subjects with moderate to severe rheumatoid arthritis.
Positive preliminary topline data announced in January 2021. In this trial, ATI-450 demonstrated durable clinical activity, as defined by a marked and sustained reduction in DAS28-CRP and improvement of ACR20/50/70 responses over 12 weeks. This clinical activity was further supported by pharmacodynamic analyses showing a marked and durable inhibition of TNFα, IL1β, IL6, and IL8 in ex vivo stimulated samples as well as an endogenous inflammation biomarker analysis which also demonstrated a marked and sustained inhibition of median concentrations of hsCRP, TNFα, IL6, IL8, and MIP1β in the treatment arm over the 12 week period.
ATI-450 was generally well tolerated. The most common adverse events (AE) (each reported in 2 subjects) were urinary tract infection (UTI), elevated lipids and ventricular extrasystoles, all of which were determined to be unrelated to treatment except for one UTI. There was one non-treatment-related serious adverse event (COVID-19) reported in the four-week safety follow-up phase of the trial in a subject who was no longer receiving treatment.
Aclaris intends to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the second half of 2021.
Aclaris is currently evaluating additional potential indications driven by TNFα, IL1β and IL6 as part of its planned expansion of its Phase 2 immuno-inflammatory clinical development programs.

ATI-450-PKPD-102: A Phase 1, placebo-controlled, randomized, observer-blind clinical trial to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ATI-450 at 80 mg and 120mg twice daily in health subjects.
Positive preliminary topline data announced in January 2021. Pharmacodynamic analysis demonstrated incremental cytokine suppression at these higher doses. ATI-450 was generally well tolerated. The most common AEs (reported by 2 or more subjects who received ATI-450) were headache, dizziness, nausea, parasthesia and, in the post-dosing safety follow-up phase of the trial, dry skin. These AEs were all mild in severity.
A final analysis of this trial is underway.

ATI-450-CAPS-201: A Phase 2a, multicenter, open-label, single-arm clinical trial to investigate the safety, tolerability, efficacy and pharmacodynamics of ATI-450 for the maintenance of remission in subjects with cryopyrin-associated periodic syndrome (CAPS) previously managed with anti-IL1 therapy. Due to the COVID-19 pandemic, subject enrollment in this trial was paused. As a result of the ongoing pandemic and given the positive preliminary topline data from the ATI-450-RA-201 trial, Aclaris has decided to focus its efforts and resources on other immuno-inflammatory diseases.
ATI-1777, an investigational topical "soft" Janus Kinase (JAK) 1/3 inhibitor compound:

ATI-1777-AD-201: An ongoing Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-group clinical trial to investigate the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with moderate to severe atopic dermatitis. The primary endpoint is the percentage change from baseline in the Eczema Area and Severity Index (EASI) score at week 4. Data from this trial are expected mid-year 2021.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound:

Aclaris is developing ATI-2138 as a potential treatment for T-cell mediated diseases such as psoriasis and/or inflammatory bowel disease and expects to submit an Investigational New Drug Application for ATI-2138 in the second half of 2021.
Financial Highlights:

Liquidity and Capital Resources

As of December 31, 2020, Aclaris had aggregate cash, cash equivalents, restricted cash and marketable securities of $54.1 million compared to $75.0 million as of December 31, 2019. The changes in cash and cash equivalents and marketable securities during the year included:

Net cash used in operating activities was $38.6 million resulting from net loss of $51.0 million and changes in operating assets and liabilities of $2.4 million, partially offset by non-cash adjustments of $14.7 million.

Net borrowings of $10.9 million pursuant to a Loan and Security Agreement.

Net proceeds of $7.7 million from the sale of 2.1 million shares of common stock under an equity line of credit agreement.
In January 2021, Aclaris closed a public offering in which it sold approximately 6.3 million shares of common stock. Proceeds from the offering were $103.5 million, net of underwriting discounts, commissions and offering expenses. Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2020 in combination with the proceeds from the January 2021 public offering will be sufficient to fund its operations through the end of 2023, without giving effect to any potential business development transactions or financing activities.

Financial Results

Fourth Quarter 2020

Net loss was $13.2 million for the fourth quarter of 2020 compared to $18.6 million for the fourth quarter of 2019. Loss from continuing operations was $13.6 million for the quarter ended December 31, 2020 compared to $19.2 million for the prior year period.
Total revenue was $1.6 million for the fourth quarter of 2020 compared to $1.1 million for the fourth quarter of 2019.

Research and development (R&D) expenses were $9.0 million for the quarter ended December 31, 2020 compared to $11.5 million for the prior year period.

The quarter-over-quarter decrease of $2.6 million was primarily the result of expenses related to Aclaris’ legacy JAK inhibitors ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019.
General and administrative (G&A) expenses were $4.9 million for the quarter ended December 31, 2020 compared to $5.8 million for the prior year period.

The quarter-over-quarter decrease of $0.9 million was primarily the result of lower non-cash stock-based compensation expense resulting from headcount reductions. Stock-based compensation expense was $1.6 million compared to $2.6 million in the prior year period.
Full Year 2020

Net loss was $51.0 million for the year ended December 31, 2020 compared to $161.4 million for the year ended December 31, 2019. Loss from continuing operations was $51.2 million for the year ended December 31, 2020 compared to $113.5 million for the prior year period. Income from discontinued operations was $0.1 million for the year ended December 31, 2020 compared to a loss from discontinued operations of $47.8 million for the year ended December 31, 2019.

Total revenue was $6.5 million for the year ended December 31, 2020 compared to $4.2 million for the year ended December 31, 2019.

R&D expenses were $31.7 million for the year ended December 31, 2020 compared to $64.9 million for the prior year period.

The decrease of $33.2 million was primarily the result of expenses related to ATI-501 and ATI-502, including the substantial completion of Aclaris’ various Phase 2 clinical trials, and the substantial completion of two pivotal Phase 3 clinical trials of A-101 45% Topical Solution in 2019.
R&D expenses in 2020 included non-cash stock-based compensation expense of $2.9 million compared to $5.1 million in the prior year period.
G&A expenses were $20.5 million for the year ended December 31, 2020 compared to $27.8 million for the prior year period.

The decrease of $7.3 million was primarily the result of lower personnel and non-cash stock-based compensation resulting from headcount reductions.
G&A expenses in 2020 included non-cash stock-based compensation expense of $7.3 million compared to $10.3 million in the prior year period.
For the year ended December 31, 2019, there was also a $18.5 million non-cash charge for the impairment of goodwill.

On February 25, 2021 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that it will host a conference call and webcast in conjunction with its fourth quarter and year end 2020 update and financial results on Thursday, March 4 at 4:30 p.m. ET (Press release, Acorda Therapeutics, FEB 25, 2021, View Source [SID1234575671]).

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To participate in the Webcast/Conference Call, please note there is a new pre-registration process.

To register for the Webcast, use the link below:
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To register for the Conference Call, use the link below:
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**When registering please type your phone number with no special characters**
Once you have registered, you will receive a confirmation email with Webcast/Conference Call details. For the Webcast you will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. ET on March 4, 2021 until 11:59 p.m. ET on April 3, 2021. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 9854802. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On February 25, 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 fourth quarter and full-year financial results for the period ended December 31, 2020 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, FEB 25, 2021, View Source [SID1234575670]).

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"2020 was a year characterized by strong performance for SpringWorks as we advanced our diversified targeted oncology portfolio, which currently spans 10 development programs across our three core focus areas: late-stage rare oncology, BCMA combinations in multiple myeloma and biomarker-defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our clinical execution has set the stage for multiple important data readouts in 2021, including the interim ReNeu data announced this morning and topline data from our Phase 3 DeFi trial later this year, and our business development efforts have allowed us to pursue additional combination therapy programs, continuing to grow our portfolio of opportunities across a broad range of potentially high-value and high-unmet need oncology patient settings. We look forward to reporting on our 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 AEs reported. SpringWorks reported that the ReNeu trial has reached approximately 70% of its final enrollment target of 100 patients and the Company expects to complete enrollment of the ReNeu 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.
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 a Phase 1b trial sponsored by GSK evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), GSK’s anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, in adult patients with relapsed or refractory multiple myeloma. Initial clinical data from this study are expected in 2021.
Two collaborator-sponsored Phase 1 studies were initiated evaluating nirogacestat in combination with BCMA therapies: nirogacestat + Allogene’s ALLO-715 and nirogacestat + Janssen’s teclistamab. SpringWorks also expects that two additional collaborator-sponsored trials will initiate in the first half of 2021, as previously disclosed: nirogacestat + Pfizer’s elranatamab (PF‐06863135) and nirogacestat + Precision’s PBCAR269A.
Biomarker-Defined Metastatic Solid Tumors

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.
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.
In December 2020, SpringWorks entered into a research collaboration agreement with Dr. Tatu Pantsar and Dr. Antti Poso at the University of Eastern Finland to conduct novel computational biology research to characterize the effector protein dynamics of different RAS mutations. The objective of the collaboration is to understand the biological consequences of KRAS-effector protein interactions and downstream oncogenic signaling with the goal of guiding patient selection strategies and exploring novel targets for future targeted therapy development.
General Corporate

In February 2021, a Notice of Allowance was received from the United States Patent and Trademark Office for Patent Application No. 16/886,622 (the ‘622 application) with composition of matter claims covering several polymorphic forms of nirogacestat, including the polymorphic form that is currently in clinical development, which is also covered by U.S. Patent 10,590,087 (the ‘087 patent, issued in March 2020). Pursuant to an existing worldwide license agreement with Pfizer, SpringWorks has exclusive rights to the patent issuing from the ‘622 application. The patent granting from the ‘622 application, as well as the ‘087 patent, will both expire in August 2039.
In October 2020, SpringWorks completed a follow-on public offering that raised $269.5 million in net proceeds, which included the underwriters’ full exercise of their option to purchase additional shares of common stock.
In October 2020, SpringWorks and Jazz Pharmaceuticals entered into an asset purchase and exclusive license agreement under which Jazz acquired SpringWorks’ fatty acid amide hydrolase (FAAH) inhibitor program. Under the terms of the agreement, Jazz made an upfront payment of $35 million to SpringWorks, with potential future milestone payments of up to $375 million payable to SpringWorks based upon the achievement of certain clinical development, regulatory and commercial milestones. In addition, SpringWorks will receive incremental tiered royalties on future net sales of JZP-150 (formerly PF-04457845) in the mid- to high-single digit percentages.
Fourth Quarter and Full Year 2020 Financial Results

Revenue: Revenue of $35.0 million for the fourth quarter and year ended December 31, 2020 was attributable to the nonrefundable upfront payment from Jazz in October 2020, related to the asset purchase and exclusive license agreement between SpringWorks and Jazz.
Research and Development (R&D) Expenses: R&D expenses were $15.3 million and $51.9 million for the fourth quarter and year-to-date periods, respectively, compared to $12.2 million and $42.5 million for the comparable periods of 2019. The increases in R&D expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of R&D personnel, including non-cash share-based compensation expenses, and increases in external costs related to drug manufacturing and clinical trial costs.
General and Administrative (G&A) Expenses: G&A expenses were $8.5 million and $29.5 million for the fourth quarter and year-to-date periods, respectively, compared to $5.2 million and $16.7 million for the comparable periods of 2019. The increases in G&A expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of G&A personnel supporting the growth of the organization, including non-cash share-based compensation expenses, as well as increases in expenses related to the expansion of business activities.
Net Loss Attributable to Common Stockholders: SpringWorks reported net income of $11.3 million, or $0.24 per share, for the fourth quarter, and a net loss of $45.6 million, or $1.05 loss per share, for the year ended December 31, 2020. This compares to net losses of $16.2 million, or $0.39 loss per share, and $50.6 million, or $3.81 loss per share, for the comparable periods of 2019, respectively.
Cash Position: Cash, cash equivalents and marketable securities were $561.8 million as of December 31, 2020. This includes the net proceeds of $269.5 million from the Company’s follow-on public offering completed in October 2020, and the $35.0 million upfront payment from Jazz Pharmaceuticals in October 2020, related to the asset purchase and exclusive license agreement between SpringWorks and Jazz.
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 February 25, 2021 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a late-stage targeted oncology company,reported financial results for the fourth quarter and full year of 2020, which reflect the company’s progress across its clinical and discovery pipeline and strength as it expands its operational structure and capabilities in preparation for potential commercialization in the United States (Press release, Mirati, FEB 25, 2021, View Source [SID1234575669]).

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"Our commitment to patients with cancer has enabled us to advance our two clinical programs, adagrasib and sitravatinib, as well as our preclinical pipeline. These preclinical programs include MRTX1133, a potentially first-in-class KRAS G12D selective inhibitor and a first-in-class synthetic lethal PRMT5 inhibitor," said Charles M. Baum, M.D., Ph.D., president and chief executive officer, Mirati Therapeutics, Inc. "We have added significant new talent and capabilities across the organization which combined with our financial resources will allow us to accelerate and expand development across our pipeline."

Corporate Updates:

Announced a strategic research and development collaboration with MD Anderson Cancer Center to expand the clinical and preclinical evaluation of Mirati’s two investigational small molecule, potent and selective KRAS inhibitors, adagrasib, a G12C inhibitor, and MRTX1133, a G12D inhibitor, each as monotherapy and in combination with other agents. (View Release)
Entered into McKesson’s MYLUNG consortium ("Molecularly Informed Lung Cancer Treatment in a Community Cancer Network: A Pragmatic Consortium"), a collaborative research endeavor to deepen understanding of molecular testing barriers, improve care for patients with lung cancer, and expand the opportunity for patients to participate in clinical trials. (View Release)
Ended the fourth quarter with approximately $1.4 billion in cash, cash equivalents, and short-term investments.
Adagrasib:

Initiated KRYSTAL-7 (849-007), a Phase 2 clinical trial of adagrasib in combination with pembrolizumab (KEYTRUDA)1 in 1st line non-small cell lung cancer (NSCLC).
Initiated an additional Phase 2 cohort of the KRYSTAL-1 (849-001) in patients with KRAS G12C and the STK11 co-mutation in 1st line NSCLC.
Initiated KRYSTAL-12 (849-012), a Phase 3 clinical trial of monotherapy adagrasib versus docetaxel in 2nd line NSCLC.
Plan to initiate by Q2 2021 KRYSTAL-10 (849-010), a Phase 3 clinical trial of adagrasib in combination with cetuximab (ERBITUX)2 in 2nd line colorectal cancer.
1KEYTRUDA is a registered trademark of Merck
2ERBITUX is a registered trademark of Eli Lilly and Company in the U.S. and Merck KGaA outside the U.S.

Preclinical Updates:

Advanced a first-in-class PRMT5 inhibitor, leveraging a synthetic lethal strategy in MTAP-deleted cancers. This agent specifically inhibits the PRMT5 enzyme in the presence of methylthioadenosine (MTA), a nucleoside cofactor, which is uniquely elevated in cancers exhibiting the most commonly observed gene deletion event (MTAP/CDKN2A) across human cancers. Because PRMT5 is critical to the survival of normal human cells, the ability to specifically target the PRMT5/MTA complex in MTAP-deleted cancer cells while sparing normal human cells is expected to offer a wide therapeutic index compared with conventional first generation PRMT5 or MAT2A inhibitors.
Financial Results for the Fourth Quarter 2020

License and collaboration revenues for the three months ended December 31, 2020, were $1.7 million, and relate to the manufacturing supply services agreement with BeiGene. License and collaboration revenues for the twelve months ended December 31, 2020 were $13.4 million, of which $11.4 million related to a license agreement with ORIC Pharmaceuticals, Inc. and $2.0 million related to the manufacturing supply services agreement with BeiGene. License and collaboration revenues for the three and twelve months ended December 31, 2019 were $0.5 million and $3.3 million, respectively, and relate to the manufacturing supply services agreement with BeiGene.
Research and development expenses for the fourth quarter of 2020 were $82.7 million, compared to $62.9 million for the same period in 2019. Research and development expenses for the year ended December 31, 2020 were $299.3 million, compared to $182.9 million for the same period in 2019. The increase in research and development expenses is due to an increase in expense associated with the development of adagrasib, MRTX1133, and other preclinical and early discovery activities, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The Company recognized research and development-related share-based compensation expenses of $12.2 million during the fourth quarter of 2020, compared to $10.6 million for the same period in 2019, and $48.0 million during the year ended December 31, 2020, compared to $31.0 million for the same period in 2019.
General and administrative expenses for the fourth quarter of 2020 were $25.3 million, compared to $12.2 million for the same period in 2019. General and administrative expenses for the year ended December 31, 2020 were $83.4 million, compared to $42.6 million for the same period in 2019. The increase is due primarily to an increase in share-based compensation expense and an increase in employee-related expenses and professional service expense. The Company recognized general and administrative-related share-based compensation expenses of $9.6 million during the fourth quarter of 2020, compared to $6.1 million for the same period in 2019, and $37.8 million during the year ended December 31, 2020, compared to $24.5 million for the same period in 2019.
Net loss for the fourth quarter of 2020 was $101.1 million, or $2.08 per share basic and diluted, compared to net loss of $72.4 million, or $1.83 per share basic and diluted for the same period in 2019. Net loss for the year ended December 31, 2020 was $357.9 million, or $7.96 per share basic and diluted, compared to net loss of $213.3 million, or $5.69 per share basic and diluted for the same period in 2019.
Cash, cash equivalents, and short-term investments were $1.4 billion at December 31, 2020.