On May 1, 2024 United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, reported its financial results for the quarter ended March 31, 2024 (Press release, United Therapeutics, MAY 1, 2024, View Source [SID1234642530]). Total revenues in the first quarter of 2024 grew 34 percent year-over-year to $677.7 million, compared to $506.9 million in the first quarter of 2023.
"The first quarter of 2024 represents another quarter of record revenue and double-digit year-over-year revenue growth," jointly said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer and Michael Benkowitz, President and Chief Operating Officer. "That growth, coupled with our upcoming clinical reads, puts us in a unique position in the biotech industry: a company with a solid and growing commercial foundation, near-term clinical catalysts, and the potential to provide an unlimited supply of tolerable, transplantable organs."
"Our distinctive commercial and clinical profile, alongside our solid balance sheet and cash flow potential, makes United Therapeutics a compelling investment opportunity," said James Edgemond, Chief Financial Officer and Treasurer. "That’s why we implemented a $1 billion accelerated share repurchase that concretely demonstrates our belief in our near-term and long-term potential, despite potential competition emerging this year."

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On May 1, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) reported its financial results for the first quarter of 2024, along with recent company developments (Press release, TG Therapeutics, MAY 1, 2024, View Source [SID1234642529]).

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Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, stated, "We were extremely pleased with the strong sales of just over $50 million in BRIUMVI U.S. net revenue for the first quarter of 2024, which was ahead of our guidance. We believe this strong momentum will continue to build throughout 2024 and are pleased to update our yearly guidance to $270 to $290 million in BRIUMVI U.S. net revenue in 2024." Mr. Weiss continued, "We are also pleased to be making strides towards our clinical goals for the year which include enhancing the convenient dosing of IV BRIUMVI, developing a subcutaneous form of BRIUMVI, moving BRIUMVI into additional indications beyond MS, and advancing our recent pipeline addition, azer-cel, an allogeneic CAR T, into clinical development."

Recent Highlights & Developments

United States (U.S.) Commercialization of BRIUMVI(ublituximab-xiiy)

BRIUMVI U.S. net product revenue of $50.5 million for the first quarter of 2024, representing >25% quarter over quarter growth
Approximately 4,450 BRIUMVI new patient prescriptions received by the TG Therapeutics hub since launch, including more than 1,250 in the first quarter of 2024, from approximately 800 healthcare providers at approximately 450 centers
Awarded a national contract with the Department of Veterans Affairs (VA) for BRIUMVI to be the preferred agent listed on the VA National Formulary for anti-CD20 antibody indications for patients with relapsing forms of multiple sclerosis
European Commercialization of BRIUMVI

Launched BRIUMVI in the first European country, Germany, with our partner, Neuraxpharm Group (Neuraxpharm)
General Business

Presented updated data from the ENHANCE Phase 3b trial evaluating patients who switch from another CD20 antibody to BRIUMVI at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) annual forum
Obtained three additional patents from the United States Patent and Trademark Office (USPTO) for BRIUMVI, extending patent protection through 2042
Entered into a global license agreement with Precision BioSciences, Inc. (Precision) for the development and commercialization of Precision’s allogeneic CD19 CAR T therapy program, azercabtagene zapreleucel (azer-cel), for the treatment of autoimmune disorders and all other non-oncology indications
2024 Updated Target U.S. BRIUMVI Guidance

Q2 2024 target BRIUMVI U.S. net product revenue of approximately $65 million
Updating BRIUMVI U.S. net product revenue target to approximately $270 million to $290 million for the full year 2024 (prior guidance of $220 to $260 million for full year 2024)
2024 Development Pipeline Anticipated Milestones

Commence clinical development of subcutaneous BRIUMVI
Commence a clinical trial evaluating BRIUMVI in an additional autoimmune disease outside of multiple sclerosis (MS)
Commence a clinical trial evaluating azer-cel in autoimmune disease
Present data from the ENHANCE Phase 3b CD20 switch trial at multiple conferences throughout the year
Financial Results for First Quarter 2024

Product Revenue, Net: Product revenue, net was approximately $50.5 million for the three months ended March 31, 2024, compared to $7.8 million for the three months ended March 31, 2023. Product revenue, net for both the three months ended March 31, 2024 and March 31, 2023, consisted of net product sales of BRIUMVI in the United States.
License, milestone, royalty and other revenue: License, milestone, royalty and other revenue was approximately $13.0 million and less than $0.1 million for the three months ended March 31, 2024 and March 31, 2023, respectively. License, milestone, royalty and other revenue for the three months ended March 31, 2024 is predominantly comprised of a $12.5 milestone payment under the Neuraxpharm Commercialization Agreement for the first key market commercial launch of BRIUMVI in the European Union.
R&D Expenses: Total research and development (R&D) expense was $32.7 million for the three months ended March 31, 2024, compared to $15.9 million for the three months ended March 31, 2023. The increase in R&D expense during the three months ended March 31, 2024, was primarily attributable to license and milestone expense of $8.8 million related to the license agreement with Precision, as well as additional manufacturing and development costs incurred in connection with our ublituximab subcutaneous development work during the period.
SG&A Expenses: Total selling, general and administrative (SG&A) expense was $34.6 million for the three months ended March 31, 2024, compared to $28.1 million during the three months ended March 31, 2023. The increase was primarily due to the scale-up of the BRIUMVI commercial launch, including personnel and consultants, during the three months ended March 31, 2024.
Net Loss: Net loss was $10.7 million for the three months ended March 31, 2024, compared to a net loss of $39.2 million for the three months ended March 31, 2023.
Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $209.8 million as of March 31, 2024. We anticipate that our cash, cash equivalents and investment securities as of March 31, 2024, combined with the projected revenues from BRIUMVI, will be sufficient to fund our planned operations to cash flow positivity based on our current operating plan.
CONFERENCE CALL INFORMATION
The Company will host a conference call today, May 1, 2024, at 8:30 AM ET, to discuss the Company’s financial results from the first quarter, ended March 31, 2024.

To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for a period of 30 days after the call.

On May 1, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported the preliminary data from Phase 2a trial of SLS009, a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML) and successful filing of a provisional patent application around the ASXL1 mutation and SLS009, including all CDK9 inhibitor drugs (Press release, Sellas Life Sciences, MAY 1, 2024, View Source [SID1234642528]). ASXL1 mutations are associated with poor prognosis in all myeloid diseases, owing to the reduced response to the current treatment options.

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SELLAS has observed a high rate of responses in patients with myelodysplasia-related molecular mutations, as defined by the World Health Organization (WHO). Among all myelodysplasia-related mutations, those in the ASXL1 gene accounted for most responders across all dose cohorts. SELLAS has now expanded the ongoing Phase 2 r/r AML study to include two additional cohorts, one with ASXL1 mutated AML patients and one with patients with myelodysplasia-related molecular abnormalities other than ASXL1.

"These early clinical results are very promising and could open up a new avenue in the treatment of AML and potentially beyond," said Joshua Zeidner, MD, Associate Professor of Medicine, Chief of Leukemia Research, Associate Chief of Research in the Division of Hematology, Director of Clinical Cancer Research Commercial Integration at the University of North Carolina Lineberger Comprehensive Cancer Center and the study’s principal investigator. "ASXL1 is a relatively common mutation in AML which leads to poor outcomes with conventional therapies. There are no known targeted therapies that are effective for these AML patients. I am extremely hopeful that SLS009 will make an impact in the management of patients with ASXL1-mutated AML and potentially other myeloid malignancies with similar disease biology."

Study highlights:

As of April 19, 2024 data cutoff, a 57% overall response rate has been achieved thus far, in the selected optimal dose regimen of 30 mg BIW, far surpassing the targeted 20% rate.
4/4 (100%) r/r AML patients with ASXL1 truncating mutations at the selected dose level achieved an overall response (CR/CRi/MLFS) and are alive.
5/8 (63%) of r/r AML patients, across all dose levels, with ASXL1 truncating mutations treated with SLS009 achieved an overall response.
A review of the mutational status of the patient in the Phase 1 trial with SLS009 monotherapy, who achieved a CR lasting 8+ months, revealed that the patient also harbored an ASXL1 mutation.
The ASXL1 mutation is found in both hematological malignancies as well as solid tumors.
All patients in the study are diagnosed with AML refractory to or relapsed after venetoclax-containing regimens. Enrollment and treatment will be focused on the participants in the expansion cohort receiving 30 mg BIW dose and diagnosed with the ASXL1 mutation.
"The remarkable responses achieved in patients with the ASXL1 mutation underscores the transformative potential of SLS009 in addressing the unmet medical needs of AML but also targeting colorectal cancer, and other tumor types with this alteration," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "This is evidenced by strong enthusiasm from the participating investigators reflected in robust enrollment from the clinical sites. These compelling results from the Phase 2a study further reinforce our belief that SLS009 represents a potential breakthrough for relapsed and/or refractory AML patients, and could pave the way for a potential accelerated approval in this defined patient population."

SELLAS intends to initiate discussions with the U.S. Food and Drug Administration (FDA) about the potential for an accelerated approval pathway with SLS009 in the ASXL1 molecularly defined r/r AML population as well as in patients harboring this mutation in other indications.

The Phase 2a clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with aza/ven at two dose levels, 45 and 60 mg. In the 60 mg dose cohort patients were randomized into either a 60 mg dose once per week or a 30 mg dose two times per week. The target response rate at the optimal dose level is 20% with a target median survival over 3 months. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit clinicaltrial.gov identifier NCT04588922.

About ASXL1
ASXL1 mutations are associated with poor prognosis in all myeloid diseases, owing to the reduced response to the current treatment options. In AML, ASXL1 mutations were an unfavorable prognostic factor as regards survival, with a significantly lower complete response rate. In MDS, ASXL1 mutations are independently associated with worse overall survival, as well as AML transformation. The tables below demonstrate ASXL mutation frequency across hematologic malignancies and solid cancers.

ASXL1 mutations in Hematologic Malignancies with ASXL1m Frequency ≥5%

Condition ASXL1m Frequency US Condition Incidence
AML (Acute Myeloid Leukemia) 20% 20,800
MDS (Myelodysplastic Syndrome) 20% 10,000
MPN (Myeloproliferative Neoplasms) 10% 20,000
CMML (Chronic Myelomonocytic Leukemia) 43% 1,100
Total 51,900

ASXL1 in Solid Cancers with ASXL1m Frequency ≥5%

Condition ASXL1m Frequency US Condition Incidence
CRC MSI-high (Colorectal Cancer with High Microsatellite Instability) 55% 22,500
Cervical Ca. (invasive) 5% 13,800
Liver Ca. 10% 42,400
Total 78,700

On May 1, 2024 Pfizer reported First-Quarter 2024 Results (Press release, Seagen, MAY 1, 2024, View Source [SID1234642527]).

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On May 1, 2024 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based computational platform is transforming the way therapeutics and materials are discovered, reported financial results for the first quarter of 2024 (Press release, Schrodinger, MAY 1, 2024, View Source [SID1234642526]).

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"Over the past year, we have witnessed unprecedented interest in computational drug discovery, and we are excited by the growing appreciation for using computation to drive pharmaceutical innovation. We are excited about the opportunities we have to increase customer adoption of our platform this year and drive revenue growth going forward," said Ramy Farid, Ph.D., chief executive officer of Schrödinger. "We are also pleased with the continued progress of our proprietary pipeline. Today we reported FDA clearance of our IND application for SGR-3515, our novel Wee1/Myt1 inhibitor, and expect to initiate a Phase 1 clinical study in patients with solid tumors this year. Our Phase 1 clinical studies of SGR-1505 and SGR-2921 remain on track, and we look forward to reporting data from these two trials in late 2024 or 2025."

First Quarter 2024 Financial Results
•Total revenue for the first quarter was $36.6 million, compared to $64.8 million in the first quarter of 2023. The decrease was primarily due to a large collaboration milestone payment that favorably impacted revenue in the first quarter of 2023.
•Software revenue for the first quarter increased 3.7% to $33.4 million, compared to $32.2 million in the first quarter of 2023. The increase reflects a growing contribution from hosted licenses, partially offset by multi-year agreements signed in the first quarter of 2023.
•Drug discovery revenue was $3.2 million for the first quarter, compared to $32.6 million in the first quarter of 2023. The first quarter of 2023 included a large collaboration milestone payment associated with the progression of a collaboration project.
•Software gross margin decreased to 76% for the first quarter, compared to 78% in the first quarter of 2023, largely due to higher technology expenses.
•Operating expenses were $86.3 million for the first quarter, compared to $76.2 million for the first quarter of 2023. The increase was primarily due to higher R&D expenses.
•Other income, which includes changes in fair value of equity investments and interest income/expense, was $13.2 million for the first quarter, compared to other income of $186.0 million for the first quarter of 2023. Other income for the first quarter of 2023 included a $147.3 million gain relating to the cash distributions from Nimbus in connection with the sale of Nimbus’s TYK2 inhibitor to Takeda.
•Net loss for the first quarter was $54.7 million, compared to net income of $129.1 million in the first quarter of 2023. For the three months ended March 31, 2024, Schrödinger reported a non-GAAP net loss of $62.4 million compared to a non-GAAP net loss of $27.6 million for the three months ended March 31, 2023. A reconciliation of non-GAAP net loss to GAAP net (loss) income can be found in "Non-GAAP Information" and financial tables below.
•At March 31, 2024, Schrödinger had cash, cash equivalents, restricted cash and marketable securities of approximately $435.7 million, compared to approximately $468.8 million at December 31, 2023.

Three Months Ended
March 31,
2024 2023 % Change
(in millions)
Total revenue $ 36.6 $ 64.8 (44)%
Software revenue 33.4 32.2 3.7%
Drug discovery revenue 3.2 32.6 (90)%
Software gross margin 76 % 78 %
Operating expenses $ 86.3 $ 76.2 13%
Other income $ 13.2 $ 186.0 —
Net (loss) income $ (54.7) $ 129.1 —

2024 Financial Outlook
As of May 1, 2024, Schrödinger maintained its financial guidance for the fiscal year ending December 31, 2024:
•Software revenue growth is expected to range from 6% to 13%.
•Drug discovery revenue is expected to range from $30 million to $35 million.
•Software gross margin is expected to be similar to software gross margin for the full year 2023.
•Operating expense growth in 2024 is expected to range from 8% to 12%.
•Cash used for operating activities in 2024 is expected to be above cash used for operating activities in 2023.

For the second quarter of 2024, software revenue is expected to range from $31 million to $33 million.
Key Highlights
Platform
•In March, Schrödinger launched LiveDesign Biologics, an enterprise cloud-based, informatics solution that allows drug discovery teams designing biologics to centralize access to computational modeling tools and data in a single interface. LiveDesign Biologics builds on Schrödinger’s existing LiveDesign offering, which is primarily focused on integrating tools and data for the design of small molecules.

•In March, Schrödinger presented the discovery of SGR-1505, its MALT1 inhibitor, during the First Time Disclosure Session at the American Chemical Society Spring 2024 Meeting. The presentation provided an overview of how Schrödinger leveraged its computational platform at scale to discover SGR-1505. Schrödinger’s platform enabled vast exploration of chemical space, evaluating 8.2 billion compounds, allowing Schrödinger to synthesize only 78 of the most promising molecules in the lead series, and identify SGR-1505 in approximately 10 months.

•Schrödinger is accelerating the validation of computational predictive models of off-target drug activity. There is an emerging requirement for such models to predict drug toxicity risks before animal or human studies are initiated. Schrödinger is exploring the deployment of its core technologies and capabilities, at scale, to provide computational solutions to meet these requirements. The company’s recent advances characterizing the structure of key proteins such as hERG (recently published in Cell) and cytochrome P450 enzymes are examples of these efforts.

Proprietary Pipeline

•Today, Schrödinger also announced that the U.S. Food and Drug Administration (FDA) cleared the investigational new drug (IND) application for SGR-3515, its Wee1/Myt1 inhibitor. Schrödinger expects to initiate a Phase 1 dose-escalation trial of SGR-3515 in patients with solid tumors in the third quarter of 2024.

•The company is advancing the Phase 1 dose-escalation study of SGR-1505, its MALT1 inhibitor, in patients with relapsed/refractory B-cell malignancies, and enrollment is ongoing in the U.S. and EU. The company expects to report initial data from this study in late 2024 or 2025.

•Schrödinger also continues to advance its Phase 1 study of SGR-2921, its CDC7 inhibitor, in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome. Patient enrollment is ongoing in the U.S., and the company expects to report initial data from the study in late 2024 or 2025.
•Today Schrödinger announced that the rights to the SOS1 inhibitor discovered and developed as part of the collaboration with Bristol Myers Squibb have reverted to Schrödinger based on portfolio prioritization decisions at BMS. In 2023, Schrödinger completed and delivered a development candidate, and BMS had been conducting IND-enabling studies.

•Schrödinger is progressing its discovery pipeline, including programs targeting EGFRC797S, PRMT5-MTA and NLRP3. The company continues to anticipate submitting at least one IND in 2025.

Webcast and Conference Call Information
Schrödinger will host a conference call to discuss its first quarter 2024 financial results on Wednesday, May 1, 2024, at 4:30 p.m. ET. The live webcast can be accessed under "News & Events" in the investors section of Schrödinger’s website, View Source To participate in the live call, please register for the call here. It is recommended that participants register at least 15 minutes in advance of the call. Once registered, participants will receive the dial-in information. The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.