On November 3, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and third quarter 2022 financial results (Press release, Sangamo Therapeutics, NOV 3, 2022, View Source [SID1234622961]).

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"This has been a year marked by progress across our pipeline. In the third quarter, we continued to advance our clinical trials and preclinical activities while maintaining fiscal discipline and operational excellence," said Sandy Macrae, Chief Executive Officer of Sangamo. "We were proud to present promising updated preliminary data from our wholly owned Fabry study, to resume our partnered Hemophilia A pivotal trial, and to continue dosing in our renal transplant rejection and sickle cell studies. Our pipeline progress is expected to yield additional data in Q4 and into 2023. As we look to next year and beyond, I am confident in Sangamo’s ability to carry out our mission of developing transformational therapies for patients in need."

Recent Business Highlights

Fabry disease – Reported data updates from the Phase 1/2 STAAR study’s dose escalation phase; Dose expansion phase underway and dosing commenced; Phase 3 planning progresses.

Presented updated preliminary data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry Disease at three separate conferences, most notably at the 29th Congress of the European Society of Gene & Cell Therapy (ESGCT), presenting updated data as of July 21, 2022.
Preliminary data showed all nine patients from the dose escalation phase exhibited sustained elevated α-Gal A activity, ranging from nearly 2-fold to 30-fold of mean normal, for up to 23 months post dosing, as of the last date of measurement.
Four patients were withdrawn from enzyme replacement therapy (ERT) and maintained significantly elevated levels of α-Gal A activity up to 28 weeks post withdrawal. Since the cutoff date, the fifth and final patient in the dose escalation phase who started the study on ERT has been withdrawn from ERT. All patients withdrawn have remained off ERT.
The Phase 1/2 STAAR study has transitioned into the expansion phase, with the first five expansion patients dosed at the 5e13 vg/kg dose level, including the first two female patients.
We expect to present additional clinical updates from the STAAR study, including the first data from the expansion cohort, in the first half of 2023.
We continue to actively prepare for a potential pivotal Phase 3 trial.
Sickle cell disease – Dosed sixth patient, the second with a product candidate manufactured using improved methods; Phase 3 planning progresses.

We dosed the sixth patient in the Phase 1/2 PRECIZN-1 study of BIVV003, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease. This is the second patient in the study to receive a product candidate manufactured using improved methods that have been shown in internal experiments to increase the number of long-term progenitor cells in the final product.
Received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BIVV003.
We expect to present updated data from the Phase 1/2 PRECIZN-1 study via a poster presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition on December 10-13, 2022 in New Orleans, Louisiana.
Phase 3 study design, enabling activities and manufacturing readiness are in progress.
Renal Transplant Rejection – Dosed the second patient in the Phase 1/2 STEADFAST study; progressed clinical activities in preparation for patient three.

Dosed the second patient in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
The product candidate continues to be generally well tolerated in both patients.
Progressed clinical activities in preparation for the third patient.
We plan to provide guidance on timing for dosing for the third patient once the kidney transplant has been scheduled.
Hemophilia A – Announced, with Pfizer, the resumption of recruitment in the Phase 3 AFFINE trial; dosing is expected to resume shortly; pivotal data read-out expected in the first half of 2024.

Pfizer and Sangamo announced that recruitment has re-opened in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A.
Trial sites resumed enrollment in September, and dosing is expected to resume shortly.
A pivotal readout is expected in the first half of 2024.
We expect to present updated data from the Phase 1/2 ALTA study via a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December.
Third Quarter 2022 Financial Results

Consolidated net loss for the third quarter ended September 30, 2022 was $53.2 million, or $0.34 per share, compared to a net loss of $47.7 million, or $0.33 per share, for the same period in 2021.

Revenues

Revenues for the third quarter ended September 30, 2022 were $26.5 million, compared to $28.6 million for the same period in 2021.

The decrease of $2.1 million in revenues was primarily attributable to a decrease of $1.9 million and $1.6 million related to our collaboration agreements with Novartis and Biogen respectively, and a decrease of $1.1 million due to the termination of our collaboration agreement with Sanofi. These decreases were partially offset by a $1.9 million adjustment to revenue during 2021 related to the collaboration agreement with Sanofi and an increase of $0.5 million in revenue related to our collaboration agreement with Kite.

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2022 were $81.3 million, compared to $77.0 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2022 were $73.5 million, compared to $69.1 million for the same period in 2021.

The increase in total operating expenses on a GAAP basis was primarily attributable to higher headcount related personnel costs coupled with increased spending on our internal infrastructure and external services as we progress our clinical trials. These increases were partially offset by reimbursement of certain research and development expenses by Sanofi under the termination agreement.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of September 30, 2022, were $350.3 million, compared to $464.7 million as of December 31, 2021. We have raised approximately $75.1 million in net proceeds under our at-the-market offering program from January 1, 2022 through October 31, 2022.

Financial Guidance for 2022 Narrowed (initial guidance provided on February 24, 2022)

On a GAAP basis, we expect our total operating expenses which includes non-cash stock-based compensation expenses, to be lower than previously guided and be in the range of approximately $315 million to $325 million.

We expect our non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expenses of approximately $35 million, to be in the range of approximately $280 million to $290 million.

Upcoming Events

Sangamo plans to participate in the following events in the fourth quarter:

Scientific / Medical Conferences

64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 10-13, New Orleans, Louisiana
Investor Conferences

31st Annual Credit Suisse Healthcare Conference, November 8, 2022
Barclays Gene Editing & Gene Therapy Summit, November 14, 2022
Stifel Healthcare Conference, November 15, 2022
Jefferies London Healthcare Conference, November 16, 2022
EvercoreISI HealthCONxConference, November 29 – December 1, 2022
Access links for available webcasts for these investor conferences will be available on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Available materials will be found on the Sangamo Therapeutics website after the event.

Conference Call to Discuss Third Quarter 2022 Results

The Sangamo management team will discuss these results on a conference call today, Thursday November 3, 2022, at 4:30 p.m. Eastern Time.

Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.

The link to access the live webcast can also be found on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

A replay will be available following the conference call, accessible under Events and Presentations.

On November 3, 2022 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based computational platform is transforming the way therapeutics and materials are discovered, reported financial results for the third quarter of 2022 (Press release, Schrodinger, NOV 3, 2022, View Source [SID1234622960]).

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"We delivered a strong quarter, with third quarter software revenue in line with our expectations and strong drug discovery revenue, reflecting progress across our portfolio of collaborative and proprietary programs," stated Ramy Farid, Ph.D., chief executive officer at Schrödinger. "As we progress through the remainder of the year, we are focused on continuing to drive software adoption and advancing our pipeline. Today we announced that we’ve initiated our Phase 1 study for our MALT1 program and we are looking forward to presenting new preclinical data from our CDC7 program at the upcoming ASH (Free ASH Whitepaper) meeting. We are very positive about the long-term potential of our underlying business – this quarter’s results highlight the benefits of our balanced model encompassing software licensing, collaborations and proprietary programs."

At September 30, 2022, Schrödinger had cash, cash equivalents, restricted cash and marketable securities of approximately $479 million, compared to approximately $513 million at June 30, 2022.

Recent Company Highlights

In October, Schrödinger hosted Platform Day during which the company provided a detailed review of its technology platform and demonstrated the impact of this platform across a growing portfolio that includes 9 collaborative programs in the clinic, 12 active collaborative projects and 18 proprietary programs. During the event, Schrödinger highlighted areas for future innovation and discussed the opportunities for value creation. The company also described six drug discovery case studies, including collaborative programs with Nimbus Therapeutics and Morphic Therapeutic as well as case studies from Schrödinger’s proprietary MALT1, CDC7 and Wee1 programs. Additionally, the company reviewed near- and longer-term opportunities for continued innovation of its physics-based computational platform, including increasing the number and type of discovery targets the platform can advance through hit identification, improving the effectiveness and efficiency with which the platform can advance targets through lead optimization, and expanding the applicability of the platform to new high-value areas.
In October, Schrödinger announced that it entered into a collaboration with Eli Lilly and Company. Under the terms of the agreement, Schrödinger is responsible for the discovery and optimization of small molecule compounds addressing an undisclosed target. Lilly is responsible for preclinical development, clinical development and commercialization. Schrödinger received an upfront payment and is eligible to receive up to $425 million in discovery, development and commercial milestone payments. Schrödinger is also eligible to receive low single- to low double-digit royalties on net sales of any products emerging from the collaboration in all markets.
Schrödinger reported that its Phase 1 clinical trial of its MALT1 inhibitor, SGR-1505, is open to patient enrollment. This dose-escalation study is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and early signals of clinical activity of SGR-1505 as a monotherapy in patients with relapsed or refractory B-cell malignancies. Once the recommended dose is determined, an expansion cohort is planned to evaluate SGR-1505 in combination with other anti-cancer agents, such as BTK and BCL-2 inhibitors, in patients with specific B-cell malignancies.
Earlier today, Schrödinger announced that new preclinical data for SGR-2921, a CDC7 inhibitor, will be presented during a poster session at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting in December 2022. The data demonstrate the strong anti-tumor activity of SGR-2921 in preclinical models of acute myeloid leukemia (AML) both alone and in combination with standard of care therapies for the treatment of AML. SGR-2921 is advancing through IND-enabling studies to support a planned IND submission in the first half of 2023.
Schrödinger has identified multiple novel Wee1 inhibitors, and preclinical studies are ongoing. Schrödinger’s goal is to select a differentiated Wee1 inhibitor that demonstrates strong anti-tumor activity with limited off-target effects. The company now expects to select a Wee1 development candidate in the first half of 2023 and submit an IND in the first half of 2024. Wee1 is emerging as a potentially important therapeutic target for a range of solid tumors, including ovarian and uterine cancer.
In October, Schrödinger’s collaborator Morphic Therapeutic presented Phase 1 data from MORF-057 at the United European Gastroenterology Week 2022. These data reinforce previously reported safety and pharmacodynamic data from the MORF-057-101 study. MORF-057 is being developed as an oral α4β7 inhibitor candidate for the treatment of inflammatory bowel disease, with an initial focus in ulcerative colitis.
In August, Schrödinger’s collaborator Nimbus Therapeutics presented data at the American Chemical Society Fall 2022 meeting describing how structural biology and computational chemistry insights were leveraged to produce NDI-034858, its novel TYK2 inhibitor. Phase 2 clinical studies are ongoing to evaluate NDI-034858 in moderate-to-severe plaque psoriasis and psoriatic arthritis.
Schrödinger was recently ranked Number 21 on Newsweek’s List of "America’s 100 Most Loved Workplaces."
2022 Financial Outlook
As of November 3, 2022, Schrödinger outlined the following expectations for the fiscal year ending December 31, 2022:

Total revenue is now expected to range from $167 million to $175 million, compared to the prior expectation of $161 million to $181 million. The updated range represents growth of 21 percent to 27 percent over 2021.
Total software revenue is now expected to range from $122 million to $127 million, compared to the prior expectation of $126 million to $136 million. The updated range represents eight percent to 12 percent growth over 2021. The full year 2022 software revenue expectation implies fourth quarter 2022 software revenue to range from $34 million to $39 million.
Total drug discovery revenue is now expected to range from $45 million to $48 million, compared to the prior expectation of $35 million to $45 million. This higher range represents 82 percent to 94 percent growth over 2021.
Operating expense growth is now expected to be approximately 40 percent for 2022 compared to the prior expectation of slightly lower than 42 percent.
Software gross margin percentage is still expected to be in the mid-70s.
"Despite the challenging macroeconomic and industry environment, our tightened total revenue guidance for 2022 remains within our original range," stated Geoff Porges, MBBS, chief financial officer at Schrödinger. "We consider our balanced business model to be one of our greatest strengths, and this quarter’s results reflect the growing contribution of our drug discovery portfolio to our operating results and overall value."

Webcast and Conference Call Information
Schrödinger will host a conference call to discuss its third quarter 2022 financial results on Thursday, November 3, 2022, 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.

On November 3, 2022 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported financial results for the third quarter of 2022 (Press release, Oncternal Therapeutics, NOV 3, 2022, View Source [SID1234622959]).

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"This past quarter was a pivotal one for Oncternal’s leading ROR1 programs, highlighted by initiation of the Company’s first global registrational clinical study for zilovertamab in MCL and the FDA clearance of the IND for ONCT-808, our ROR1 targeting CAR T product candidate, for the treatment of patients with advanced aggressive B-cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "These exciting developments are backed by a strong clinical data foundation, as highlighted by acceptance of our zilovertamab Phase 1/2 data update in MCL and CLL for an oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December. We are now looking forward to ramping up site enrollment for our Phase 3 clinical study globally, to the initiation of our first CAR T Phase 1/2 study from which we expect an initial clinical data readout next year, and to the further advancement towards the clinic of ONCT-534, our DAARI product candidate that may address key resistance mechanisms in metastatic prostate cancer. We continue to exercise prudent cash management and expect our existing cash and cash equivalents will last into the first half of 2024."

Recent Highlights

Initiated the Phase 3 global registrational study of zilovertamab, Study ZILO-301 (NCT05431179), for the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL)
Received a ‘Study May Proceed’ letter from the U.S. Food and Drug Administration (FDA), 30 days after submitting our Investigational New Drug (IND) application for our Phase 1/2 study of ONCT-808, an autologous chimeric antigen receptor (CAR) T therapy targeting ROR1, in patients with aggressive B-cell lymphoma, including those who have failed previous CD19 CAR T treatment
Expected Upcoming Milestones

Zilovertamab, our ROR1 antibody program
Opening of sites outside of the U.S. for global clinical registrational Phase 3 Study ZILO-301, in the first quarter of 2023
Interim clinical data update for patients with MCL and CLL treated with zilovertamab plus ibrutinib in ongoing Phase 1/2 Study CIRM-0001, as oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2022
ONCT-808, lead candidate in our autologous ROR1-targeted CAR T cell therapy program
Initiation of Phase 1/2 Study in patients with aggressive B-cell lymphoma in the first quarter of 2023
Initial clinical data update in 2023
ONCT-534, lead candidate in our DAARI program
U.S. FDA pre-IND feedback in December 2022
Third Quarter 2022 Financial Results
Our grant revenue was $0.4 million for the third quarter ended September 30, 2022. Our grant revenue is derived from two research and development grant awards from the National Institutes of Health (NIH).

Our total operating expenses for the third quarter ended September 30, 2022 were $11.7 million, including $2.0 million in non-cash stock-based compensation expense. Research and development expenses for the quarter totaled $8.4 million, and general and administrative expenses for the quarter totaled $3.3 million. Net loss for the third quarter was $11.1 million, or a loss of $0.21 per share, basic and diluted.

As of September 30, 2022, we had approximately 55.5 million shares of common stock outstanding, $70.6 million in cash and cash equivalents and no debt. Based on our current operating plan, we believe these funds will be sufficient to fund our operations into the first half of 2024. Our cash guidance is subject to a number of assumptions, including those related to the pace of our research and clinical development programs, among other aspects of our business and the geopolitical environment.

On November 3, 2022 MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing and commercializing innovative antibody-based therapeutics for the treatment of cancer, reported financial results for the quarter ended September 30, 2022 (Press release, MacroGenics, NOV 3, 2022, View Source [SID1234622958]).

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"Over the past few months, we have strengthened our financial position by generating non-dilutive capital through our partnering efforts. We have achieved this through the receipt of $30 million in milestone payments from Incyte during the quarter and the subsequent receipt of a $60 million upfront payment from Gilead for our recently announced MGD024 collaboration. In addition, over the next few weeks, we await the U.S. Food and Drug Administration (FDA) decision regarding Provention Bio’s teplizumab biologics license application (BLA), which would generate an additional $60 million milestone payment obligation to MacroGenics, if approved," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Moreover, we are focused on advancing our pipeline of clinical and preclinical product candidates. We look forward to initiating the MGC018 TAMARACK study later this year, continuing to enroll the MGC018 combination study with lorigerlimab and the MGD024 dose-escalation study, as well as reporting data from the lorigerlimab monotherapy expansion cohorts in early 2023."

Updates on Proprietary Investigational Programs

Recent progress and anticipated events related to MacroGenics’ investigational product candidates in clinical development are highlighted below.

MGC018, now also known as vobramitamab duocarmazine, is an antibody-drug conjugate (ADC) that targets B7-H3, an antigen with broad expression across multiple solid tumor types and a member of the B7 family of molecules involved in immune regulation.
MacroGenics continues to expect to start the Phase 2 portion of the TAMARACK study of vobramitamab duocarmazine in patients with metastatic castration-resistant prostate cancer (mCRPC) by year-end 2022. The Company believes that this should enable interim data from the Phase 2 portion of the study in 2024.
Patient recruitment continues in a Phase 1/2 dose escalation study of vobramitamab duocarmazine in combination with lorigerlimab in patients with various advanced solid tumors.
Lorigerlimab is a bispecific, tetravalent PD-1 × CTLA-4 DART molecule. MacroGenics enrolled a Phase 1/2 dose expansion study with lorigerlimab as monotherapy in cohorts of patients with microsatellite stable colorectal cancer, mCRPC, melanoma and checkpoint-naïve non-small cell lung cancer (NSCLC) and expects to provide a data update from this study in the first quarter of 2023.
MGD024 is a next-generation, humanized CD123 × CD3 DART molecule designed to minimize cytokine-release syndrome, while maintaining anti-tumor cytolytic activity, and permitting intermittent dosing through a longer half-life. MacroGenics continues to enroll patients in a Phase 1 dose-escalation study of MGD024 in patients with CD123-positive neoplasms, including acute myeloid leukemia and myelodysplastic syndromes.
Other Program Updates:

Teplizumab is an investigational, anti-CD3 mAb acquired from MacroGenics by Provention Bio, Inc. under an asset purchase agreement in 2018. Provention Bio is developing teplizumab for the prevention and treatment of type 1 diabetes (T1D). The Prescription Drug User Fee Act (PDUFA) target date for action on the BLA for teplizumab for the prevention of T1D is November 17, 2022. MacroGenics is eligible to receive royalties on net sales of teplizumab, if approved, in addition to milestone payments, including $60 million upon approval of a BLA in the United States.
Retifanlimab is an investigational anti-PD-1 mAb that has been exclusively licensed to Incyte Corporation. MacroGenics is eligible to receive royalties on net sales of retifanlimab, if approved, in addition to milestone payments. In July 2022, MacroGenics received $30 million in milestone payments from Incyte as part of its collaboration agreement. Retifanlimab is currently being studied as monotherapy or in combination with other agents across multiple studies.
Gilead Collaboration

On October 14, 2022, MacroGenics and Gilead Sciences, Inc. entered into an exclusive option and collaboration agreement to develop MGD024 and up to two additional bispecific research programs. The agreement grants Gilead the option to license MGD024. As part of the agreement, Gilead paid MacroGenics an upfront payment of $60 million and MacroGenics will be eligible to receive up to $1.7 billion in target nomination, option fees, and development, regulatory and commercial milestones. MacroGenics will also be eligible to receive tiered, low double-digit royalties on worldwide net sales of MGD024 and a flat royalty on worldwide net sales of products resulting from the two additional research programs.

MacroGenics will be responsible for the ongoing Phase 1 study of MGD024 during which Gilead may elect to exercise its option to license the program at predefined decision points. The Phase 1 study includes a dose escalation segment and an expansion segment that is intended to evaluate MGD024 as monotherapy and in combination with other therapies across multiple indications.

Third Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2022, were $123.6 million, compared to $243.6 million as of December 31, 2021. The September 30, 2022 balance did not include $60 million subsequently received from Gilead in October 2022.
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $41.7 million for the quarter ended September 30, 2022, compared to total revenue of $15.7 million for the quarter ended September 30, 2021. Revenue for the quarter ended September 30, 2022 included MARGENZA net sales of $4.4 million, compared to $3.6 million for the quarter ended September 30, 2021.
R&D Expenses: Research and development expenses were $48.2 million for the quarter ended September 30, 2022, compared to $49.8 million for the quarter ended September 30, 2021. The decrease was primarily related to decreased retifanlimab manufacturing costs for Incyte, and decreased costs related to discontinued studies. These decreases were partially offset by increased development, manufacturing and clinical trial costs related to vobramitamab duocarmazine, increased expenses related to discovery projects and preclinical molecules, and increased clinical expenses related to lorigerlimab and MGD024.
SG&A Expenses: Selling, general and administrative expenses were $15.4 million for the quarter ended September 30, 2022, compared to $17.2 million for the quarter ended September 30, 2021. The decrease was primarily related to decreased selling costs for MARGENZA as well as decreased consulting expenses.
Net Loss: Net loss was $24.8 million for the quarter ended September 30, 2022, compared to net loss of $52.9 million for the quarter ended September 30, 2021.
Shares Outstanding: Shares of common stock outstanding as of September 30, 2022 were 61,462,189.
Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $123.6 million as of September 30, 2022, $60 million subsequently received from Gilead, projected and anticipated future payments from partners and product revenues should extend its cash runway into mid-2024. This cash runway guidance reflects anticipated expenditures related to the planned Phase 2 portion of the TAMARACK study as well as MacroGenics’ other ongoing studies.
Conference Call Information

To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call.

The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at View Source A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on MacroGenics’ website for 30 days following the call.

On November 3, 2022 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the third quarter ended September 30, 2022, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, NOV 3, 2022, View Source [SID1234622957]).

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"We are excited to have several poster presentations at ASH (Free ASH Whitepaper) that showcase our hematology-oncology portfolio. In particular, updated data from the Phase 2 registrational study of olutasidenib in patients with mIDH1 relapsed or refractory acute myeloid leukemia demonstrate durable remissions, which we believe truly differentiates olutasidenib as a potential market leading therapy," said Raul Rodriguez, Rigel’s president and CEO. "In addition, during the third quarter of 2022, we made meaningful progress to position the company for the potential launch of olutasidenib and to drive growth in TAVALISSE ITP sales."

Business Update

In the third quarter of 2022, TAVALISSE net product sales were $19.2 million, an increase of 20% compared to the same period of 2021.
In August, Rigel announced an exclusive license agreement with Forma Therapeutics, Inc. (Forma) to develop, manufacture and commercialize olutasidenib, an investigational, oral, small molecule inhibitor of mutant isocitrate dehydrogenase-1 (mIDH1) for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML) and other malignancies. Forma’s New Drug Application (NDA) for olutasidenib is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of February 15, 2023.
Today, Rigel announced five poster presentations at the 64th ASH (Free ASH Whitepaper) Annual Meeting, including updated data from the interim analysis of 147 efficacy evaluable patients with mIDH1 R/R AML who received olutasidenib monotherapy 150 mg twice daily. Results from the interim analysis of patients with mIDH1 R/R AML demonstrated a 35% CR+CRh* rate with a median duration of 25.9 months. The abstract concluded that the observed activity is clinically meaningful and represents a potential therapeutic advance in the treatment of this patient population.
This week, Rigel announced top-line results from the FOCUS Phase 3 clinical trial of fostamatinib in high-risk hospitalized COVID-19 patients. While the trial approached but did not meet statistical significance (p=0.0603) in the primary efficacy endpoint of the number of days on oxygen through Day 29, all prespecified secondary endpoints in the study numerically favored fostamatinib over placebo, including mortality, time to sustained recovery, change in ordinal scale assessment, and number of days in the ICU. The Company is evaluating the opportunity and next steps in collaboration with its partner, the U.S. Department of Defense.
In October, Rigel announced that it does not expect to file a supplemental NDA for fostamatinib for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA) based on guidance from the FDA’s review of the Company’s re-analysis of data from the FORWARD Phase 3 trial. Rigel will continue to explore its options for the wAIHA program in relation to its complete portfolio of development opportunities.
In October, Rigel also announced a 16% reduction in its workforce, resulting in the elimination of 30 positions primarily in development and administration.
*CR+CRh: Complete remission (CR) plus a complete remission with partial hematological recovery (CRh)

Financial Update
For the third quarter of 2022, Rigel reported a net loss of $19.0 million, or $0.11 per basic and diluted share, compared to a net loss of $21.0 million, or $0.12 per basic and diluted share, for the same period of 2021.

For the third quarter of 2022, total revenues were $22.4 million, consisting of $19.2 million in TAVALISSE net product sales, $0.7 million in contract revenues from collaborations and $2.5 million in government contract revenue. TAVALISSE net product sales of $19.2 million increased by 20%, compared to $16.0 million in the third quarter of 2021. Contract revenues from collaborations during the third quarter of 2022 consisted primarily of revenue from Grifols related to the delivery of fostamatinib supply, performance of certain research and development services pursuant to the collaboration agreement and royalty revenue. Government contract revenue for the third quarter of 2022 was related to the income recognized pursuant to the agreement with the U.S. Department of Defense (DOD) to support Rigel’s ongoing Phase 3 clinical trial of fostamatinib in hospitalized patients with COVID-19.

For the third quarter of 2022, total costs and expenses were $40.8 million, compared to $41.3 million for the same period of 2021. The decrease in costs and expenses was primarily due to a decrease in research and development costs related to the Phase 3 clinical trial for wAIHA, the Phase 3 clinical trial in high-risk hospitalized patients with COVID-19 and the IRAK 1/4 inhibitor program. These decreases were partially offset by increased personnel related costs and commercial activities.

For the nine months ended September 30, 2022, Rigel reported a net loss of $60.0 million, or $0.35 per basic and diluted share, compared to a net income of $4.7 million, or $0.03 per basic and diluted share, for the same period of 2021.

For the nine months ended September 30, 2022, total revenues were $69.0 million, consisting of $53.9 million in TAVALISSE net product sales, $12.5 million in contract revenues from collaborations and $2.5 million in government contract revenue. TAVALISSE net product sales of $53.9 million increased by 19% compared to $45.4 million in the same period of 2021. Contract revenues from collaborations for the nine months ended September 30, 2022, consisted of $7.6 million in revenue from Kissei primarily related to a milestone payment and delivery of fostamatinib supply, $2.0 million in revenue related to the license agreement with Knight, $2.4 million in revenue from Grifols related to the delivery of fostamatinib supply, performance of certain research and development services pursuant to the collaboration agreement and royalty revenue, and $0.5 million in revenue related to the license agreement with Eli Lilly. Government contract revenue for the nine months ended September 30, 2022, was related to the income recognized pursuant to the agreement with the DOD as mentioned above.

For the nine months ended September 30, 2022, total costs and expenses were $126.6 million, compared to $119.9 million for the same period of 2021. The increase in costs and expenses was primarily due to increased personnel costs from the sales force expansion, increased commercial-related activities, and increased research and development costs for the IRAK1/4 inhibitor program. These increases were partially offset by decreased research and development costs related to the Phase 3 clinical trial for wAIHA and the ongoing Phase 3 clinical trial in high-risk hospitalized patients with COVID-19.

As of September 30, 2022, Rigel had cash, cash equivalents and short-term investments of $81.6 million, compared to $125.0 million as of December 31, 2021.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will host a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss financial results and provide an update on the business.

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AML
Acute myeloid leukemia (AML) is a cancer that starts in a person’s bone marrow but often quickly moves into the blood. AML develops from immature blood cells, known as myeloid cells, that are supposed to mature into white blood cells. However, the diseased myeloid cells do not function properly. They instead multiply rapidly, which causes normal blood cell production to fail. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.1

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.2 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.3

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that lead to the destruction of the body’s own red blood cells. Warm antibody AIHA (wAIHA), which is the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature. wAIHA affects approximately 36,000 adult patients in the U.S.4 and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for wAIHA, despite the unmet medical need that exists for these patients.

About COVID-19 & SYK Inhibition
COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.5 Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.6

SYK is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology such as pro-inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.7,8,9,10 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thrombo-inflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions
Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to ≥3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions
Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions
Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.