On May 5, 2022 Novartis reported the European Commission (EC) has approved Jakavi (ruxolitinib) for the treatment of patients aged 12 years and older with acute or chronic GvHD who have inadequate response to corticosteroids or other systemic therapies (Press release, Novartis, MAY 5, 2022, View Source [SID1234614189]).

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"Today, 30-60% of patients with GvHD do not respond to first-line steroid treatment, underscoring the need for new approaches to ensure long-term treatment goals are met," said Dr. Robert Zeiser, University Hospital Freiburg, Department of Haematology, Oncology and Stem Cell Transplantation, Freiburg, Germany. "The approval of Jakavi offers healthcare providers and patients with GvHD who remain dependent on or refractory to steroids a new way to manage this debilitating and life-threatening condition."

The approval of Jakavi follows the positive opinion granted in March by the Committee for Medicinal Products for Human Use of the European Medicines Agency, based on the Phase III REACH2 and REACH3 trials in which Jakavi demonstrated superiority in overall response rate (ORR) compared to best available therapy (BAT). Results of REACH2 showed 62% ORR with Jakavi at Day 28, compared to 39% for BAT; and REACH3 demonstrated a significantly improved ORR at week 24 (50% vs. 26%) with a higher best ORR (76% vs. 60%) vs. BAT, among steroid-refractory/dependent chronic GvHD patients2,3.

"Five out of ten patients who receive allogeneic stem cell transplants experience the serious and sometimes fatal symptoms of graft-versus-host disease," says Marie-France Tschudin, Novartis President of Innovative Medicines International and Chief Commercial Officer. "Jakavi, with this new indication in GvHD, will help to redefine treatment for patients who do not respond to first-line care."

GvHD occurs when donor cells see the recipient’s healthy cells as foreign and attack them. Symptoms of GvHD can appear in the skin, gastrointestinal tract, liver, mouth, eyes, genitals, lungs and joints. Approximately 50% of allogeneic stem cell transplant recipients will develop either acute or chronic GvHD. Both acute and chronic GvHD can be fatal and until now both have lacked an established standard of care for patients who do not adequately respond to first-line steroid treatment1,4-9. Currently, there are no other approved therapies for the treatment of GvHD after steroid failures1,2.

About Jakavi (ruxolitinib)
Jakavi (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. Jakavi is approved by the European Commission for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea and for the treatment of disease- related splenomegaly or symptoms in adult patients with primary myelofibrosis (MF) (also known as chronic idiopathic MF), post-polycythemia vera MF or post-essential thrombocythemia MF, and also for patients aged 12 years and older with acute or chronic GvHD who have inadequate response to corticosteroids or other systemic therapies. Jakavi is approved in over 100 countries for patients with MF, including EU countries, Switzerland, Canada, Japan and in more than 85 countries for patients with PV, including EU countries, Switzerland, Japan and Canada. The exact indication for Jakavi varies by country. Additional worldwide regulatory filings are underway in MF and PV.

Novartis licensed ruxolitinib from Incyte for development and commercialization outside the United States. Ruxolitinib is marketed in the United States by Incyte as Jakafi for adults with PV who have had an inadequate response to or are intolerant of hydroxyurea, for adults with intermediate or high-risk MF, for adult and pediatric patients 12 years and older with steroid-refractory acute GvHD, and adult and pediatric patients 12 years and older with chronic GvHD after failure of one or two lines of corticosteroids or other systemic therapy.

Jakavi is a registered trademark of Novartis AG in countries outside the United States. Jakafi is a registered trademark of Incyte. The safety and efficacy profile of Jakavi has not yet been established outside of its approved indications.

On May 5, 2022 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported financial results for the first quarter 2022 and provided a business update (Press release, argenx, MAY 5, 2022, View Source [SID1234613607]).

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In a separate press release issued today, argenx also announced positive results from the Phase 3 ADVANCE trial evaluating VYVGART for the treatment of adult patients with ITP. The primary endpoint, demonstrating a significantly higher proportion of VYVGART-treated patients achieved a sustained platelet response than patients receiving placebo, and additional key secondary endpoints were met.

"Our VYVGART commercial launch is off to a strong start, underscoring the significant unmet need for a new treatment option in gMG. We are very encouraged by the early demand from patients and physicians and our team continues to meet the challenge with outstanding execution and deep engagement with our key stakeholders. We look forward to our imminent commercial launch in Japan and an upcoming regulatory decision in Europe, which support our goal to make VYVGART available worldwide. We are confident that the relationships we are building today with the gMG community will establish a strong foundation to continue to deliver on behalf of patients," commented Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"The positive readout from our first registrational ITP trial highlights the promise of efgartigimod as a pipeline-in-a-product with the potential to reach a variety of IgG-mediated autoimmune diseases, even beyond the ten indications we are currently evaluating. We are on track to achieve our argenx 2025 goal to build the next great immunology company while bringing breakthrough innovations to patients and creating long-term value for our stakeholders."

FIRST QUARTER 2022 AND RECENT BUSINESS UPDATE

VYVGART Launch Progress

VYVGART is the first-approved neonatal Fc receptor (FcRn) blocker in the U.S. and Japan. VYVGART is approved in the U.S. for the treatment of adult generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for adult gMG patients. The global launch strategy is on track to make VYVGART available in Europe, China and Canada, as well as select additional regions.

Generated net product revenues of $21.2 million for first full quarter of VYVGART commercial launch in U.S.
Japan commercial launch to start this month following addition of VYVGART to National Health Insurance (NHI) drug price list on April 20, 2022
Decision from European Medicines Agency on Marketing Authorization Application expected in second half of 2022
Zai Lab to file for approval in China in mid-2022 and Medison in Israel in second quarter of 2022
Efgartigimod Research and Development

argenx is positioned to expand its leadership position in FcRn blockade to include ten total autoimmune indications by the end of 2022. Six registrational trials are ongoing with four new proof-of-concept trials to start this year across multiple therapeutic franchises.

Neuromuscular franchise
Biologics License Application (BLA) on track to be filed by end of year for subcutaneous (SC) efgartigimod for gMG, following positive topline results from Phase 3 ADAPT-SC trial
Topline data from registrational ADHERE trial of SC efgartigimod for chronic inflammatory demyelinating polyneuropathy (CIDP) expected in first quarter of 2023
Registrational ALKIVIA trial on track to start this quarter for three subtypes of idiopathic inflammatory myopathies (myositis), including immune-mediated necrotizing myopathy, anti-synthetase syndrome and dermatomyositis; interim analysis planned of first 30 patients of each subtype
Hematology franchise
Positive topline data of VYVGART for primary ITP reported today
Primary endpoint was met; significantly higher proportion of patients receiving VYVGART achieved a sustained platelet response than patients receiving placebo
Statistically significant separation from placebo in key platelet-derived secondary endpoints
Safety and tolerability profile confirmed in second indication
Topline data from second registrational ADVANCE-SC trial of SC efgartigimod for primary ITP expected in first quarter of 2023
Dermatology franchise
Enrollment expanded in registrational ADDRESS trial of SC efgartigimod for pemphigus vulgaris and foliaceus in order to manage ongoing impact of war in Ukraine; topline data now expected in second half of 2023
Registrational BALLAD trial ongoing of SC efgartigimod for bullous pemphigoid with interim analysis planned of first 40 patients
Proof-of-concept trials to be launched in collaboration with Zai Lab and IQVIA
Zai Lab to launch Phase 2 trials in lupus nephritis and membranous nephropathy in 2022 with argenx to lead global registrational programs for each potential indication
IQVIA to launch Phase 2 trials in primary Sjogren’s syndrome in second half of 2022 and COVID-19-mediated postural orthostatic tachycardia syndrome (POTS) in mid-2022
Pipeline Progress

argenx is developing ARGX-117 and ARGX-119, which both have pipeline-in-a-product potential for multiple autoimmune indications.

ARGX-117 (C2 inhibitor)
Proof-of-concept ARDA trial ongoing to evaluate safety, tolerability, and potential dosing regimen in multifocal motor neuropathy (MMN)
Phase 2 proof-of-concept trial expected to start in 2022 for prevention of delayed graft function and/or allograft failure after kidney transplantation
ARGX-119 (muscle-specific kinase (MuSK) agonist)
Phase 1 dose-escalation trial in healthy volunteers expected to start after Clinical Trial Application filing in fourth quarter of 2022
A subsequent Phase 1b trial will assess early signal detection in patients with congenital myasthenic syndrome and MuSK-associated myasthenia gravis
Upcoming Medical Meeting Presentations

14th Myasthenia Gravis Foundation of America International Conference on Myasthenia and Related Disorders (May 10-12, Miami, FL)
Society for Investigative Dermatology Annual Meeting (May 18-21, Portland, Oregon)
Annual Meeting of the Japanese Society of Neurology (May 18-22, Tokyo, Japan)
8th Congress of the European Academy of Neurology (June 25-28, Vienna, Austria)
17th International Congress on Neuromuscular Diseases (July 5-9, Brussels, Belgium)
DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three months ended March 31, 2022 was $31.5 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:

Product net sales from sales of VYVGART in the U.S. for the three months ended March 31, 2022 were $21.2 million, following the approval of VYVGART by the U.S. Food and Drug Administration (FDA) on December 17, 2021. No product sales were recognized during the comparable prior period.
Collaboration revenue for the three months ended March 31, 2022 was $2.2 million, compared to $158.2 million for three months ended March 31, 2021, resulting in a decrease of $155.9 million. The collaboration revenue for the three months ended March 31, 2021 was primarily attributable to the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue.
Other operating income for the three months ended March 31, 2022 was $8.1 million, compared to $20.4 million for three months ended March 31, 2021, resulting in a decrease of $12.3 million. During the three months ended March 31, 2021, the fair value of the argenx profit share in AgomAb Therapeutics NV increased by $11.2 million. There was no change in the fair value during the three months ended March 31, 2022.
Total operating expenses for the three months ended March 31, 2022 were $254.2 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:

Cost of sales for the three months ended March 31, 2022 amounted to $1.4 million. The cost of sales was recognized with respect to the sale of VYVGART in the U.S. during the first quarter of 2022. There was no cost of sales recognized in the comparable prior period.
Research and development expenses increased by $29.6 million for the three months ended March 31, 2022 to $152.0 million, compared to $122.3 million for the three months ended March 31, 2021. The increase resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in various indications and other clinical and preclinical programs.
Selling, general and administrative expenses totaled $100.9 million for the three months ended March 31, 2022, compared to $56.3 million for the three months ended March 31, 2021. The increase resulted primarily from higher professional and marketing fees linked to the commercialization of VYVGART in the U.S. and Japan and higher personnel expenses increased due to a planned increase in headcount.
Exchange losses totaled $7.2 million for the three months ended March 31, 2022, compared to $28.9 million for the three months ended March 31, 2021 and are mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial assets position in Euro.

Income tax totaled $2.9 million of tax income for the three months ended March 31, 2022, compared to $11.2 million of tax expense for the comparable prior period. Tax income for the three months ended March 31, 2022 consists of $5.0 million of income tax expense and $7.9 million of deferred tax income, compared to $6.2 million of income tax expense and $5 million of deferred tax expense for the comparable prior period.

Net loss for the three months ended March 31, 2022 was $227.2 million compared to $40.4 million for the comparable prior year period. On a per weighted average share basis, the net loss was $4.36 and $0.81 for the three months ended March 31, 2022 and 2021, respectively.

Cash, cash equivalents and current financial assets totaled $2,855.4 million as of March 31, 2022, compared to $2,336.7 million as of December 31, 2021. The increase in cash and cash equivalents and current financial assets resulted primarily from the closing of a global offering of shares, including a U.S. offering and a European private placement, which resulted in the receipt of $761.0 million in net proceeds in March 2022, partially offset by the net cash flows used in operating activities, primarily towards the commercial launch of VYVGART in the U.S. and Japan and continued investment in pipeline expansion.

FINANCIAL GUIDANCE

As of March 31, 2022, argenx had $2.9 billion in cash, cash equivalents and current financial assets. Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects to utilize approximately $1 billion of its available cash in 2022. The increased spend will support the global VYVGART launches, clinical development of efgartigimod in 10 indications and ARGX-117 in two indications, investment in the global supply chain, and continued focus on pipeline expansion through the Immunology Innovation Program.

EXPECTED 2022 FINANCIAL CALENDAR

July 28, 2022: HY 2022 financial results and business update
October 27, 2022: Q3 2022 financial results and business update
CONFERENCE CALL DETAILS

The first quarter 2022 business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

On May 5, 2022 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases, enhance gene therapies and mitigate unwanted immune responses to biologics, reported financial results for the first quarter ended March 31, 2022 and provided a business update (Press release, Selecta Biosciences, MAY 5, 2022, View Source [SID1234613636]).

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"In the first quarter of 2022, we executed across our pipeline and proactively took measures to extend our cash runway into mid-2024," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "In early April, we successfully completed an underwritten offering, raising an additional $38.7 million in gross proceeds, further strengthening our financial resources which we believe will carry us through key value-driving milestones and help Selecta realize the full potential of our leading precision immune tolerance platform. The synergistic effects of ImmTOR in combination with a Treg-selective IL-2 (ImmTOR-IL) has the potential to be a first-in-class, antigen-specific immunotherapy, with broad applications across all three pillars of our pipeline. Furthermore, we believe we are well-positioned to deliver on our clinical development strategy across our wholly owned pipeline while continuing to pursue strategic partnerships in gene therapy and biologics. We remain on track to deliver on multiple upcoming catalysts in 2022, including the initiation of a Phase 1 trial of SEL-302, our wholly owned gene therapy in combination with ImmTOR for the treatment MMA, the completion of the Phase 3 DISSOLVE clinical program in chronic refractory gout and several IND-enabling studies that collectively advance our strategy for re-imagining immunotherapy for autoimmune disease, unlocking the potential of AAV gene therapy and amplifying the efficacy of biologics."

Recent Highlights and Anticipated Upcoming Milestones:

Tolerogenic Therapies for Autoimmune Disease

ImmTOR with proprietary IL-2 protein agonist (ImmTOR-IL): Pre-clinically, Selecta has observed synergistic activity when ImmTOR is combined with engineered IL-2 molecules that are selective for Tregs. When ImmTOR-IL was co-administered with an antigen of interest in a preclinical study, the resulting preclinical data suggest that ImmTOR may have profound synergistic effects in further expanding antigen-specific Tregs when compared to ImmTOR alone, positioning it to be a potential first-in-class antigen-specific therapy for the treatment of autoimmune disease.
Selecta is working with its partner, Cyrus Biotechnology, to develop a next generation IL-2 molecule to combine with ImmTOR.
Selecta continues work toward identifying suitable target indications and accelerating the development of ImmTOR-IL to the clinic.
Primary biliary cholangitis (PBC): Selecta continues IND-enabling work on an ImmTOR platform approach to treating PBC.
Gene Therapies:

SEL-302 for methylmalonic acidemia (MMA): On March 9, 2022, the FDA removed the clinical hold on SEL-302 for the treatment of patients with MMA.
Selecta expects initiation of the Phase 1 clinical trial of SEL-302 in the second half of 2022.
SEL-018 IgG Protease (Xork): In collaboration with Genovis, Selecta continues to advance Xork, a next-generation IgG protease, to help address disease in those patients who are ineligible for gene therapies due to pre-existing anti-AAV antibodies. Selecta believes the novel combination of Xork and ImmTOR has the potential to simultaneously address two of the key hurdles in gene therapy today: pre-existing immunity and the inability to re-dose AAV gene therapies.
IND-enabling studies are expected to commence in 2022.
Biologic Therapies:

SEL-212 for chronic refractory gout: Selecta continues to advance DISSOLVE, the Phase 3 development program of SEL-212, which has been licensed to Swedish Orphan Biovitrum AB (publ.) ("Sobi").
On December 1, 2021, Selecta announced complete enrollment for DISSOLVE I, currently being run in the United States.
DISSOLVE II continues to enroll, with trial sites in the United States and four Eastern European countries. Screening and randomization in both Russia and Ukraine have been temporarily closed to preserve study supplies in these countries for those already enrolled. Moreover, 11 additional sites in the United States have been activated to speed enrollment and help mitigate any potential disruptions from the closure of screening and randomization in Russia and Ukraine, and DISSOLVE II enrollment has been increased to approximately 140 study participants.
DISSOLVE I & II studies are on track for completion in Q4 2022 with joint topline readout expected in Q1 2023.
ImmTOR with IgA1 protease for IgA nephropathy: Selecta is working with both Ginkgo Bioworks and IGAN Biosciences to identify and develop a next generation IgA protease to combine with ImmTOR.
Selecta anticipates enzyme candidate selection to be completed in 2022.
Corporate Updates:

Completed underwritten offering of common stock and warrants, raising approximately $38.7 million in gross proceeds.
Amended outstanding term loan to defer principal amortization period to April 1, 2023.
Performed strategic review and portfolio prioritization, pausing SEL-313, OTC-D gene therapy development and increasing focus on providing modality-enabling solutions to AAV gene therapy companies.
First Quarter 2022 Financial Results

Cash Position: Selecta had $118.8 million in cash, cash equivalents, marketable securities, and restricted cash as of March 31, 2022, as compared to cash, cash equivalents, marketable securities, and restricted cash of $129.4 million as of December 31, 2021. With the approximately $36.0 million in net proceeds raised from the April underwritten offering, Selecta believes its available cash, cash equivalents, restricted cash, and marketable securities will be sufficient to meet its operating requirements into mid-2024. Net cash used in operating activities was $11.9 million for the quarter ended March 31, 2022, as compared to $12.1 million of cash used in operating activities for the same period in 2021.

Collaboration and License Revenue: Collaboration and license revenue for the first quarter of 2022 was $34.0 million, as compared to $11.1 million for the same period in 2021. Revenue was primarily driven by the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program under the license agreement with Sobi.

Research and Development Expenses: Research and development expenses for the first quarter of 2022 were $17.7 million, as compared to $13.0 million for the same period in 2021. The increase in cost was primarily the result of expenses incurred for the preclinical programs, salaries and contract license and milestone payments.

General and Administrative Expenses: General and administrative expenses for the first quarter of 2022 were $5.5 million, as compared to $5.2 million for the same period in 2021. The increase in costs was primarily the result of stock compensation expenses.

Net Income (loss): For the first quarter of 2022, Selecta reported net income of $28.8 million, or basic net income per share of $0.23, compared to net loss of $(24.6) million, or $(0.22) per share, for the same period in 2021.

Conference Call and Webcast Reminder
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s first quarter 2022 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10157872. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On May 5, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and first quarter 2022 financial results (Press release, Sangamo Therapeutics, MAY 5, 2022, View Source [SID1234613652]).

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"This quarter, we have continued to progress multiple programs through clinical development and demonstrated, once again, Sangamo’s track record of advancing groundbreaking therapies in genomic medicine," said Sandy Macrae, Chief Executive Officer of Sangamo. "We dosed a total of five patients across three programs, including the first patient in our study for the treatment of kidney transplant rejection, in what we believe was the first in human dosing of an engineered CAR-Treg cell therapy product candidate. We believe this progress positions us well to advance transformational genomic medicines for patients in need and to generate long-term value for our shareholders."
Recent Business Highlights
Fabry disease – Dosed three additional patients, resulting in a total of nine patients dosed to date, thereby completing dose escalation for the Phase 1/2 study; Phase 3 planning progresses.
•We dosed two patients in Cohort 4 in the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry disease, at a dose level of 5e13 vg/kg.
•In addition, we dosed a third patient in Cohort 3, at the dose level of 3e13 vg/kg.
•In total, we have successfully dosed a total of nine patients across four cohorts to complete the dose escalation portion of the study.
•Enzyme replacement therapy (ERT) withdrawal was completed for a second patient, with no reports to date that the resumption of ERT is required in either patient.
•We expect to provide updated results from the STAAR study in the second half of 2022.
•We continue to actively prepare for the expansion cohorts, as well as a potential pivotal Phase 3 trial.
Sickle cell disease – Dosed fifth patient, the first with a product candidate manufactured using improved methods; Phase 3 planning progresses.
•We dosed the fifth patient in the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease, which is under development with Sanofi. This is the first 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.
•We plan to dose the remaining patients in this study by the end of the third quarter of 2022.
•We expect to provide updated results from the PRECIZN-1 study in the second half of 2022.
•Phase 3 enabling activities and manufacturing readiness are in progress.
•We continue to collaborate with Sanofi on an orderly transition of Sanofi’s rights and obligations under this program back to Sangamo on June 28.

Hemophilia A – FDA lifted clinical hold; Trial remains voluntarily paused; Pfizer expects to resume trial in Q3 2022.
•Pfizer announced that, in March 2022, the FDA lifted the clinical hold that had been placed on 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. Pfizer previously paused this trial when some of the patients experienced FVIII activity greater than 150% following treatment.
•Pfizer also announced that the voluntary pause remains in place until all necessary conditions are met, including approval of updated trial protocols by regulatory authorities.
•In addition, Pfizer announced that a patient with elevated FVIII levels reported a below-the-knee deep vein thrombosis. The patient had a history of thrombotic events prior to participation in the trial, which is a known risk factor for subsequent events and an exclusion criterion for participation in the AFFINE trial. The case was assessed to understand all potential contributing factors, including missed doses of investigator-prescribed direct oral anti-coagulants. The patient is reported to be doing well. The information was shared with trial investigators, health authorities and the independent external Data Monitoring Committee and Pfizer responded to queries from health authorities.
•Pfizer announced that it anticipates resuming this trial in the third quarter of 2022, with a pivotal data readout estimated in the second half of 2023.
•Over 50% of the patients have been enrolled in the Phase 3 AFFINE trial.
Renal Transplant Rejection – Believed to be first-ever in human dosing with an engineered CAR-Treg cell therapy candidate.
•We dosed the first patient in our 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 patient continues to do well, and no adverse events related to treatment have been reported.
•Dosing of the second patient is expected around the middle of 2022, based on their transplant schedule.
•We expect to complete dosing of the first cohort, comprised of three patients, by the end of 2022.
American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) – Eight abstracts accepted.
•A total of eight Sangamo abstracts were accepted for presentation at ASGCT (Free ASGCT Whitepaper) on May 16-19, 2022, including pre-clinical updates across our CAR-Treg autoimmune cell therapy platform, innovations in our genome engineering platform and advances in our AAV capsid engineering program.
First Quarter 2022 Financial Results
Consolidated net loss for the first quarter ended March 31, 2022 was $44.0 million, or $0.30 per share, compared to a net loss of $45.9 million, or $0.32 per share, for the same period in 2021.
Revenues
Revenues for the first quarter ended March 31, 2022 were $28.2 million, compared to $26.3 million for the same period in 2021.
The increase of $2.0 million in revenues was primarily attributed to an increase of $1.0 million related to our collaboration agreement with Novartis, an increase of $0.7 million related to our collaboration agreement with Biogen, and an increase of $0.4 million related to our collaboration agreement with Sanofi.

Total operating expenses on a GAAP basis for the first quarter ended March 31, 2022 were $73.5 million, compared to $72.6 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the first quarter ended March 31, 2022 were $65.8 million, compared to $65.1 million for the same period in 2021.
The increase in total operating expenses on a GAAP basis was primarily driven by our higher preclinical, clinical and lab supply and other R&D expenses along with our increased headcount to support the advancement of our clinical trials and our ongoing collaborations. Manufacturing and overhead costs also increased as we ramp up our internal manufacturing operations.
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities as of March 31, 2022 were $400.3 million, compared to $464.7 million as of December 31, 2021.
Financial Guidance for 2022 Reiterated (initial guidance provided on February 24, 2022)
On a GAAP basis, we continue to expect total operating expenses in the range of approximately $320 million to $350 million in 2022, which includes non-cash stock-based compensation expense.
We continue to expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $40 million, in the range of approximately $280 million to $310 million in 2022.
Conference Call
Sangamo will host a conference call today, May 5, 2022, at 4:30 p.m. Eastern Time, which will be open to the public. The call will also be webcast with live Q&A and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.
The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 3090098. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 3090098.

On May 5, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported business highlights and financial results for the first quarter ended March 31, 2022 (Press release, Agios Pharmaceuticals, MAY 5, 2022, View Source [SID1234613667]).

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"The U.S. approval of PYRUKYND in adults with PK deficiency was Agios’ first step toward changing treatment paradigms for people with genetically defined diseases, starting with developing the first disease-modifying therapy for a rare, debilitating, lifelong hemolytic anemia," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We are now poised to expand our impact for many more patients, including adults with PK deficiency in the EU, pediatric PK deficiency patients and people living with thalassemia, sickle cell disease and low- to intermediate-risk myelodysplastic syndrome. I am tremendously proud of our team’s incredible work on the U.S. launch of PYRUKYND, ongoing regulatory interactions in the EU, five pivotal clinical trials planned or underway, the expansion of our PK activation portfolio with AG-946 and our innovative research engine. We continue to execute in each of these arenas, motivated and inspired by our connections to patients."

First Quarter 2022 & Recent Highlights

Received approval from the U.S. Food and Drug Administration (FDA) for PYRUKYND, the first therapy for the treatment of hemolytic anemia in adults with PK deficiency and Agios’ first genetically defined disease medicine.
Executed commercial launch of PYRUKYND and generated approximately $0.8 million in net U.S. revenue for the first partial quarter following launch.
Published results from the ACTIVATE Phase 3 clinical study evaluating PYRUKYND in adults with PK deficiency who do not receive regular transfusions in the New England Journal of Medicine.
Completed the single ascending dose and multiple ascending dose healthy volunteer cohorts of the Phase 1 study of novel PK activator AG-946 and identified doses for the Phase 1 sickle cell disease cohort and Phase 2a study in low- to intermediate risk myelodysplastic syndrome (MDS).
Initiated the sickle cell disease cohort of the Phase 1 study of AG-946.
Published 2022 Environmental, Social and Governance (ESG) Report disclosing ESG initiatives and metrics aligned with the United Nations Sustainable Development Goals (UN SDGs) and the standards for the Biotechnology and Pharmaceuticals industry set by the Sustainability Accounting Standards Board (SASB).
Key Upcoming Milestones & Priorities

Agios expects to execute on the following key milestones and priorities in 2022:

Adult PK Deficiency: Receive European Medicines Agency (EMA) regulatory decision for PYRUKYND in adults with PK deficiency by year-end.
Pediatric PK Deficiency: Initiate Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in not regularly transfused and regularly transfused pediatric patients with PK deficiency, respectively, in mid-2022.
Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease by year-end.
Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
Data Presentations

Submitted new clinical and translational data to the European Hematology Association (EHA) (Free EHA Whitepaper) congress, to be held June 9-12 in Vienna and virtually, including:
New patient-reported outcomes (PRO) data from ACTIVATE Phase 3 study of PYRUKYND
Data demonstrating the normalization of hemoglobin levels with long-term treatment of PYRUKYND in adults with PK deficiency
Additional PK deficiency comorbidities and complications data from the PEAK registry
Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
First Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND for the first quarter of 2022 was $0.8 million. This revenue reflects the first partial quarter of PYRUKYND launch, following FDA approval on February 17, 2022.

Cost of Sales: Cost of sales for the first quarter of 2022 were $0.3 million.

Non-Operating Income: Non-operating income included approximately $2.7 million from TIBSOVO (ivosidenib) royalties for the first quarter of 2022.

Research and Development (R&D) Expenses: R&D expenses were $70.1 million for the first quarter of 2022 compared to $57.7 million for the first quarter of 2021. The year-over-year increase in R&D was driven primarily by start-up costs for the PYRUKYND pivotal studies in thalassemia and sickle cell disease and planned increases in research activities, offset by closeouts of the ACTIVATE and ACTIVATE-T studies.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $31.5 million for the first quarter of 2022 compared to $33.6 million for the first quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to lower workforce expenses.

Net Loss from Continuing Operations: Net loss from continuing operations was $94.8 million for the first quarter of 2022 compared to a net loss of $90.9 million for the first quarter of 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2022, were $1.2 billion compared to $2.4 billion as of March 31, 2021. The year-over-year decrease is attributable to operating expenses and 16.2 million shares of common stock that the company repurchased for $802.5 million during the second through fourth quarters of 2021. Agios expects that its cash, cash equivalents and marketable securities will enable the company to execute its operating plan 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 first quarter 2022 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 refer to conference ID 5738266. 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.