Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2021 Financial Results

On August 5, 2021 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported recent business highlights and second quarter 2021 financial results.

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"This is an exciting time at Sangamo, as we look forward to multiple clinical catalysts over the next several quarters representing potential near-term value drivers, while we continue to advance our promising preclinical pipeline focused on the emerging areas of CAR-Tregs and transcriptional regulation, representing potential mid-term value drivers"

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"This is an exciting time at Sangamo, as we look forward to multiple clinical catalysts over the next several quarters representing potential near-term value drivers, while we continue to advance our promising preclinical pipeline focused on the emerging areas of CAR-Tregs and transcriptional regulation, representing potential mid-term value drivers," said Sandy Macrae, Chief Executive Officer of Sangamo.

Recent Business Highlights

We dosed a fourth patient in our wholly-owned Phase 1/2 STAAR study evaluating ST-920 gene therapy for Fabry disease, completing dosing of the second cohort. Based on initial safety data from patients dosed to date, the Safety Monitoring Committee recommended dose escalating to the third dose as planned under the study protocol. We are currently screening patients for the third dose cohort.
Biogen selected a fourth neurological disease gene target under our collaboration agreement, and we have begun early research activities on therapies addressing this target.
We appointed Prathyusha Duraibabu as Chief Financial Officer and Scott Willoughby as General Counsel and Corporate Secretary.
Second Quarter 2021 Financial Results

Consolidated net loss attributable to Sangamo for the second quarter ended June 30, 2021 was $47.2 million or $0.33 per share, compared to a net loss attributable to Sangamo of $35.9 million or $0.26 per share for the same period in 2020.

Revenues

Revenues for the second quarter ended June 30, 2021 were $27.9 million, compared to $21.6 million for the same period in 2020.

The increase of $6.3 million in revenues was primarily due to the recognition of upfront license fees and research revenue of $9.6 million under our collaboration agreement with Novartis and $2.6 million higher revenue related to our collaboration agreement with Biogen. These increases were partially offset by a decrease of $3.8 million in research revenue related to our giroctocogene fitelparvovec and C9ORF72 collaboration agreements with Pfizer, due to completion of our activities under these collaborations, a decrease of $1.1 million in research revenue related to our collaboration agreement with Kite, and a decrease of $0.9 million in research revenue related to our collaboration agreement with Sanofi.

GAAP and Non-GAAP operating expenses

Total operating expenses on a GAAP basis for the second quarter ended June 30, 2021 were $76.6 million compared to $59.4 million for the same period in 2020. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the second quarter ended June 30, 2021 were $67.1 million compared to $52.7 million for the same period in 2020.

The increase in total operating expenses on a GAAP basis was primarily driven by our higher clinical and manufacturing supply expenses along with our increased headcount to support the advancement of our clinical trials and our new collaborations.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of June 30, 2021 were $578.9 million compared to $692.0 million as of December 31, 2020.

Guidance for 2021 Reiterated (initial guidance provided on February 24, 2021)

On a GAAP basis, we expect total operating expenses, including non-cash stock-based compensation expenses, to be in the range of approximately $285 million to $305 million for the year ended December 31, 2021.

On a non-GAAP basis, we expect total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $30 million, to be in the range of approximately $255 million to $275 million for the year ended December 31, 2021.

Conference Call

Sangamo will host a conference call today, August 5, 2021, 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 1971045. 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 1971045.

Vaxart Provides Business Update and Reports Second Quarter 2021 Financial Results

On August 5, 2021 Vaxart, Inc. (Nasdaq: VXRT) reported its business update today for the second quarter of 2021, reporting strong forward momentum in development of oral tablet vaccines that it believes can revolutionize public health (Press release, Aviragen Therapeutics, AUG 5, 2021, View Source [SID1234585870]).

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Vaxart, a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, announced its business progress and provided financial results for the second quarter ended June 30, 2021.

"By any measure, Vaxart showed real forward momentum during the last quarter," CEO Andrei Floroiu said. "We strengthened our balance sheet, made major research advances, deepened our scientific knowledge, grew the company, and took the company closer to our goal of developing disruptive oral tablet vaccines that can have a material impact on the world’s public health."

Recent Business Highlights

Corporate Developments

Vaxart raised $36.2 million in net proceeds from its $250 million at-the-market facility in the three months ended June 30, 2021.
Vaxart signed an exclusive worldwide licensing agreement with Altesa Biosciences, Inc. to allow Altesa to develop and commercialize Vaxart’s Vapendavir, a clinical-stage broad spectrum antiviral.
The licensing agreement provides for milestone payments up to $130 million and royalties for global Vapendavir sales.
Building out the infrastructure necessary to support its groundbreaking research, Vaxart added to its management team and strengthened its research, clinical, and manufacturing groups and R&D infrastructure. The number of R&D employees grew by 36% in the quarter to 49 full-time employees.
Pre-Clinical and Clinical

Platform-Wide Developments

Vaxart completed its first boosting study, which showed in clinical trials that its norovirus vaccine built on the VAAST platform can successfully boost immune responses in subjects previously vaccinated with a Vaxart oral vaccine more than a year earlier.
The research supports the company’s thesis that its oral tablet vaccines have the potential to be used annually for indications that may require a boost such as flu or COVID-19.
The data announced that Vaxart’s vaccines may not suffer from certain antibody response challenges that can occur with viral vector-based vaccines.
COVID-19 Vaccine Developments

While the nation’s death and illness tolls have fallen markedly since the beginning of the last quarter, new variants such as the Delta strain continue to worry national political and health leaders. Vaxart broadened its research into the various COVID-19 strains while continuing its development of an oral tablet vaccine. Among the most significant developments in the second quarter:

The U.S. Food and Drug Administration cleared Vaxart to move to its next phase of COVID-19 testing with a study of its next generation S-1 construct.
Vaxart is manufacturing the vaccine necessary to start the Phase 2 clinical study of its S-Wuhan construct and expects to begin this study shortly.
A Non-Human Primate study of the S&N construct along with S-Wuhan and S-South Africa constructs showed optimal performance by the S-Wuhan construct and also cross reactivity against all variants tested. The decision was made to the S-Wuhan vaccine construct into Phase 2.
Norovirus Vaccine Developments

Norovirus is a highly infectious illness that affects around 20 million Americans annually, with an annual economic impact of approximately $10.5 billion in the United States.

In addition to its boosting study, Vaxart enrolled the first subjects in a Phase 1b placebo-controlled, dose-ranging, repeat dose trial investigating its oral norovirus vaccine candidate in elderly subjects aged 55 – 80 years.
This study is designed to evaluate the safety and immunogenicity of Vaxart’s candidate, which is the only clinical stage norovirus oral tablet vaccine actively being developed.
Financial Results for the Three Months Ended June 30, 2021

Vaxart ended the quarter with cash, cash equivalents, and available-for-sale debt securities of $198.9 million, compared to $177.3 million as of March 31, 2021. The increase was primarily due to net receipts of $36.2 million from the Company’s $250 million at-the-market facility entered into in October 2020 and $0.9 million from the exercise of warrants and options, partially offset by $13.2 million of cash used in operations and $2.2 million spent on property and equipment.
Vaxart reported a net loss of $16.1 million for the second quarter of 2021 compared to $9.0 million for the second quarter of 2020. Net loss per share for the second quarter of 2021 was $0.13, compared to a net loss of $0.12 in the second quarter of 2020. The small increase in net loss per share was due to the increase in net loss being mostly offset by the increase in the weighted average number of shares outstanding.
Revenue for the second quarter of 2021 was $112,000 compared to $523,000 in the second quarter of 2020. The decrease was principally due to the absence of royalty revenue related to Relenza sales in Japan as a result of the patent expiring and a reduction in royalty revenue related to Inavir sales in Japan as a result of lower incidences of seasonal influenza.
Research and development expenses were $10.7 million for the second quarter of 2021 compared to $5.1 million for the second quarter of 2020. The increase was mainly due to manufacturing and clinical trial expenses related to the COVID-19 and norovirus vaccine candidates.
General and administrative expenses were $5.2 million for the second quarter of 2021 compared to $3.9 million for the second quarter of 2020. The increase was mainly due to higher insurance expenses and an increase in headcount and related costs.

Codiak BioSciences Reports Second Quarter 2021 Financial Results and Operational Progress

On August 5, 2021 Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported second quarter 2021 financial results and operational progress (Press release, Codiak Biosciences, AUG 5, 2021, View Source [SID1234585869]).

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"We continue to be intensely focused on execution of our two lead clinical programs, with plans to deliver near-term data readouts from both the exoSTING and exoIL-12 trials. We anticipate sharing safety data and a differentiating pharmacologic profile from initial dose escalation cohorts in the Phase 1/2 trial of exoSTING in the fourth quarter of 2021, and we expect to report initial safety, PK/PD and efficacy data from CTCL patients in the exoIL-12 Phase 1 trial by year end," said Douglas E. Williams, Ph.D., President and Chief Executive Officer of Codiak. "We are also prosecuting multiple preclinical programs that together demonstrate the versatility of our platform and we look forward to filing an IND this year for our third engineered exosome therapeutic candidate, exoASO-STAT6 for the treatment of myeloid-rich cancers."

Second Quarter 2021 and Recent Highlights

Presented new preclinical data on exoAAV, exoVACC, exoASO-STAT6, and the ability of engineered exosomes to direct tropism at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 24th Annual Meeting
Published manuscript highlighting the exoSTING preclinical program in Communications Biology, a Nature Research publication
Presented tolerability, pharmacokinetic (PK)/pharmacodynamic (PD) data from healthy volunteer portion of the exoIL-12 Phase 1 clinical trial and preclinical data from the exoASO-STAT6 program at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
Appointed Jennifer Wheler, M.D., as Chief Medical Officer
Added Anne-Virginie Eggimann, M.Sc., to Board of Directors
Anticipated Milestones and Events

Initial safety, PK, and PD data from dose escalation cohorts 1-3 on the Phase 1/2 trial of exoSTING (CDK-002) in patients with advanced/metastatic solid tumors with injectable lesions expected in the fourth quarter of 2021
IND filing for exoASO-STAT6 (CDK-004) anticipated during the second half of 2021
Initial safety, PK/PD and efficacy data in CTCL patients from the Phase 1 trial of exoIL-12 (CDK-003) expected by year-end 2021
Second Quarter 2021 Financial Results

Total revenues for the quarter ended June 30, 2021, were $0.9 million, compared to $0.2 million for the same period in 2020. This increase was due to deferred revenue recognized from ongoing research and development collaborations.

Net loss for the quarter ended June 30, 2021, was $21.8 million, compared to a net loss of $15.9 million for the same period in 2020. Net loss for the quarter was driven primarily by clinical development, general and administrative, and personnel expenses, and ongoing development of the engEx Platform.

Research and development expenses were $15.4 million for the quarter ended June 30, 2021, compared to $11.6 million for the same period in 2020. The increase in research and development expenses was driven by an increase in engEx Platform expenses, partially offset by decreased manufacturing and preclinical costs as Codiak’s lead candidates progressed into the clinic during the second half of 2020.

General and administrative expenses were $6.9 million for the quarter ended June 30, 2021, compared to $4.4 million for the same period in 2020. The increase was driven primarily by an increase in general and administrative headcount to support our overall growth and our transition to becoming a public company.

As of June 30, 2021, Codiak had cash and cash equivalents of approximately $113.7 million.

Kadmon Provides Business Update and Reports Second Quarter 2021 Financial Results

On August 5, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the second quarter of 2021 (Press release, Kadmon, AUG 5, 2021, View Source [SID1234585868]).

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"The recent U.S. FDA approval of REZUROCK marked a transformative event for Kadmon and for patients living with cGVHD. REZUROCK represents a paradigm shift in the cGVHD treatment landscape by uniquely addressing both the immune and fibrotic components of the disease," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to bringing this meaningful new therapy to patients in the U.S. by the end of this month."

Dr. Waksal added, "Our momentum continues as we advance our portfolio of product candidates. Initial data from our open-label, Phase 2 trial of belumosudil for the treatment of systemic sclerosis is anticipated by year-end 2021. The recent positive initial safety data presented at ASCO (Free ASCO Whitepaper) on KD033, our anti-PD-L1/IL-15 fusion protein, supports our confidence in the therapeutic potential of IL-15 for cancer. We look forward to sharing additional clinical data from this trial in the fourth quarter of 2021."

2021 Program Updates and Milestones:

REZUROCK (belumosudil)

On July 16, 2021, the U.S. Food and Drug Administration (FDA) approved REZUROCK (belumosudil) for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy:
Commercial launch activities are underway, with a focus on generating awareness of REZUROCK’s differentiated clinical value and facilitating market access
Experienced hematology/oncology sales team and account directors are fully trained and have begun engaging with healthcare providers at the top 100 transplant centers, where ~90% of patients are treated
REZUROCK availability is anticipated by late August through a network of rare hematology/oncology specialty pharmacies and distributors
Kadmon launched Kadmon ASSIST, a comprehensive suite of patient financial and hub support services, including dedicated nurse practitioners on call to facilitate patient education and a successful clinical experience
Kadmon announced on August 4 that the National Comprehensive Cancer Network (NCCN) added REZUROCK tablets to its Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Hematopoietic Cell Transplantation (HCT) in the Pre-Transplant Recipient Evaluation and Management of Graft-Versus-Host Disease in the United States.
Results from the pivotal ROCKstar clinical trial of REZUROCK for the treatment of cGVHD were published in the journal Blood; available online here
Clinical

Belumosudil in SSc (systemic sclerosis)

Present initial data from the open-label Phase 2 clinical trial of belumosudil in patients with SSc (KD025-215) by year-end 2021
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in SSc (KD025-209)
KD033

Enrollment is ongoing in Cohort 4 (100 µg/kg) in the dose-escalation, Phase 1 clinical trial of KD033, Kadmon’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors (KD033-101)
Initial safety data presented at ASCO (Free ASCO Whitepaper) 2021 demonstrated that KD033 has been well tolerated at doses up to 50 µg/kg, with no dose-limiting toxicities reported
The Company plans to present additional clinical data from KD033-101 in the fourth quarter of 2021
Financial Results

Second Quarter 2021 Results

Loss from operations for the three and six months ended June 30, 2021 was $32.0 million and $59.4 million, respectively, compared to $26.9 million and $43.0 million for the same period in 2020. The six months ended June 30, 2020 included $6.0 million in one-time license revenues related to the Meiji strategic partnership.

The $5.6 million and $11.2 million increase in operating expenses for the three and six months ended June 30, 2021, respectively, as compared to 2020 was primarily related to belumosudil commercial launch readiness activities, non-cash stock compensation expenses and direct external research and development costs of developing our preclinical product candidates from our immuno-oncology platform.

Liquidity, Capital Resources and Cash Runway

At June 30, 2021, the Company’s cash, cash equivalents and marketable debt securities totaled $270.5 million, compared to $123.9 million at December 31, 2020. The Company expects its current financial position to be sufficient to fund its operations and capital expenditures into 2023.

About REZUROCK (belumosudil)

REZUROCK (belumosudil) is the first and only approved therapy targeting Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. REZUROCK is approved in the United States for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy. For more information, visit www.REZUROCK.com.

Kadmon is also developing belumosudil for the treatment of systemic sclerosis. The FDA has granted Orphan Drug Designation to belumosudil for the treatment of systemic sclerosis.

Acceleron Reports Second Quarter 2021 Financial Results

On August 5, 2021 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Acceleron Pharma, AUG 5, 2021, View Source [SID1234585867]).

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"We were very pleased to highlight clinical updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept reported at the annual ATS medical meeting and outline our plans for future long-term growth in rare pulmonary diseases at our Research & Development Day during the second quarter," said Habib Dable, President and Chief Executive Officer of Acceleron. "Beyond our sotatercept clinical program in pulmonary arterial hypertension, most recently, we announced plans to expand the development of sotatercept into a Phase 2 trial in patients with Group 2 pulmonary hypertension and develop ACE-1334 in systemic sclerosis-associated interstitial lung disease. To date, our pulmonary pipeline has grown to include seven ongoing and planned clinical trials, showcasing our long-term commitment to becoming a global leader in rare pulmonary disease."

Added Mr. Dable: "With respect to our hematology program, along with our commercial partner Bristol-Myers Squibb Company (Bristol Myers Squibb), we continue to be pleased with product uptake. Our joint commercial teams are presently focused on expanding the reach to appropriate patients earlier in their MDS journey—where there is particular demand—along with the optimal dose of REBLOZYL to maximize patient benefit and increased duration of treatment in this population to drive further growth this year and beyond. In June, we presented results from the BEYOND Phase 2 trial of luspatercept during the Presidential Symposium at EHA (Free EHA Whitepaper). The study achieved its primary endpoint of an increase in hemoglobin of at least 1 gram per deciliter in the luspatercept treated group compared to placebo for the treatment of anemia in adults with non-transfusion dependent beta-thalassemia, supporting the rationale for its potential development in additional patient groups."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Hypertension
Sotatercept acts as an investigational reverse-remodeling agent proposed to rebalance TGF-beta superfamily signaling. In preclinical models of PAH, sotatercept reversed pulmonary arterial wall and right ventricular remodeling that are hallmarks of the disease.

In May, Acceleron presented updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept in patients with PAH at the American Thoracic Society (ATS) 2021 International Conference.
Enrollment is ongoing in the registrational STELLAR Phase 3 trial in patients with PAH.
Study start up activities are underway for the HYPERION and ZENITH Phase 3 trials of sotatercept in expanded PAH patient populations.
Acceleron expects to initiate the CADENCE Phase 2 trial in patients with pulmonary hypertension with left heart disease this year.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)
ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis.

Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD by year-end 2021.
Hematology

REBLOZYL (luspatercept-aamt):
REBLOZYL is the first and only approved erythroid maturation agent designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

Acceleron recognized approximately $25.6 million in royalty revenue from approximately $128 million in net sales of REBLOZYL in the second quarter of 2021. This compares with approximately $22.4 million in royalty revenue from approximately $112 million in net sales of REBLOZYL in the first quarter of 2021.
In June, the Companies presented results from multiple abstracts on luspatercept at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress.
Results from the BEYOND Phase 2 trial of luspatercept in adult patients with non-transfusion dependent beta-thalassemia were presented during the Presidential Symposium, which honors the top six research papers submitted for presentation at the meeting.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Enrollment is ongoing in the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
Corporate Highlights

Acceleron published its inaugural Environmental, Social, and Governance (ESG) report in June, which provides a comprehensive summary of the Company’s broad range of ESG initiatives. Acceleron has adopted the accounting standards for the biotechnology and pharmaceuticals industry from the Sustainability Accounting Standards Board (SASB) to help develop and prioritize areas for inclusion in its ESG report. The full ESG report can be found on Acceleron’s Investor Relations website by clicking here.
On June 22, 2021, members of the Acceleron leadership team, along with external experts, reviewed the Company’s numerous ongoing and planned trials in rare pulmonary diseases, and highlighted Acceleron’s vision and strategy for long-term growth. An archived recording of the video webcast of the presentations and question and answer sessions is accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com.
Financial Results

Cash Position – Cash, cash equivalents and investments as of June 30, 2021 were $712.5 million, compared with $857.5 million as of December 31, 2020. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the second quarter of 2021 was $27.9 million, which includes $2.3 million of cost share revenue and $25.6 million of royalty revenue from net sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
R&D Expenses – GAAP R&D expenses were $56.1 million for the second quarter of 2021. Non-GAAP R&D expenses were $49.3 million for the second quarter of 2021, excluding $5.9 million and $0.9 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $35.5 million for the second quarter of 2021. Non-GAAP SG&A expenses were $27.8 million for the second quarter of 2021, excluding $7.6 million and $0.1 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Net Loss – The Company’s GAAP net loss for the second quarter of 2021 was $63.5 million, or $1.05 per share. Non-GAAP adjusted net loss for the second quarter was $49.0 million, or $0.81 per share, excluding $13.5 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Non-GAAP Financial Measures

Acceleron supplements its results of operations prepared in accordance with U.S. generally accepted accounting principles, or GAAP, with certain non-GAAP financial measures, including non-GAAP R&D expense, non-GAAP SG&A expense, adjusted net loss and adjusted net loss per share, that exclude stock-based compensation expense and depreciation and amortization expense. These results should not be viewed as a substitute for the Company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures provide investors with additional insight into underlying trends of the Company’s ongoing business, and are important in comparing current results with prior period results. Investors are cautioned that there are material limitations associated with the use of non-GAAP financial measures. In addition, other companies may report similarly titled non-GAAP measures, but calculate them differently, which reduces their usefulness as a comparative measure. In the reconciliation tables below, Acceleron presents these non-GAAP financial measures reconciled to their comparable GAAP financial measures.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its second quarter 2021 financial results on August 5, 2021, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #5736809.

An archived version of the webcast will be available for replay on the Company’s website for approximately one year.