On November 4, 2021 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported financial results for the quarter ended September 30, 2021 (Press release, Allogene, NOV 4, 2021, View Source [SID1234594427]).

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"While our clinical studies are on hold, our work to bring AlloCAR T products to patients continues with a strong sense of urgency. We remain confident in our platform and the potential of our AlloCAR T candidates to meaningfully improve the lives of patients living with cancer," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our teams are busy progressing our preclinical programs, advancing production at our Cell Forge 1 manufacturing facility, and preparing for data presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. In the meantime, we continue to work closely with the U.S. Food and Drug Administration (FDA) on the review of the end of Phase 1 materials in support of a pivotal trial for ALLO-501A as well as to resolve the clinical hold and we look forward to providing an update after further interaction with the agency."

Pipeline Updates
The FDA placed a hold on the Company’s AlloCAR T clinical trials (ALPHA, ALPHA2, IGNITE, TRAVERSE, UNIVERSAL) following a report of a chromosomal abnormality in a single patient treated in the ALPHA2 study. Allogene is working with the FDA to address next steps to resolve the hold.

In parallel with the clinical hold investigation, the FDA is actively reviewing the end of Phase 1 materials the Company submitted in support of the pivotal trial for ALLO-501A.
Anti-CD19 Program: ALPHA and ALPHA2 Trials

Abstracts from the ALPHA (poster session) and ALPHA2 (oral session) trials were selected for presentation at the ASH (Free ASH Whitepaper) meeting in early December. The data contained in the abstracts continue to show the utility of AlloCAR T therapy in patients with relapsed/refractory non-Hodgkin lymphoma. Updated results will be presented at the meeting.

In the ALPHA2 trial, consolidation dosing appeared to be well tolerated with the potential for enhanced efficacy compared to a single dose of ALLO-501A. In the consolidation cohort, both the overall response rate (ORR) and complete response (CR) rate were 67% with all three partial responses (PRs) converting to CR following consolidation. All four consolidation patients who achieved a CR remained in CR as of the July 2021 data cut-off. The safety profile of ALLO-501A was manageable in both single dose and consolidation cohorts. Events of interest in the single dose cohort were previously reported at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. In the consolidation cohort, there was no cytokine release syndrome (CRS), no graft-versus-host disease (GvHD), no immune effector cell-associated neurotoxicity syndrome (ICANS), no dose-limiting toxicities (DLTs), no dose reductions, no Grade 3+ infections and infusion-related reactions were Grade 2. Among all treated patients, cytopenias were the most common adverse event and occurred in 72% of patients.

In the ALPHA trial as of the July data cutoff for the ASH (Free ASH Whitepaper) abstract, five additional patients were treated relative to the data previously reported at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. ORR and CR rates remain at 75% and 50%, respectively. In patients with LBCL (n=13), the ORR was 62% and the CR rate was 46%. In patients with FL (n=23), the ORR was 83% and the CR rate was 52%. Four of the seven patients (all FL) enrolled in the consolidation cohort were evaluable for assessment after consolidation dosing at the time of the data cutoff with an ORR and CR rate of 100% and 75%, respectively. The percent of patients remaining in CR at six months following a single infusion was 36% in LBCL, which is similar to 6-month CR rates reported in the pivotal trials of autologous CAR T therapies, with the longest ongoing CR at 15+ months, as of the data cut-off. The 6-month CR rate in FL was 28%. No cases of GvHD or DLTs were observed. As noted previously, one case of Grade 3 ICANS was reported. Grade 1/2 CRS occurred in 22% of patients with one case of Grade 3 CRS. All were managed with standard protocols. Cytopenias were the most common adverse event and occurred in 83% of patients. Infection rates remained similar to those observed in autologous CAR T trials.
Anti-BCMA AlloCAR T Program: UNIVERSAL Trial

In August, ALLO-715 received Orphan Drug Designation (ODD) from the FDA for the treatment of multiple myeloma (MM).

Updated results from the UNIVERSAL trial investigating ALLO-715 as a monotherapy in patients with relapsed/refractory MM will be presented at an oral session at ASH (Free ASH Whitepaper). Findings from the UNIVERSAL trial indicate an allogeneic CAR T therapy can be delivered rapidly without the need for bridging therapy to patients with relapsed/refractory multiple myeloma, with single dose of therapy capable of inducing deep responses. The ORR was 62% with a very good partial response or better (VGPR+) rate of 39% in the 26 patients treated at the highest two dose levels (320 and 480 x 106 CAR+ cells). Median follow-up for these patients was 7.4 months with a median duration of response of 8.3 months. Of the 10 patients with a best response of VGPR+, eight were found to be minimal residual disease (MRD) negative. No GvHD was observed. The most common Grade 3+ adverse events included anemia, neutropenia, lymphopenia, and thrombocytopenia. CRS was reported in 52% of patients, in all cases Grade 1/2 except for one patient with Grade 3. One patient with Grade 2 CRS experienced Grade 1 neurotoxicity that resolved. Grade 3+ infections occurred in 13% of patients, including two previously reported Grade 5 events (fungal pneumonia and adenovirus hepatitis).
Third Quarter Financial Results

Research and development expenses were $58.7 million for the third quarter of 2021, which includes $10.1 million of non-cash stock-based compensation expense.

General and administrative expenses were $19.0 million for the third quarter of 2021, which includes $10.8 million of non-cash stock-based compensation expense.

Net loss for the third quarter of 2021 was $78.2 million, or $0.57 per share, including non-cash stock-based compensation expense of $20.9 million.

The Company had $861.7 million in cash, cash equivalents, and investments as of September 30, 2021.
2021 Financial Guidance

Allogene continues to expect full year GAAP Operating Expenses to be between $300 and $330 million including estimated non-cash stock-based compensation expense of $80 to $90 million and excluding any impact from potential business development activities.
Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 1924859. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On November 4, 2021 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the third quarter of fiscal year 2021, which include the consolidated financial results of GRAIL (Press release, Illumina, NOV 4, 2021, View Source [SID1234594426]).

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Third quarter consolidated results:

Revenue of $1,108 million, a 40% increase compared to the prior year period
GAAP net income for the quarter of $317 million, or $2.08 per diluted share, which included a $900 million gain from our previously held investment in GRAIL and $654 million in day one compensation expense related to the GRAIL acquisition. This compared to $179 million, or $1.21 per diluted share, for the prior year period
Non-GAAP net income for the quarter of $221 million, or $1.45 per diluted share, which included dilution from GRAIL non-GAAP operating loss of $0.19 per diluted share and incremental dilution from the 9.8 million shares issued to fund the GRAIL acquisition of $0.06 per diluted share. This compared to $150 million, or $1.02 per diluted share, for the prior year period. Non-GAAP net income excludes gain on previously held investment in GRAIL, day one compensation related expense and other acquisition-related expenses (see the "Reconciliation Between GAAP and Non-GAAP Net Income" table for a reconciliation of these GAAP and non-GAAP financial measures)
Cash flow from operations of $(272) million compared to $153 million in the prior year period
Free cash flow (cash flow from operations less capital expenditures) of $(324) million for the quarter compared to $105 million in the prior year period
"Illumina’s financial results again exceeded expectations in the third quarter led by record shipments for both clinical and research," said Francis deSouza, Chief Executive Officer. "Clinical market expansion is driving momentum in the core business, including significant demand in testing for therapy selection in oncology as we continue to advance genomics as a standard of care in the clinic. Our teams’ continued execution to meet this robust demand will enable a strong finish to an exceptional 2021."

Gross margin in the third quarter of 2021 was 69.5% compared to 66.2% in the prior year period. Excluding amortization of acquired intangible assets, non-GAAP gross margin was 71.2% for the third quarter of 2021 compared to 67.4% in the prior year period.

Research and development (R&D) expenses for the third quarter of 2021 were $436 million compared to $172 million in the prior year period. Excluding acquisition-related compensation expense, non-GAAP R&D expenses as a percentage of revenue were 21.7% compared to 21.2% in the prior year period.

Selling, general and administrative (SG&A) expenses for the third quarter of 2021 were $879 million compared to $192 million in the prior year period. Excluding day one compensation related expense and other acquisition-related expenses, and fair value adjustments on contingent consideration liabilities, non-GAAP SG&A expenses as a percentage of revenue were 26.0% compared to 24.8% in the prior year period.

Depreciation and amortization expenses were $65 million and capital expenditures for free cash flow purposes were $52 million during the third quarter of 2021. At the close of the quarter, the company held $1.3 billion in cash, cash equivalents and short-term investments, compared to $3.5 billion as of January 3, 2021.

Third quarter segment results:

Following the acquisition of GRAIL on August 18, 2021, we have two reportable segments, Core Illumina and GRAIL. GRAIL financial results are reflected for the period after the acquisition.

Updates since our last earnings release:

Closed the GRAIL acquisition to accelerate patient access to life-saving, multi-cancer early-detection tests; we are holding GRAIL as a separate entity pending EU regulatory review of the merger
Announced TSO 500 partnership with Merck to develop and commercialize tests that identify genetic mutations used in the assessment of homologous recombination deficiency (HRD)
Co-authored study published in JAMA Pediatrics reporting findings from the NICUSeq Randomized Time-Delayed Trial, demonstrating whole genome sequencing outperforms usual care by two-fold in diagnostic efficacy and clinical management of acutely-ill newborns with suspected genetic disease
Announced an agreement with Israel’s Ministry of Health for a pilot program to implement the use of whole-genome sequencing in critically-ill infants suspected of having a genetic disorder in neonatal intensive care units
Expanded presence in Latin America with the creation of Illumina Colombia S.A.S. and Illumina México Productos de Biotecnología to advance clinical business in the region and increase support for local customers and partners
Announced net zero global greenhouse gas emissions commitment by 2050, including milestone targets of 46% reduction in emissions and 100% renewable electricity by 2030
GRAIL announced Galleri was approved for prescription by the New York State Department of Health, making the test commercially available in New York, and marking a significant regulatory milestone.
NHS launched UK trial of Galleri under a commercial partnership program that aims to confirm Galleri’s clinical and economic performance in the NHS system as a precursor to its routine use by the NHS
Appointed Robert Ragusa, former Chief Operations Officer of Illumina, as Chief Executive Officer of GRAIL
Financial outlook and guidance

The non-GAAP financial guidance discussed below reflects certain pro forma adjustments to assist in analyzing and assessing our core operational performance, including our Core Illumina and GRAIL segments. Please see our Reconciliation of Non-GAAP Financial Guidance included in this release for a reconciliation of the GAAP and non-GAAP financial measures.

For fiscal 2021, the company now expects both consolidated and Core Illumina revenue growth of approximately 36%. The company now expects GAAP earnings per diluted share of $4.41 to $4.51, and non-GAAP earnings per diluted share of $5.50 to $5.60, which includes dilution from GRAIL non-GAAP operating loss of approximately $1.00 and incremental dilution from the 9.8 million shares issued to fund the GRAIL acquisition of $0.15.

Conference call information

The conference call will begin at 2:00 p.m. Pacific Time (5:00 p.m. Eastern Time) on Thursday, November 4, 2021. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (844) 200-6205 or 1 (929) 526-1599 outside North America, both using conference ID 808648.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

Statement regarding use of non-GAAP financial measures

The company reports non-GAAP results for diluted net income per share, net income, gross margins, operating expenses, including research and development expenses and selling general and administrative expenses, operating income (loss), operating margins, gross profit, other income, and free cash flow (on a consolidated and as applicable, segment basis for our Core Illumina and GRAIL segments) in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The company’s financial measures under GAAP include substantial charges such as amortization of acquired intangible assets, non-cash interest expense associated with the company’s convertible debt instruments that may be settled in cash, and others that are listed in the itemized reconciliations between GAAP and non-GAAP financial measures included in this press release. Management has excluded the effects of these items in non-GAAP measures to assist investors in analyzing and assessing past and future operating performance, including in the non-GAAP measures relating to our Core Illumina and GRAIL segments. Additionally, non-GAAP net income and diluted earnings per share are key components of the financial metrics utilized by the company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

The company encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP information and the reconciliation between these presentations, to more fully understand its business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

On November 4, 2021 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of innovative therapies designed to treat or reverse disease, reported a corporate update and financial results for the three and nine months ended September 30, 2021 (Press release, Caladrius Biosciences, NOV 4, 2021, View Source [SID1234594425]).

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"Caladrius continued to advance and optimize its development programs in the third quarter despite the ongoing challenges to clinical development posed by the global COVID-19 pandemic. A cash position of approximately $100 million, coupled with continued prudent cash management, enabled the Company to focus on refining and executing its development plans while identifying and evaluating attractive strategic corporate development opportunities," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "Additionally, Caladrius strengthened and further diversified its operations and management team with the addition of several highly qualified and experienced personnel, including the appointment of veteran drug developer, Kristen K. Buck, M.D., as Executive VP of R&D and Chief Medical Officer. Dr. Buck will play a key role in defining, optimizing, and implementing development strategies that maximize the probability of clinical and commercial success of existing programs. She will also contribute to one of our corporate priorities of adding promising new assets to our development portfolio by leading our technical evaluation efforts."

Product Development and Financing Highlights

HONEDRA (CLBS12) for the treatment of critical limb ischemia ("CLI")

With respect to HONEDRA, the Company’s SAKIGAKE-designated product candidate for the treatment of CLI and Buerger’s disease in Japan, the headwinds to enrollment in the ongoing registration-eligible trial, as discussed in prior quarters, have persisted. The multiple states of emergency declared by the Japanese government over the past 18 months due to the COVID-19 pandemic have made incremental enrollment exceedingly challenging, prompting Caladrius to consider alternate approaches to achieving development success. Since the trial continues to demonstrate positive trends in both safety and efficacy, the key criteria for consideration of conditional approval in Japan under the SAKIGAKE designation, the Company has decided to suspend enrollment activities in favor of focusing efforts in Japan on securing a partner to complete study enrollment with four remaining patients, if necessary, and/or to explore the possibility of submitting the existing data to the Japanese Regulatory Authorities under the SAKIGAKE designation. This decision is motivated by the Company’s desire to minimize additional operational and financial burden caused by enrollment delays and the lack of visibility on time to completion.

XOWNA (CLBS16) for the treatment of coronary microvascular dysfunction ("CMD")

XOWNA is an experimental regenerative therapy for the treatment of CMD. It was the subject of a recently reported positive Phase 2a study (the "ESCaPE-CMD trial") and is currently being evaluated in a U.S. Phase 2b study (the "FREEDOM Trial"). The FREEDOM Trial is a double-blind, randomized, placebo-controlled trial designed to assess the efficacy and safety of delivering autologous CD34+ cells (XOWNA) to subjects with CMD and without obstructive coronary artery disease. Early enrollment in the FREEDOM Trial proceeded as planned with the first patient treated in January 2021, however, the impact of the COVID-19 pandemic in the U.S. since then has contributed to a general slowing of enrollment. Caladrius has taken steps to accelerate enrollment by expanding the number of participating investigational sites and modifying the study protocol. These protocol amendments were implemented in the latter part of the quarter after agreement with the U.S. Food and Drug Administration, and we will assess their impact on enrollment in the coming months, at which time the Company expects to be in a better position to provide an informed estimate for enrollment feasibility and completion. Final data from the study are expected approximately 6 months after last patient/last visit in the study.

CLBS201 for the treatment of diabetic kidney disease ("DKD")

Progressive kidney failure is associated with attrition of the microcirculation of the kidney. Pre-clinical studies in kidney disease and injury models have demonstrated that protection or replenishment of the microcirculation results in improved kidney function. Based on these observations, the Company plans to initiate a Phase 1, open-label, proof-of-concept trial evaluating CLBS201, a CD34+ regenerative cell therapy investigational product for intra-renal artery administration, in patients with DKD. Although still pre-dialysis, these patients exhibit rapidly progressing stage 3b disease. The protocol, pending final approval from the Institutional Review Board, will be a staggered, sequentially dosed cohort of six patients overseen by an independent Data Safety Monitoring Board with the objective of determining the tolerance of intra-renal cell therapy injection in DKD patients and the ability of CLBS201 to regenerate kidney function. A key read-out of data will occur at the 6-month follow-up visit for all patients. The Company projects enrollment of this study to begin in the first quarter of 2022 with data from all subjects expected by the first quarter of 2023.

Third Quarter 2021 Financial Summary

Research and development expenses were approximately $4.1 million for the three months ended September 30, 2021, compared to $3.0 million for the three months ended September 30, 2020, representing an increase of 36%. Research and development in both periods focused on the advancement of our ischemic repair platform and related to:

Expenses associated with efforts to continue execution and acceleration of enrollment of the FREEDOM Trial;

Expenses associated with the planning and preparation of an IND and Phase 1 proof-of-concept protocol for CLBS201 as a treatment for DKD; and

Ongoing expenses for HONEDRA in CLI and Buerger’s disease in Japan associated with maintenance of manufacturing facility and personnel qualification as well as Contract Research Organization engagement despite no subject treatment execution due to the COVID-19 imposed state of emergency in Japan.
General and administrative expenses were approximately $2.8 million for the three months ended September 30, 2021, compared to $2.3 million for the three months ended September 30, 2020, representing an increase of 22%. This increase was primarily due to an increase in directors and officers insurance premiums and strategic consulting expenses.

Overall, net losses were $6.9 million for the three months ended September 30, 2021, compared to $5.3 million for the three months ended September 30, 2020.

Balance Sheet Highlights

As of September 30, 2021, we had cash, cash equivalents and marketable securities of approximately $100.1 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations for the next several years. Based on this favorable cash position, the Company continues its concerted efforts to identify and acquire additional development assets to diversify our portfolio of product candidates and to enhance the opportunity for near-term shareholder value creation.

Conference Call

Caladrius will hold a live conference call today, November 4, 2021, at 4:30 p.m. (ET) to discuss financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. A live webcast of the call will also be available under the Investors & News section of the Caladrius website, View Source, and will be available for replay for 90 days after the conclusion of the call.

Please dial-in 10 minutes before the conference call starts.

For those unable to participate on the live conference call, an audio replay will be available that day starting at 7:30 p.m. (ET) until November 11, 2021, by dialing 855-859-2056 (U.S. Toll-Free) or 404-537-3406 (International) and by entering the replay passcode: 8378269.

On November 4, 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 third quarter ended September 30, 2021 (Press release, Acceleron Pharma, NOV 4, 2021, View Source [SID1234594424]).

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Corporate Highlights

On September 30, Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Acceleron Pharma Inc. announced that the companies entered into a definitive agreement under which Merck, through a subsidiary, will acquire Acceleron for $180 per share in cash for an approximate total equity value of $11.5 billion.
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 pulmonary arterial hypertension (PAH), sotatercept reversed pulmonary arterial wall and right ventricular remodeling that are hallmarks of the disease.

Enrollment remains ongoing in the STELLAR Phase 3 trial of sotatercept in patients with PAH.
In August, Acceleron initiated the HYPERION Phase 3 trial of sotatercept in newly diagnosed patients with intermediate- and high-risk PAH.
The Company recently initiated the ZENITH Phase 3 trial of sotatercept in patients with WHO functional class III or IV PAH at high risk of mortality.
Study start up activities are underway for the CADENCE Phase 2 trial of sotatercept in patients with pulmonary hypertension due to left heart disease.
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.

In October, Acceleron initiated a Phase 1b study to evaluate the activity of ACE-1334 in patients with SSc-ILD.
Hematology

REBLOZYL (luspatercept-aamt):

REBLOZYL is the first and only erythroid maturation agent approved in the United States, Europe, and Canada 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 $32.0 million in royalty revenue from approximately $160 million in net sales of REBLOZYL in the third quarter of 2021. This compares with approximately $25.6 million in royalty revenue from approximately $128 million in net sales of REBLOZYL in the second quarter of 2021.
Net sales of REBLOZYL for the third quarter include approximately $20 million to $25 million of sales from an inventory build due to the transition to wholesaler distribution and approximately $13.5 million of net sales outside of the United States.
In September, the United States Food and Drug Administration granted luspatercept Orphan Drug designation for the treatment of anemia in patients with alpha-thalassemia.
Enrollment remains ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS.
Enrollment remains ongoing in the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
Three clinical abstracts on REBLOZYL have been accepted for presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition scheduled for December 11 to 14, 2021.
Financial Results

Cash Position – Cash, cash equivalents and investments as of September 30, 2021 were $652.5 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 third quarter of 2021 was $34.2 million, which includes $2.2 million of cost share revenue and $32.0 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 $59.7 million for the third quarter of 2021. Non-GAAP R&D expenses were $53.1 million for the third quarter of 2021, excluding $5.6 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $45.1 million for the third quarter of 2021. Non-GAAP SG&A expenses were $39.1 million for the third quarter of 2021, excluding $5.8 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 third quarter of 2021 was $70.5 million, or $1.16 per share. Non-GAAP adjusted net loss for the third quarter was $57.9 million, or $0.95 per share, excluding $11.4 million and $1.2 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 not be holding a quarterly earnings call or webcast.

On November 4, 2021 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in viral and liver diseases, reported recent business progress and financial results for the third quarter ended, September 30, 2021 (Press release, Aligos Therapeutics, NOV 4, 2021, View Source [SID1234594423]).

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"The last quarter has been busy and productive for Aligos," said Lawrence Blatt, PhD, MBA, Chairman and CEO of Aligos. "We now have three of our four drug candidates targeting chronic hepatitis B, each of which has a distinct additive or synergistic mechanism of action, dosing in the clinic. Additionally, enrollment of CHB patients has improved, and our planned safety and viral kinetic data readout for the first three cohorts of STOPS remains on track for the first half of 2022. In parallel to this effort, the Aligos team was able to successfully file the CTA for our first drug candidate for the treatment of NASH, ALG-055009. The progress made by our team over the last quarter positions us well to be able to deliver key data for multiple programs in 2022."

Recent Business Progress
Aligos’ portfolio of drug candidates has continued to advance during the quarter with the following accomplishments achieved:

S-Antigen Transport-Inhibiting Oligonucleotide Polymers (STOPS; ALG-010133)
Our Phase 1b dose range finding trial (NCT04485663) evaluating subjects with CHB is ongoing. Enrollment in the second cohort, evaluating the 200 mg dose level, was completed and enrollment in the third cohort, evaluating a 400 mg dose level, is now underway. We remain on track to unblind and share topline safety and viral kinetic data, including Hepatitis B S-antigen (HBsAg) data, from the first three cohorts in the first half of 2022.
Capsid Assembly Modulator (CAM; ALG-000184)
Our Phase 1b dose range finding trial (NCT04536337) evaluating CHB subjects is ongoing. Enrollment of hepatitis B E-antigen (HBeAg) negative subjects into 50 and 100 mg dose cohorts is now complete. Enrollment of a 10 mg dose cohort is underway to determine the minimum efficacious dose. A cohort of HBeAg-positive CHB patients receiving a 100 mg dose for 28 days has also been fully enrolled in China.
A poster describing the safety, PK, and antiviral activity of the 50 and 100 mg doses of ALG-000184 given for 28 days in HBeAg-negative CHB subjects will be presented at The Liver Meeting (American Association for the Study of Liver Disease) later this month.
Antisense Oligonucleotide (ASO; ALG-020572)
Our Phase 1a/b multi-part study (NCT05001022) evaluating ALG-020572 in healthy volunteers and CHB subjects has recently commenced. Enrollment of healthy volunteers in the single ascending dose (SAD) portion of the study was initiated in October 2021.
Nonalcoholic Steatohepatitis (NASH; ALG-055009, a thyroid hormone receptor beta (THR-B) agonist)
The CTA for ALG-055009 was filed in France to enable a SAD and multiple ascending dose (MAD) study in healthy volunteers and hyperlipidemic subjects, respectively.
Study startup activities remain on track with dosing in healthy volunteers expected to begin in the fourth quarter of 2021.
Aligos has a broad CHB portfolio that targets different clinically validated mechanisms of action in the hepatitis B virus life cycle. The portfolio includes a STOPS molecule, ALG-000184, a class II CAM, ALG-010133, ALG-020572, an ASO, and ALG-020755, an siRNA drug candidate. The properties of these candidates indicate that their use in combination could yield potentially best-in-class treatment regimens that may achieve higher rates of functional cure than current standard of care. For each of these drug candidates, Aligos plans to initially establish proof of concept as monotherapy in Phase 1 dose range finding trials before evaluating them in combination in subsequent trials.

Financial Results for the Third Quarter 2021
Net losses for the three months ended September 30, 2021 were $33.1 million or basic and diluted net loss per common share of $(0.78) compared to net losses of $33.3 million or basic and diluted net loss per common share of $(11.00) for the three months ended September 30, 2020.

Research and development (R&D) expenses for the three months ended September 30, 2021, were $28.1 million compared with $17.3 million for the same period of 2020. The increase in R&D expenses for this comparative period is primarily attributable to increased expenses related to the Company’s continued development and manufacturing of ALG-010133, ALG-000184 and ALG-020572 clinical trial activities, as well as increases in salaries and employee-related expenses and preclinical programs. Total R&D stock-based compensation expense incurred for the three months ended September 30, 2021, was $1.9 million compared with $0.3 million for the same period for 2020.

General and administrative (G&A) expenses for the three months ended September 30, 2021, were $6.5 million compared with $4.2 million for the same period of 2020. The increase in G&A expenses for this comparative period is primarily attributable to higher employee-related costs associated with the growth of the Company’s operations and additional professional, legal and consulting services related to being a public company. Total G&A stock-based compensation expense incurred for the three months ended September 30, 2021, was $1.7 million compared with $0.7 million for the same period for 2020.

Cash, cash equivalents and investments totaled $242.7 million as of September 30, 2021 compared with $243.5 million as of December 31, 2020.