Terns Pharmaceuticals Reports Third Quarter 2021 Financial Results and Corporate Highlights

On November 15, 2021 Terns Pharmaceuticals, Inc. ("Terns" or the "Company") (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule single-agent and combination therapy candidates to address serious diseases such as non-alcoholic steatohepatitis (NASH), reported financial results for the third quarter ended September 30, 2021 and corporate highlights (Press release, Terns Pharmaceuticals, NOV 15, 2021, View Source [SID1234595605]).

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"Recent positive clinical data for TERN-101 and TERN-501 presented at AASLD validate part one of Terns’ strategy, which is demonstrating monotherapy proof of concept. Notably, TERN-501 produced significant and dose-dependent changes in sex hormone binding globulin (SHBG), a marker of target engagement linked to NASH efficacy, suggesting that it has potential to be a best-in-class THR-β agonist. We look forward to the top-line data readout from the AVIATION Trial of TERN-201 in the first quarter of 2022, which could mark our third clinical candidate with promising monotherapy data," said Senthil Sundaram, chief executive officer at Terns. "I am proud of the quality of execution at Terns and the momentum with which we are advancing our pipeline towards the second part of our strategy: achieving compelling and differentiated efficacy results in studies involving multiple clinically validated mechanisms of action. With this goal in mind, we are excited to move towards the planned initiation in the first half of 2022 of Terns’ first clinical trial in NASH patients including both monotherapy and combination arms of TERN-101 and TERN-501."

Recent Developments and Anticipated Milestones

TERN-201: Vascular adhesion protein-1 (VAP-1) inhibitor

Fully enrolled Part 1 of Phase 1b AVIATION Trial in NASH in September 2021
Top-line data from AVIATION Part 1 expected in 1Q 2022, including:
Key efficacy readout in corrected T1 (cT1) levels, an imaging marker of liver inflammation and fibrosis linked to clinical outcomes
Safety, tolerability and plasma VAP-1 activity
TERN-501: Thyroid hormone receptor-beta (THR-β) agonist

Reported positive top-line data from Phase 1 proof of concept clinical trial in November 2021, including a presentation at AASLD 2021 demonstrating:
Significant, dose-dependent effects on SHBG, a key pharmacodynamic marker of THR-β engagement linked to NASH histologic efficacy
TERN-501 was generally safe and well-tolerated with a predictable pharmacokinetic (PK) profile with low variability
Significant, dose-dependent reductions in atherogenic lipids including low-density lipoprotein (LDL) cholesterol and apolipoprotein B (Apo-B) in all TERN-501 dose groups compared to placebo
The safety, PK and PD results support continued development of TERN-501 and indicate that it is well-suited for co-formulation with other small molecule NASH agents as an oral, once-daily fixed dose combination

Data support plans to initiate in 1H 2022 Terns’ first NASH trial of a THR-β agonist (TERN-501) alone and in combination with a farnesoid X receptor (FXR) agonist (TERN-101)
TERN-101: Liver-distributed FXR agonist

Presented positive data from Phase 2a LIFT clinical trial in NASH at AASLD’s The Liver Meeting Digital Experience 2021, which demonstrated:
cT1 declined significantly as early as Week 6 with persistent decreases through Week 12 in all TERN-101 groups compared to placebo, with cT1 changes at Week 6 strongly correlated with changes at Week 12
TERN-101 treatment led to study population shifts to cT1 categories associated with lower risk of clinical events in chronic liver disease patients
In 10 and 15 mg groups of TERN-101, numerical reductions in alanine aminotransferase (ALT) and MRI protein density fat fraction (MRI-PDFF) were observed, with significant reductions in gamma glutamyl transferase (GGT) in all dose groups
TERN-101 was overall safe and well-tolerated at all doses studied with no discontinuations due to adverse events, including pruritus
In 5 and 10 mg groups of TERN-101, no differences from placebo in LDL cholesterol and high-density lipoprotein (HDL) cholesterol percentage change from baseline to Week 12 were observed
GLP1-R: Oral, small-molecule glucagon-like peptide-1 (GLP1) receptor agonist

Driven by computational interaction mapping, chemical synthesis and in vitro characterization of approximately 100 GLP-1R agonist compounds; lead candidates currently undergoing higher species in vivo profiling
Synthetic GLP-1 peptides have been approved for indication such as diabetes and obesity, which are conditions often accompanying NASH
Development candidate anticipated to be designated as TERN-601 in 4Q 2021
Key Appointments

Ann E. Taylor, M.D. joined the Board of Directors in September 2021, bringing more than 35 years of experience in drug development, having served most recently as chief medical officer of AstraZeneca plc
Pamela Danagher joined Terns as vice president and head of regulatory affairs in August 2021, bringing more than 20 years of experience in the pharmaceutical and biotechnology sectors
Third Quarter Financial Results

Cash Position: As of September 30, 2021, cash, cash equivalents and marketable securities were $177.2 million as compared with $74.9 million as of December 31, 2020. Based on its current operating plan, Terns expects these funds will be sufficient to support its planned operating expenses into 2024.
Research and Development (R&D) Expenses: R&D expenses were $7.2 million for the quarter ended September 30, 2021, as compared with $5.4 million for the quarter ended September 30, 2020
General and Administrative (G&A) Expenses: G&A expenses were $4.7 million for the quarter ended September 30, 2021, as compared with $3.3 million for the quarter ended September 30, 2020
Net Loss: Net loss was $11.8 million for the quarter ended September 30, 2021, as compared with $11.6 million for the quarter ended September 30, 2020

Fortress Biotech Reports Record Third Quarter 2021 Financial Results and Recent Corporate Highlights

On November 15, 2021 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, reported financial results and recent corporate highlights for the third quarter ended September 30, 2021 (Press release, Fortress Biotech, NOV 15, 2021, View Source [SID1234595604]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman and Chief Executive Officer, said, "Fortress and our partner companies continue to achieve significant clinical and corporate milestones as we develop new treatment options for patients in need and create long-term value for our shareholders. We attained a new quarterly record for net revenue, $21.1 million, which is an increase of 123% year-over-year. Journey Medical Corporation ("Journey"), our partner company, launched its initial public offering ("IPO") of 3,520,000 shares at $10.00 per share for gross proceeds of $35.2 million. Additionally, AstraZeneca’s Alexion acquired Caelum Biosciences, Inc. ("Caelum") a company founded by Fortress, for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress.1 The agreement also provides for additional contingent payments to Caelum stockholders totaling up to $350 million, payable upon the achievement of regulatory and commercial milestones. Fortress is eligible to receive 42.4% of all proceeds from the transaction, totaling up to approximately $212 million. Our CAR T cell therapy portfolio continues to advance, with a $2 million grant awarded by the National Cancer Institute ("NCI") to partially fund our Mustang Bio, Inc. ("Mustang Bio")-sponsored Phase 1/2 trial of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") or chronic lymphocytic leukemia ("CLL"). Finally, we announced positive data from two completed pivotal studies in patients with Menkes disease treated with CUTX-101 at the 2021 American Academy of Pediatrics National Conference & Exhibition."

Dr. Rosenwald continued, "Looking ahead, we anticipate continued regulatory and clinical progress including initiating the rolling submission of the new drug application ("NDA") for CUTX-101 for the treatment of Menkes disease to the U.S. Food and Drug Administration ("FDA") later this quarter and reporting top-line results from the registration-enabling study of cosibelimab in metastatic cutaneous squamous cell carcinoma around year-end. We also look forward to the upcoming data presentations of MB-106 and AL amyloidosis treatment CAEL-101 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting ("ASH2021")."

Recent Corporate Highlights2:

Marketed Dermatology Products and Product Candidates

Journey, our partner company, markets seven prescription dermatology products.
Our products generated net revenues of $19.6 million for the third quarter of 2021, compared to net revenues of $9.4 million for the third quarter of 2020.
In July 2021, Journey completed its final closings under the Cumulative Convertible Class A Preferred Stock Offering (the "Preferred Offering"), issuing an aggregate of 758,680 preferred shares at a price of $25.00 per share, raising approximately $19.0 million in gross proceeds, and after deducting commissions, fees and expenses, receiving approximately $17.0 million in net proceeds. These shares converted into Journey common stock upon the IPO.
On November 12, 2021, Journey launched its IPO of 3,520,000 shares of its common stock at $10.00 per share, totaling $35.2 million in gross proceeds, and, after deducting underwriting discounts, and estimated fees and expenses, is expected to receive approximately $31.4 million in net proceeds. Journey Medical’s common stock began trading on the Nasdaq Capital Market on November 12, 2021, under the ticker symbol "DERM." The IPO is expected to close on November 16, 2021, subject to customary closing conditions.
Journey intends to initiate the Phase 3 clinical program for DFD-29 for the treatment of rosacea in the fourth quarter of 2021.
Journey intends to launch one additional prescription product in the first half of 2022.
CUTX-101 (Copper Histidinate for Menkes disease)

We expect to initiate the rolling submission of the NDA, under its Breakthrough Designation, for CUTX-101 to the FDA this quarter.
In October 2021, we announced positive results from an efficacy and safety analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101, copper histidinate (CuHis). These data were presented as a virtual poster at the 2021 American Academy of Pediatrics National Conference & Exhibition.
CUTX-101 was sourced by Fortress and is currently in development at our partner company, Cyprium Therapeutics, Inc.
CAEL-101 (Light Chain Fibril-reactive Monoclonal Antibody for AL Amyloidosis)

On October 5, 2021, AstraZeneca’s Alexion acquired Caelum, a company founded by Fortress, for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress, net of the ten percent, 24-month escrow holdback amount and other miscellaneous transaction expenses. The agreement also provides for additional potential payments to Caelum shareholders totaling up to $350 million, payable upon the achievement of regulatory and commercial milestones. Fortress is eligible to receive 42.4% of all proceeds of the transaction, totaling up to approximately $212 million.
CAEL-101 data were selected for presentation at ASH (Free ASH Whitepaper)2021 scheduled to take place in December of 2021. Abstracts can be viewed online through the ASH (Free ASH Whitepaper)2021 website here.
There are two ongoing Phase 3 studies of CAEL-101 for AL amyloidosis.
CAEL-101 was sourced by Fortress in 2017 and was developed by Caelum until Caelum was acquired by AstraZeneca on October 5, 2021.
Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

The registration-enabling study in metastatic cutaneous squamous cell carcinoma is fully enrolled, and we are on track to report top-line results around year-end 2021. Upon a successful outcome, Checkpoint Therapeutics, Inc. ("Checkpoint") intends to submit a Biologics License Application ("BLA") for cosibelimab in 2022, followed shortly thereafter by a Marketing Authorization Application submission in Europe. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, we believe cosibelimab has the potential to be a market disruptive product in the $25 billion and growing PD-(L)1 class.
A Phase 3 registration-enabling trial is planned to begin in first-line metastatic non-small cell lung cancer ("NSCLC") in the fourth quarter of 2021.
Cosibelimab was sourced by Fortress and is currently in development at our partner company, Checkpoint.
MB-106 (CD20-targeted CAR T Cell Therapy)

In November 2021, we announced that MB-106 data were selected for presentation at ASH (Free ASH Whitepaper)2021 scheduled to take place in December 2021. Dr. Mazyar Shadman of Fred Hutchinson Cancer Research Center will present updated interim data from the ongoing Phase 1/2 clinical trial for NHL and CLL. A copy of the abstract can be viewed online through the ASH (Free ASH Whitepaper)2021 website here.
Also in November 2021, we announced that Mustang Bio was awarded a grant of approximately $2 million from NCI of the National Institutes of Health. This two-year award will partially fund the Mustang-sponsored multicenter trial to assess the safety, tolerability and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL or CLL.
MB-106 was sourced by Fortress and is currently in development at our partner company, Mustang Bio.
MB-101 (IL13Rα2-targeted CAR T Cell Therapy)

In October 2021, Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory and The Heritage Provider Network Professor in Immunotherapy at City of Hope, presented updated Phase 1 clinical data regarding MB-101 (IL13Rα2‐targeted CAR T cells) for the treatment of glioblastoma at two scientific conferences, the First Annual Conference on CNS Clinical Trials, co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Special Conference: Brain Cancer.
MB-101 was sourced by Fortress and is currently in development at our partner company, Mustang Bio.
Novel CAR T Technology

In August 2021, we announced an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.
The novel CAR T technology was sourced by Fortress and is currently in development at our partner company, Mustang Bio.
MB-107 and MB-207 (Lentiviral Gene Therapies for X-linked Severe Combined Immunodeficiency ("XSCID"))

In August 2021, we announced that the European Medicines Agency ("EMA") granted Priority Medicines ("PRIME") designation to MB-107, a lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants, also known as bubble boy disease.
MB-107 and MB-207 were sourced by Fortress and are currently in development at our partner company, Mustang Bio.
Ex Vivo Lentiviral Gene Therapy for RAG1 Severe Combined Immunodeficiency ("RAG1-SCID")

Last week, we announced the execution of an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
The ex vivo lentiviral gene therapy was sourced by Fortress and is currently in development at our partner company, Mustang Bio.
Financial Results:

To assist our stockholders in understanding our company, we have prepared non-GAAP financial results for the three months ended September 30, 2021 and 2020. These results exclude the operations of our three partner companies that were public at September 30, 2021: Avenue, Checkpoint and Mustang Bio. The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which comprise our commercial-stage business, our privately held development-stage entities, as well as our business development and finance functions.

As of September 30, 2021, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $254.4 million, compared to $235.0 million as of December 31, 2020, an increase of $19.4 million year-to-date.
On a GAAP basis, Fortress’ net revenue totaled $21.1 million for the third quarter of 2021, which included $19.6 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $9.5 million for the third quarter of 2020, which included $9.4 million in net revenue generated from our marketed dermatology products.
On a GAAP basis, consolidated research and development expenses, including license acquisitions of $0.7 million, were $28.1 million for the third quarter of 2021, compared to consolidated research and development expenses, including license acquisitions of $0.5 million, totaling $13.8 million for the third quarter of 2020. On a non-GAAP basis, research and development expenses including license acquisitions were $3.8 million for the third quarter of 2021, compared to research and development expenses including license acquisitions totaling $2.8 million for third quarter of 2020.
On a GAAP basis, consolidated selling, general and administrative expenses were $22.2 million for the third quarter of 2021, compared to $15.4 million for the third quarter of 2020. On a non-GAAP basis, consolidated selling, general and administrative expenses were $17.6 million, of which $10.8 million is attributed to Journey, for the third quarter of 2021, compared to $11.6 million, of which $5.8 million is attributed to Journey, for the third quarter of 2020.
On a GAAP basis, consolidated net loss attributable to common stockholders was $(20.8) million, or $(0.26) per share, for the third quarter of 2021, compared to consolidated net loss attributable to common stockholders of $(15.5) million, or $(0.20) per share for the third quarter of 2020.
Fortress’ non-GAAP income attributable to common stockholders was $43.7 million, which includes the partial realization of Fortress’ investment in Caelum, or $0.54 per share, for the third quarter of 2021, compared to Fortress’ non-GAAP loss attributable to common stockholders of $(5.2) million, or $(0.07) per share, for the third quarter of 2020.
The tables below have more information.
Use of Non-GAAP Measures:

In addition to the GAAP financial measures as presented in our Form 10-Q that will be filed with the Securities and Exchange Commission ("SEC") on November 15, 2021, the Company has, in this press release, included certain non-GAAP measurements. The non-GAAP net income (loss) attributable to common stockholders is defined by the Company as GAAP net income (loss) attributable to common stockholders, less net losses attributable to common stockholders from our public partner companies Avenue, Checkpoint and Mustang Bio. In addition, the Company has also provided a Fortress non-GAAP loss attributable to common stockholders which is a modified EBITDA calculation that starts with the non-GAAP income (loss) attributable to common stockholders and removes stock-based compensation expense, non-cash interest expense, amortization of licenses and debt discount, changes in fair values of investment, changes in fair value of derivative liability, Qbrexza inventory step-up and depreciation expense. The Company included the partial realization of gain in connection with the exercise of the Caelum option.

Management believes use of these non-GAAP measures provide meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key measures used by management in its financial and operational decision-making, (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s results. However, non-GAAP income (loss) attributable to common stockholders and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the manner in which they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors.

Avenue net loss from their external SEC report for the three months ended September 30, 2021 and 2020 of $0.9 million and $1.0 million, respectively, net of non-controlling interest of $0.7 million and $0.8 million, respectively. Avenue net loss from their external SEC report for the nine months ended September 30, 2021 and 2020 of $2.8 million and $4.2 million, respectively, net of non-controlling interest of $2.2 million and $3.2 million, respectively.

Checkpoint net loss from their external SEC report of $11.3 million net of non-controlling interest of $9.0 million, MSA fee to Fortress of $0.1 million and financing fee to Fortress of approximately $39,000 for the quarter ended September 30, 2021; and net loss of $4.9 million net of non-controlling interest of $3.2 million, less MSA fee to Fortress of $0.1 million and financing fee to Fortress of $0.7 million for the quarter ended September 30, 2020. Checkpoint net loss from their external SEC report of $26.9 million net of non-controlling interest of $20.6 million, MSA fee to Fortress of $0.4 million and financing fee to Fortress of $0.9 million for the nine months ended September 30, 2021; and net loss of $12.9 million net of non-controlling interest of $9.0 million, less MSA fee to Fortress of $0.4 million and financing fee to Fortress of $0.8 million for the nine months ended September 30, 2020.

Mustang net loss from their external SEC report of $17.0 million net of non-controlling interest of $13.7 million, MSA fee to Fortress of $0.1 million and financing fee to Fortress of $0.1 million for the quarter ended September 30, 2021; and net loss of $13.0 million net of non-controlling interest of $9.3 million, MSA fee to Fortress of $0.1 million and financing fee to Fortress of $0.4 million for the quarter ended September 30, 2020. Mustang net loss from their external SEC report of $46.3 million net of non-controlling interest of $35.8 million, MSA fee to Fortress of $0.4 million and financing fee to Fortress of $1.7 million for the nine months ended September 30, 2021; and net loss of $39.4 million net of non-controlling interest of $27.0 million, MSA fee to Fortress of $0.4 million and financing fee to Fortress of $1.6 million for the nine months ended September 30, 2020.

Increase in fair value of investment in Caelum Biosciences for the quarter and nine months ended September 30, 2021.

Increase in fair value of derivative liabilities of Journey Medical Corporation for the quarter and nine months ended September 30, 2021.

Step-up related to FV of Qbrexza inventory recorded in COGS for the quarter and nine months ended September 30, 2021.

Partial realization of gain in connection with the exercise of the Caelum option. The Company backed out of Fortress non-GAAP income (loss) attributable to stockholders until actual realization of gain due to exercise of option.
Reconciliation to non-GAAP research and development and selling, general and administrative costs:

Includes Research and development expense and Research and development – licenses acquired expense for the quarter and nine months ended September 30, 2021 and 2020, respectively.

Excludes $0.1 million and $0.1 million of Fortress MSA expense for the quarter ended September 30, 2021 and 2020, respectively, and $0.2 million and $0.2 million for the nine months ended September 30, 2021 and 2020, respectively.

Excludes $0.1 million of Fortress MSA expense and approximately 39,000 of Fortress financing fee for the quarter ended September 30, 2021; and $0.1 million of Fortress MSA expense and $0.7 million Fortress financing fee for the quarter ended September 30, 2020. Excludes $0.4 million of Fortress MSA expense and $0.9 million Fortress financing fee for the nine months ended September 30, 2021; and $0.4 million of Fortress MSA expense and $0.8 million Fortress financing fee for the nine months ended September 30, 2020.

Excludes $0.1 million of Fortress MSA expense and $0.1 million Fortress financing fee for the quarter ended September 30, 2021; and $0.1 million of Fortress MSA expense and $0.4 million Fortress financing fee for the quarter ended September 30, 2020. Excludes $0.2 million of Fortress MSA expense and $1.7 million Fortress financing fee for the nine months ended September 30, 2021; and $0.2 million of Fortress MSA expense and $1.6 million Fortress financing fee for the nine months ended September 30, 2020.

Athersys Reports Third Quarter 2021 Results and Provides Corporate Update

On November 15, 2021 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended September 30, 2021 and provided a corporate update (Press release, Athersys, NOV 15, 2021, View Source [SID1234595602]).

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"The third quarter brought several important milestones in the development and potential commercialization of MultiStem cell therapy," stated Mr. William (B.J.) Lehmann, Jr., Interim Chief Executive Officer of Athersys. "First, our partner Healios announced positive topline data from its ONE-BRIDGE acute respiratory distress syndrome (ARDS) study, giving us further confidence in the potential of our cell therapy for the treatment of ARDS and critical care more generally. Second, enrollment has been completed for the Japan TREASURE study of MultiStem treatment for ischemic stroke patients, starting the countdown to the release of pivotal study data in this area. And third, we realigned our collaboration with Healios to put us in the best position to prepare together for commercialization in Japan.

"Additionally, we demonstrated steady progress in our MASTERS-2 study as well as in the steps necessary to establish commercial manufacturing capability," added Mr. Lehmann. "We look forward to important data readouts ahead of us, our partner’s progress in Japan in the application process for marketing approval, and further development of our commercial capabilities."

Highlights of the third quarter of 2021 and recent events include:

Positive topline results from the ONE-BRIDGE study announced by HEALIOS K.K. (Healios). The ONE-BRIDGE study evaluated the Company’s proprietary cell therapy, MultiStem (HLCM051; invimestrocel), for the treatment of pneumonia-induced and COVID-induced acute respiratory distress syndrome (ARDS) in Japan. Data from the study are consistent with the Company’s MUST-ARDS study results, and analyses of the pooled data of both studies reflect positive benefit trends;
Completion of TREASURE study enrollment announced by Healios. The TREASURE study is evaluating MultiStem for the treatment of ischemic stroke patients in Japan. Based on the advice from the Pharmaceuticals and Medical Devices Agency (PMDA), in order to avoid any potential bias to the 365-day data (and related secondary endpoints) that could result from unblinding and disclosure of 90-day data (primary endpoint) the decision was made that the 90-day unblinding, data analysis and release would take place after the 365-day data is locked. This will follow the last patient’s one-year follow up visit which Healios expects to occur in March of 2022;
Improved our collaboration with Healios and optimized the structure of the relationship to help drive commercial success for MultiStem therapy in Japan;
Steadily progressed our MASTERS-2 study, including the initiation of new sites, including our first sites outside of the U.S.;
Published additional important research findings covering Multipotent Adult Progenitor Cells (MAPC) and Treg biology and MAPC mechanisms of action, with references available on the Company’s website;
Achieved a "Prime" corporate rating from Institutional Shareholder Services Environmental, Social and Governance (ISS ESG) for fulfilling the requirements regarding sustainability performance in the Pharmaceuticals & Biotechnology sector;
Recognized net loss of $16.2 million, or $0.07 net loss per share, for the quarter ended September 30, 2021; and
Ended the third quarter with $49.7 million of cash and cash equivalents.
Third Quarter Results

Revenues increased to $4.8 million for the three months ended September 30, 2021 compared to $0.1 million for the three months ended September 30, 2020. Our collaboration revenues currently fluctuate from period to period based on the delivery of goods and services under our arrangement with Healios.

Research and development expenses decreased to $17.2 million for the three months ended September 30, 2021 from $18.5 million for the comparable period in 2020. The $1.3 million decrease is associated with decreases in clinical trial and manufacturing process development costs of $2.0 million and internal research supplies of $0.6 million. These decreases were partially offset by increases in personnel costs of $0.5 million, facilities costs of $0.4 million and outside service costs of $0.4 million. Our clinical development, clinical manufacturing and manufacturing process development expenses vary over time based on the timing and stage of clinical trials underway, manufacturing campaigns for clinical trials and manufacturing process development projects.

General and administrative expenses decreased to $3.6 million for the three months ended September 30, 2021 from $3.7 million for the comparable period in 2020.

Net loss for the third quarter of 2021 was $16.2 million compared to a net loss of $22.5 million in the third quarter of 2020. The difference primarily results from the above variances.

During the nine months ended September 30, 2021, net cash used in operating activities was $56.9 million compared to $44.5 million in the nine months ended September 30, 2020. At September 30, 2021, we had $49.7 million in cash and cash equivalents, compared to $51.5 million at December 31, 2020.

Conference Call

We encourage shareholders to listen using the webcast link above. If you would like to dial in using the phone to ask a question, please register for the conference call ahead of time using the call registration link above. Once registered, you will receive the toll-free number, a direct entry passcode and a registrant ID.

A replay of the event will be available on the webcast link at www.athersys.com under the investors’ section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening approximately three hours after the completion of the call until 11:59 PM EST on November 22, 2021, by dialing (800) 585-8367 or (416) 621-4642 and entering the conference code 6652068.

Aligos Therapeutics to Present Virtually at the Jefferies London Healthcare Conference 2021

On November 15, 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 that Lawrence M. Blatt, Ph.D., MBA, Chairman and CEO of Aligos, will present at the Jefferies London Healthcare Conference and participate in virtual investor 1×1 meetings on the November 18-19, 2021 (Press release, Aligos Therapeutics, NOV 15, 2021, View Source [SID1234595601]).

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A webcast of the presentation will be available starting November 18, 2021, 8:00 AM GMT / 3:00 AM ET on the events section of the Aligos Website or by clicking the following link: Webcast Registration. A replay will be available following the presentation for 30-days.

Please contact your Jefferies representative to schedule virtual one-on-one meetings with Aligos during the conference. For more information about the Jefferies London Healthcare Conference, please refer to the Jefferies Conference Website.

Syndax Pharmaceuticals Reports Third Quarter 2021 Financial Results and Provides Clinical and Business Update

On November 15, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the third quarter ended September 30, 2021 (Press release, Syndax, NOV 15, 2021, View Source [SID1234595600]). In addition, the Company provided a clinical and business update.

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"We are thrilled to announce that the pivotal Phase 2 portion of AUGMENT-101, examining SNDX-5613 across three distinct patient populations, is open and enrolling following a productive meeting with the U.S. Food and Drug Administration (FDA)," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Supported by a growing body of data, we firmly believe that SNDX-5613 is ideally positioned to be first to market and serve as a meaningful intervention for patients with mutant nucleophosmin (NPM1c) and mixed lineage leukemia rearranged (MLLr) acute leukemias who are deeply in need of effective therapies. We look forward to presenting updated data from the Phase 1 portion of the AUGMENT-101 trial, including updated durability of CR/CRh responses, during an oral session at the upcoming 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting."

"We were also pleased to recently announce our partnership with Incyte for the development and commercialization of axatilimab. This agreement represents a key step forward in our strategy to expand and maximize the program across multiple lines of treatment in chronic graft-versus-host disease (cGVHD) as well as additional fibrotic diseases, such as idiopathic pulmonary fibrosis (IPF), where the monocyte-macrophage lineage plays a vital role. As we continue to execute on advancing the program, we look forward to sharing updated data from our Phase 1/2 trial of axatilimab in cGVHD during an oral session at the ASH (Free ASH Whitepaper) Annual Meeting next month."

Recent Pipeline Progress and Anticipated Milestones

SNDX-5613

The Phase 2 portion of AUGMENT-101 is currently enrolling patients with NPM1c mutant and MLLr relapsed/refractory (R/R) acute leukemias. A total of 64 adult and up to 10 pediatric patients will be enrolled across each of the following three distinct trial populations: patients with NPM1 mutant acute myeloid leukemia (AML), patients with MLLr AML, and patients with MLLr acute lymphocytic leukemia. Discussions with the FDA have confirmed that AUGMENT-101 may potentially serve as the basis for regulatory filings in each of the three distinct trials. The primary endpoint for each of the three trials will be efficacy as measured by complete remission rate (complete response [CR] + CR with partial hematologic recovery rate [CRh]), with key secondary endpoints including duration of response and overall survival.
In November 2021, the Company announced that updated data from the Phase 1 portion of the ongoing AUGMENT-101 trial will be featured during an oral session at the 63rd ASH (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021. Data included in the abstract demonstrated robust clinical activity with durable responses and no discontinuations due to treatment-related adverse events. The oral presentation will include updated Phase 1 data from additional patients as of a more recent cutoff date, as well as further details on durability and CR/CRh rate by mutational status.
The Company reported plans to initiate a new trial to assess the anti-leukemic efficacy of SNDX-5613 in NPM1 or MLLr patients with measurable residual disease (MRD) progression following initial treatment. The trial will be conducted as part of the Australian Leukemia and Lymphoma Group (ALLG) INTERCEPT Master Clinical Trial, a collaborative clinical trial investigating novel therapies to target early relapse and clonal evolution as pre-emptive therapy in AML. SNDX-5613 is the first menin inhibitor to be included in the INTERCEPT AML Master Clinical Trial. The Company expects ALLG to initiate the trial in the first half of 2022.
In August 2021, the Company announced plans to initiate two additional trials to assess the safety, tolerability, and preliminary anti-leukemic efficacy of SNDX-5613 in combination with venetoclax and azacitidine as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial, and in combination with chemotherapy in patients with R/R NPM1 or MLLr acute leukemias in the AUGMENT-102 trial. The Company expects both trials to initiate in the first half of 2022.
Axatilimab

In November 2021, the Company announced that updated data from its Phase 1/2 trial of axatilimab in patients with cGVHD will be featured during an oral session at the 63rd ASH (Free ASH Whitepaper) Annual Meeting. Data included in the abstract demonstrated broad efficacy and tolerability for axatilimab in patients with relapsed or refractory cGVHD. The oral presentation will include additional follow up on all patients enrolled.
Enrollment is ongoing in the Company’s global pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD, with topline data expected in 2023. The trial will evaluate the safety and efficacy of three doses and schedules of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score.
In September 2021, Syndax and Incyte announced that they entered into an exclusive worldwide collaboration and license agreement to develop and commercialize axatilimab. Syndax and Incyte are seeking to develop axatilimab as a backbone therapy for patients with cGVHD as well as in additional immune-mediated diseases where CSF-1R-dependent monocytes and macrophages are believed to contribute to organ fibrosis. In addition to the ongoing global pivotal Phase 2 AGAVE-201 trial of axatilimab monotherapy in patients with cGVHD, the companies also plan to initiate additional trials of axatilimab in patients with cGVHD in 2022, including a Phase 2 trial in combination with a JAK inhibitor in patients with steroid-refractory cGVHD. Beyond cGVHD, Syndax plans to commence a Phase 2 proof of concept trial of axatilimab mid next year in patients with IPF, a serious, life-limiting orphan disease for which axatilimab may represent a much-needed treatment option with a novel mechanism of action.
Corporate Updates

In September 2021, Syndax announced the expansion of its Board of Directors to nine members with the appointment of Martin H. Huber, M.D. Dr. Huber has over 20 years of academic, biotechnology, and pharmaceutical drug development experience, currently serving as the President of Research and Development (R&D) and Chief Medical Officer of Xilio Therapeutics, Inc.
Third Quarter 2021 Financial Results

As of September 30, 2021, Syndax had cash, cash equivalents and short-term investments of $229.7 million and 52.2 million shares and share equivalents issued and outstanding. This includes 3.3 million pre-funded warrants.

Third quarter 2021 R&D expenses increased to $25.6 million from $14.4 million for the prior year period. The increase was primarily due to increased clinical trial activities and CMC activities for both SNDX-5613 and axatilimab.

General and administrative expenses for the third quarter 2021 increased to $6.8 million from $5.8 million for the prior year period. The increase is primarily due to increased professional fees.

For the three months ended September 30, 2021, Syndax reported a net loss attributable to common stockholders of $20.6 million or $0.40 per share compared to $20.4 million or $0.46 per share for the prior year period.

Financial Update and Guidance

For the full year of 2021, R&D expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, November 15, 2021.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.