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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On November 15, 2021 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a developer of next-generation biopharmaceuticals and pioneer of the sustainable FastPharming Manufacturing System, reported its financial results for the fiscal quarter ended September 30, 2021 and provides a corporate update (Press release, iBioPharma, NOV 15, 2021, View Source [SID1234595599]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are continuing to accelerate the execution of our growth strategy as we start our fiscal year 2022," said Tom Isett, Chairman & CEO of iBio. "Having only just launched our oncology initiative six months ago, we have already created six active programs, including the two new ones announced today. In addition, we have advanced our novel COVID-19 vaccine candidate to pre-IND, while concurrently building out our vaccine platform with new drug delivery technology partnerships. We have also acquired our cGMP manufacturing facility and taken sole ownership of our CDMO subsidiary, along with the attendant rights to manufacture biologics using our FastPharming System in the United States. We are confident that our investments over the past year in talent, technology, and infrastructure will return value to patients and shareholders as we go forward."

Fiscal First Quarter and Recent Business Developments:

BIOPHARMACEUTICALS

Therapeutics

In August 2021, iBio added to its pipeline by in-licensing an IL-2 sparing anti-CD25 antibody (IBIO-101) from RubrYc Therapeutics. Pre-clinical data shows that the molecule can deplete regulatory T cells from the tumor microenvironment and restore anti-tumor immunity, potentially representing an important new tool in the fight against cancer.
The Company continues to advance its endostatin E4 molecule for fibrotic diseases (IBIO-100) toward the clinic. Meanwhile, leveraging its investment in endostatin E4, iBio announced earlier today that it will separately explore the molecule’s usefulness in the treatment of solid tumors as part of a research collaboration with the University of Texas Southwestern Medical Center.
Pursuant to its new partnership with RubrYc Therapeutics wherein artificial intelligence ("AI")-based antibody discovery technologies are deployed, iBio announces today that the first new target is now being optimized using the platform. This result follows just two months after the joint discovery collaboration was initiated.
Vaccines

The Company recently conducted a preclinical dose ranging study of IBIO-202, its nucleocapsid ("N") antigen-based, intramuscularly ("IM")-delivered COVID-19 vaccine candidate. The results confirmed generation of a robust, antigen-specific, memory T-cell response and provided data that further informed iBio’s product formulation strategy.
The Company submitted a pre-IND package for IBIO-202 to the U.S. Food and Drug Administration in September 2021 and is anticipating a response in the coming months.
Today, iBio announces that is has entered a collaboration with a leading innovator of microarray patch systems, which are a painless alternative to IM injections. The first objective of the collaboration is to evaluate feasibility of intradermal delivery of a COVID-19 vaccine antigen. If successful, the partnership has the potential to drive improved access to vaccines by avoiding cold chain issues and possibly enabling self-administration. The collaboration is not expected to impact the current development timeline of IBIO-202.
The Company continues to advance its Classical Swine Fever ("CSF") vaccine candidate, IBIO-400, through the U.S. Department of Agriculture’s Centers for Veterinary Biologics’ regulatory process. Additionally, iBio announces today that studies are now underway at Texas A&M University System to evaluate alternatives to IM injection of its CSF vaccine candidate, including an oral dose option.
BIOPROCESS

In November 2021, iBio purchased the Bryan, Texas, manufacturing facility it previously operated under a lease from two affiliates of Eastern Capital Limited (the "Eastern Affiliates"). The Company also acquired the equity interest in iBio CDMO, LLC, that was formerly held by the Eastern Affiliates. As a result, the subsidiary and its intellectual property are now wholly-owned by iBio.
Fiscal First Quarter and Recent Corporate Developments:

iBio made progress in government relations during the quarter by engaging with senior-level U.S. policymakers to build awareness of the FastPharming System and to underscore the benefits of rapid, sustainable, domestic bioproduction.
iBio is encouraging shareholders who held common stock at the close of business on October 15, 2021 to vote "FOR" all proposals put forth in the proxy statement prior to its December 9, 2021, Annual Meeting of Stockholders to help the Company continue to grow its pipeline, services, and value.
"Over the course of the next year, we are focused on delivering shareholder value across multiple fronts, including announcing new oncology targets and two possible IND filings," said Mr. Isett. "We are also excited about the partnership opportunities that we see ahead for our bioprocess business as industry awareness of the need to implement sustainable, ESG-based practices continues to grow. With sole ownership of our FastPharming Facility and CDMO subsidiary now secured, we have additional strategic and operational flexibility to deploy our rapid, scalable biologics development system for our own pipeline, as well as for clients, so that we may enable more potentially life-saving therapeutics to enter the clinic."

Financial Results:

Revenues for the first fiscal quarter ended September 30, 2021, were approximately $211,000, a decrease of 49% from approximately $410,000 in the same period of 2020. As noted previously, significant quarter-to-quarter revenue variability is commonplace for early-stage pharma services companies given the timing of revenue recognition. Consistent with these business dynamics, iBio continues to expect a sequential decline in revenue during the first half of fiscal 2022 compared to the second half of fiscal 2021, followed by higher growth in the second half of fiscal 2022.

R&D and G&A expenses for the first quarter of fiscal 2022 increased 35% and 24%, respectively, over the comparable period in fiscal 2020. This reflects the Company’s growing investments in its pipeline, platform technologies, employees, and related infrastructure. iBio anticipates this trend continuing, however, the rate of growth is expected to moderate over time.

iBio’s consolidated net loss for the first quarter ended September 30, 2021, was approximately $8.9 million, or $0.04 per share, compared to a net loss of approximately $7.5 million, or $0.05 per share, in the same period of 2020.

iBio had $82.3 million dollars in cash and cash equivalents and investments in debt securities as of September 30, 2021. The Company used $9.3 million in cash during the quarter for operating activities and $5.1M for investing activities which included elements of the RubrYc transaction and for capital expenditures. Subsequent to the end of the first quarter of fiscal 2021, iBio used approximately $6.0 million in cash to help fund the purchase of its manufacturing facility in Bryan, Texas, and to secure full control of iBio CDMO, LLC, which holds the exclusive rights to manufacture using the FastPharming System in the United States. Taking into account potential long-term financing options, combined with the approximate 67% savings in facility-related cash requirements expected to be achieved through this transaction, the Company continues to believe that its current cash position is sufficient to fund its operations through Q3-FY23. If the Company cannot take advantage of the additional financial flexibility, and based on the Company’s working capital on September 30, 2021, management has concluded that there is sufficient liquidity to fund normal operations through at least Q2-FY23.

Webcast and Conference Call

iBio management will host a webcast and conference call at 4:30 p.m. Eastern Time today, November 15, 2021, to discuss these results and provide a corporate update.

The live and archived webcast may be accessed on the Company’s website at www.ibioinc.com under "News and Events" in the Investors section. The live call can be accessed by dialing (833) 672-0651 (domestic) or (929) 517-0227 (international) and referencing conference code: 7779281.