On March 10, 2022 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing cancer therapies for patients in need of new treatment options, reported important corporate events and its financial results for the full year and the fourth quarter ended December 31, 2021 (Press release, Salarius Pharmaceuticals, MAR 10, 2022, View Source [SID1234609950]).

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We believe the progress made by Salarius Pharmaceuticals during 2021 and the early weeks of 2022 has once again been remarkable, including the Company’s recent expansion into the field of targeted protein degradation (TPD)," stated David Arthur, CEO of Salarius Pharmaceuticals. "TPD is a fast-growing field of cancer drug development with commercial market potential estimated in billions of dollars and enticing significant investment from some of the world’s largest pharmaceutical companies. TPD has the ability to develop drugs for historically undruggable cancer-promoting targets and our new TPD development program, built around the portfolio of assets acquired by Salarius in January 2022, is led by the product candidate SP-3164. Salarius now boasts a multipronged pipeline built around two exciting approaches to cancer drug development – protein inhibition and targeted protein degradation."

Financial Highlights:
•Cash and cash equivalents total $29.2 million on December 31, 2021, with an estimated runway into 2023
•For the three-month and 12-month periods ended December 31, 2021, net loss per common share, basic and diluted, of $0.09 and $0.31, respectively, compared to $0.10 and $0.50, respectively, for the same periods in 2020
Recent Business and Corporate Highlights:
•Acquisition of TPD assets from DeuteRx LLC in January 2022 expands Salarius’ oncology pipeline into a fast-growing area of drug research focused on pursuing historically undruggable cancer-promoting targets with estimated multi-billion-dollar commercial market potential
◦
◦Acquisition included the drug candidate SP-3164, additional targeted protein degrader programs, and a related intellectual property (IP) portfolio that includes issued composition of matter patents
◦SP-3164 is an orally administered cereblon-binding targeted protein degrader developed as a next-generation version of the widely studied molecular glue, avadomide (CC-122) with the potential for an improved efficacy and safety profile
▪SP-3164 leverages the data from 10 clinical trials investigating avadomide in more than 400 patients
▪Preclinical data suggests the potential for SP-3164 to be more efficacious than avadomide
▪Salarius will continue collaborating with DeuteRx scientists to complete Investigational New Drug (IND) enabling studies for SP-3164
▪First clinical trial expected in 2023
◦Consideration consists of cash and stock, with an upfront payment of $1.5 million in cash and 1 million shares of restricted stock; future payments linked to clinical, regulatory, and commercial milestones, and net sales royalties
•Salarius continues to advance clinical development of seclidemstat; data updates expected in 2022 from two Phase 1/2 clinical trials
◦Sarcoma trial investigating seclidemstat in combination with chemotherapy agents topotecan and cyclophosphamide (TC), in patients with relapsed/refractory Ewing sarcoma advanced to second lead-in safety dosing cohort treating patients with seclidemstat at 900 mg BID
◦Sarcoma trial investigating seclidemstat as a single-agent therapy in Myxoid Liposarcoma and other FET-rearranged sarcomas continues enrollment
◦Enrollment continues in investigator-initiated trial (IIT) at MD Anderson Cancer Center exploring seclidemstat in combination with azacytidine as a treatment for hematologic cancers; updates on biomarkers including immature cells (blasts) and drug activity measures expected during 2022
◦Research underway to identify new indications for seclidemstat in established large markets
Mr. Arthur concluded, "2022 is a year of significant optimism at Salarius given the potential value-building opportunities and milestones on the horizon. Salarius is moving to rapidly advance the development of SP-3164 with plans to file an IND and initiate our first clinical trial for that program in 2023. Building on our optimism around SP-3164 and based on the abundance of preclinical and clinical data collected around its predecessor, avadomide, it is our belief that SP-3164 will establish a superior safety profile with the potential to be more effective. Additionally, we continue advancing the clinical exploration of seclidemstat, our oral, reversible LSD1 inhibitor and Salarius’ most advanced investigational cancer drug candidate. We look forward to providing data updates in the coming months."

Three-Month and 12-Month Financial Results:
For the three-month period ended December 31, 2021, Salarius’ reported net loss was $4.1 million, or $0.09 per basic and diluted share, compared to a net loss of $1.8 million, or $0.10 per basic and diluted share for the same period in 2020. The loss for the three-month period ended December 31, 2021, increased by $2.3 million compared to the same time span last year, primarily because the Company had higher overall costs and no grant revenue in the current period.
For the 12-month period ended December 31, 2021, Salarius’ reported net loss was $12.8 million, or $0.31 per basic and diluted share, compared to a net loss of $7.4 million, or $0.50 per basic and diluted share for the same period in 2020. The loss for the 12-month period ended December 31, 2021, increased by $5.4 million compared to the loss from operations for the same period last year, resulting from increased charges related to R&D personnel and higher clinical trial costs more than offsetting lower drug development costs.
Net cash used for operating activities during the 12-month period ended December 31, 2021, totaled $10.2 million, essentially the same as approximately $10.3 million in the prior year. During 2021, the Company collected approximately $4.1 million on its outstanding grant receivable compared to $0.8 million in the prior year.
As of December 31, 2021, total cash, cash equivalents, and restricted cash were $29.2 million, compared to $11.1 million at year-end 2020. The increase in cash was primarily driven by the sale of equity securities during the first quarter of 2021. Current cash and cash equivalents are expected to fund our current planned operations into 2023.
Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Thursday, March 10, 2022, at 5:00 p.m. ET, to discuss its corporate and financial results for the fourth quarter and full-year 2021. Interested participants and investors may access the conference call by dialing either:

An audio webcast will be accessible via the Investors Events and Presentations section of the Company’s website View Source An archive of the webcast will remain available for 90 days beginning at approximately 6:00 p.m. ET on March 10, 2022.

On March 10, 2022 Scenic Biotech BV ("Scenic"), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, reported it has closed a Series A financing of ~$31 million (€28 million) (Press release, Scenic Biotech, MAR 10, 2022, View Source [SID1234609853]).

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The funds will be used to accelerate Scenic’s transition into a development stage company, advancing its in-house pipeline and supporting the creation of new programs based on its Cell-Seq genetic modifier target discovery platform including new industry collaborations.

The investment round was co-led by seasoned European investors, Eir Ventures, a Nordic Life Science venture fund, Switzerland-based BioMedPartners and Luxemburg-based Vesalius Biocapital. Existing Dutch and UK investors Inkef Capital, BioGeneration Ventures and Oxford Science Enterprises participated, together with Scenic’s founders and management.

Scenic will now advance its lead QPCTL small molecule immuno-oncology program into the clinic. In addition, by leveraging its Cel-Seq discovery platform, Scenic has generated a pipeline of disease modifying therapeutics to treat devastating inherited rare diseases. Three of these programs, which are based on druggable genetic modifiers, are being progressed towards IND enabling studies. The first program is centered on a small molecule to treat Niemann Pick Type C (NP-C), a rare lipid storage disorder that affects lipid metabolism, or the way fats, lipids, and cholesterol are transported in human cells. The second program is for Barth syndrome, an inherited mitochondrial disorder caused by mutations in the gene encoding Tafazzin. Barth syndrome is characterized by defects in cardiolipin, a critical component of the inner mitochondrial membrane. And the third program is to treat a severe heritable metabolic syndrome.

Alongside its in-house pipeline, Scenic has a multi-year, multi-indication strategic collaboration with Genentech, a member of the Roche Group, to discover, develop and commercialize novel therapeutics that target genetic modifiers. The collaboration has been expanded twice and now includes six scientific areas.

Following the Series A investment, Stephan Christgau PhD, General Partner Eir Ventures, Michael Wacker PhD, General Partner BioMedPartners and Stephane Verdood MSc, MBA, Managing Partner Vesalius Biocapital will join Scenic’s board of directors.

Commenting on behalf of the investors, Stephan Christgau said, "Scenic Biotech has the potential to become one of Europe’s most exciting biotech companies. It is a pioneer in the ground-breaking and promising new field of genetic modifiers. We are impressed by the power of its Cell-Seq platform and how it’s fueling a portfolio of in-house and partnered programs across multiple therapeutic areas. This financing will enable Scenic to continue to develop its platform and move its lead program into human clinical trials while bringing its rare disease programs to key value infection points."

Scenic Biotech’s CEO Oscar Izeboud said, "I am delighted to welcome our new investors to the company and to thank our existing investors for their continued support. We have made very significant progress since our seed investment, proving the value of our platform, extending its utility, and expanding our team. We look forward to continuing our growth and delivering on our promise to develop novel treatments to improve patients’ lives."

Scenic anticipates more than doubling its team in the coming 18 months including building its clinical capabilities. To accommodate the expansion Scenic will be expanding its facilities within the flourishing life sciences cluster at the Science Park in the East of Amsterdam.

On March 10, 2022 Phosplatin Therapeutics Inc., a clinical stage pharmaceutical company focused on oncology therapeutics, reported an in-person poster presentation will be given at the American Academy of Cancer Research (AACR) (Free AACR Whitepaper) on its lead candidate, PT-112, and how it selectively induces potent mitochondrial stress and immunogenic cell death in human prostate cancer cell lines (Press release, Phosplatin, MAR 10, 2022, View Source [SID1234609876]). The presentation will be held at the 2022 AACR (Free AACR Whitepaper) Annual Meeting taking place on April 8-13, 2022 in New Orleans.

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The work to be presented was generated by the company under a research collaboration with the Anel Lab at the University of Zaragoza, Spain. The characterization of intracellular pathways leading to ICD and the adaptive immune response is an important research aim of the company, and a related peer-reviewed manuscript is under preparation.

Presentation Title: "PT-112 induces potent mitochondrial stress and immunogenic cell death in human prostate cancer cell lines"
Lead Author: R. Soler, University of Zaragoza/Aragón Health Research Institute, Biochemistry and Molecular and Cell Biology, Zaragoza, Spain
Session Category: Experimental and Molecular Therapeutics
Session: Mechanisms of Drug Action 3
Session Date / Time: Monday, April 11, 2022 from 9:00am – 12:30pm CST
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 24
Poster Number: 5

The presentation will be available both in person and online in Proceedings of the AACR (Free AACR Whitepaper) through the conference website.

For more information about Phosplatin’s clinical trials, visit the website at www.Phosplatin.com.

About PT-112

PT-112 is the first small-molecule conjugate of pyrophosphate in oncology, and possesses a unique pleiotropic mechanism of action that promotes immunogenic cell death (ICD), through the release of damage associated molecular patterns (DAMPs) that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 may represent the best-in-class inducer of this immunological form of cancer cell death. Further, PT-112 harbors a property known as osteotropism, or the propensity of the drug to reach its highest concentrations in certain areas of the bone, making it a candidate for treatment of patients with cancers that originate in, or metastasize to, the bone. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" within the Developmental Therapeutics category at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress. The combination Phase 1b dose escalation study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in an oral presentation at the ESMO (Free ESMO Whitepaper) 2020 Virtual Congress. The Phase 1 study in patients with relapsed or refractory multiple myeloma presented at ASH (Free ASH Whitepaper) is the third completed Phase 1 study of PT-112. Monotherapy Phase 2 development is ongoing in mCRPC, and the PD-L1 combination study is ongoing in a dose confirmation cohort of non-small cell lung cancer (NSCLC) patients, and will soon include the Phase 2 proof of concept study in thymic epithelial tumors under the company’s collaboration with the NCI.

On March 10, 2022 Histogen Inc. (NASDAQ: HSTO), a clinical-stage therapeutics company focused on developing both restorative therapeutics and pan-caspase and caspase selective inhibitors focused on treatments for infectious and inflammatory diseases, reported financial results for the fourth quarter and year ended December 31, 2021 and provided an update on its clinical pipeline and other corporate developments (Press release, Conatus Pharmaceuticals, MAR 10, 2022, View Source [SID1234609892]).

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"We have a diverse pipeline of biologics and small molecule product candidates that we believe address large unmet market needs," said Steven J. Mento, Ph.D., Interim President and Chief Executive Officer. "Looking ahead, we will continue to focus on clinical execution with HST-003 in cartilage repair, and emricasan in COVID-19 with our partner Amerimmune while we explore the feasibility of testing emricasan in animal studies for methicillin resistant staphylococcus aureus infections (MRSA), and continue to evaluate our caspase-1 inhibitors that impact the inflammasome pathway. These activities support our overarching goal of enhancing the lives of patients as we seek to build value for our shareholders."

Highlights from the Fourth Quarter and Year Ended 2021 and Business Updates

HST-003 – In the second half of 2021, we initiated our Phase 1/2 clinical study of HST-003 to evaluate the safety and efficacy of human extracellular matrix (hECM) implanted within microfracture interstices and the cartilage defect in the knee to regenerate hyaline cartilage in combination with a microfracture procedure. We have experienced recruitment challenges due to both the specific nature of the study inclusion criteria and the impact of COVID-19 on the elective surgery environment. Additional qualified clinical sites have been added to help supplement recruitment and we will continue to evaluate the need to add more sites and study resources. We now anticipate top line results in the first half of 2023, assuming we complete enrollment in the fourth quarter of 2022.

HST-004 – Our initial preclinical research has shown that HST-004 stimulates stem cells from the spinal disc to proliferate and secrete aggrecan and collagen II, regenerate normal matrix and cell tissue structure and restores disc height. HST-004 was also shown to reduce inflammation and protease activity and upregulate aggrecan production in an ex vivo spinal disc model. In the second half of 2021, we initiated toxicology studies and other IND enabling activities for HST-004. However, due to pipeline program prioritization, we now anticipate filing IND for HST-004 in the second half of 2023.

Emricasan COVID-19 – In June 2021, we, jointly with Amerimmune LLC ("Amerimmune"), announced positive results from the Phase 1 study of emricasan in mild symptomatic COVID-19 patients. Emricasan was shown to be safe and well-tolerated during the 14 days of dosing and at the day 45 follow-up, as compared to placebo with no reports of serious adverse events. Patients who completed treatment with emricasan showed a statistically significant complete resolution of the symptoms most commonly associated in mild COVID-19, such as a cough, headache, and fatigue at day 7 which continued through day 45. No patients in the placebo arm experienced COVID-19 associated symptom resolution at any time point out to day 14. The mean number of days to recovery for patients in the emricasan arm was 4.8 days compared to 37.5 days in the placebo arm (p=0.001). In March 2022, we filed our demand for arbitration ("Arbitration Demand") as we believe that Amerimmune has failed to undertake commercially reasonable efforts toward conducting and completing the Phase 2 study as required by the Collaborative Development and Commercialization Agreement that we previously entered into with Amerimmune (the "Collaborative Agreement"). As part of our Arbitration Demand, we are seeking a declaratory judgement from the arbitrator to terminate the Collaborative Agreement, which would result in us regaining all rights that we licensed to Amerimmune under the Collaborative Agreement. In such event, we intend to conduct and complete the Phase 2 study independently. If the Collaborative Agreement is not terminated, we will likely continue to jointly develop emricasan pursuant to the terms of the Collaborative Agreement. In either case, we anticipate a Phase 2 study could be initiated in the second half of 2022.

Emricasan MRSA – We are exploring the feasibility of testing emricasan in animal studies of other infectious diseases, initially focused on methicillin-resistant staphylococcus aureus ("MRSA"). We anticipate completing the feasibility assessment in the second half of 2022.

CEO Transition – In November of 2021, the board appointed Steven J. Mento, Ph.D. as Executive Chairman and Interim President and Chief Executive Officer.

Fourth Quarter and Full-Year 2021 Financial Highlights

Fourth Quarter Ended December 31, 2021 and 2020

Product and Service Revenues for the three months ended December 31, 2021 and 2020 were $0 and $0.5 million, respectively. The decrease of $0.5 million was due to fulfillment timing of CCM supply orders to Allergan PLC ("Allergan"). Our obligation to supply CCM to Allergan was satisfied in 2021, and we have no additional purchase orders with Allergan for fulfillment.

Cost of revenues for the three months ended December 31, 2021 and 2020, we recognized cost of product revenue of $0 and $0.3 million, respectively. The decrease of $0.3 million for the three months ended December 31, 2021 as compared to the three months ended December 31, 2020 was due to the decrease in product sales to Allergan.

Research and development expenses for the three months ended December 31, 2021 and 2020 were $1.6 million and $1.9 million, respectively. The decrease of $0.3 million for the three months ended December 31, 2021 as compared to the three months ended December 31, 2020 was primarily due to decreases related to personnel related expenses.

General and administrative expenses for the three months ended December 31, 2021 and 2020 were $1.5 million and $1.8 million, respectively. The $0.3 million decrease for the three months ended December 31, 2021 as compared to the three months ended December 31, 2020 was primarily due to decrease in personnel related expenses and rent expense decrease, partially offset by an increase in legal and other administrative expenses.

Twelve Months Ended December 31, 2021 and 2020

Revenues

For the years ended December 31, 2021 and 2020, we recognized license revenues of $27 thousand and $0.9 million, respectively. The revenue recognized in both periods is associated with the Allergan Agreements. During the period ended December 31, 2021, $19 thousand of deferred revenue was recognized in relation to the Potential Future Improvements remaining performance obligation currently being amortized over the remaining 9-year patent life. The year-over-year decrease is primarily attributable to the recognition of the allocated proceeds received upon the transfer of the expanded license to Allergan in January 2020.

For the years ended December 31, 2021 and 2020, we recognized product revenues of $0.9 million and $0.8 million, respectively. The increase of $0.1 million for the year ended December 31, 2021, as compared to the year ended December 31, 2020 was due to a larger quantity of CCM sales to Allergan.

Grant revenue for the years ended December 31, 2021 and 2020 was $0.1 million and $0, respectively, all of which was related to an NSF research grant awarded to us in 2017. In March 2021, the Company completed all obligations under the NSF development grant and, as such, no longer generates any revenues in connection with the research and development grant.

For the year ended December 31, 2020, we recognized professional services revenue of $0.3 million, related to the transfer of certain technology and know-how, which was completed during 2020. As such, no additional professional services revenue was recognized during the year ended December 31, 2021.

Cost of Revenues

Cost of revenues for the years ended December 31, 2021 and 2020 were $0.2 million and $0.7 million, respectively. The decrease of $0.5 million for the year ended December 31, 2021 as compared to the year ended December 31, 2020 was primarily due to the sale of CCM to Allergan in September 2021 that was originally designated for research and development purposes and therefore had no cost of goods associated with it, and costs related to scrapped inventory.

For the years ended December 31, 2021 and 2020, we recognized costs of professional services of $0 and $0.3 million, respectively, related to the completion of technology transfer obligations of Histogen under the Allergan Agreements

In-process research and development expenses were $7.1 million for the year ended December 31, 2020 for in-process research and development acquired in connection with the Merger in May of 2020.

Research and development expenses for the years ended December 31, 2021 and 2020 were $8.5 million and $6.2 million, respectively. The increase of $2.3 million for the year ended December 31, 2021, as compared to the year ended December 31, 2020 was primarily due to increases in development costs of our clinical and pre-clinical product candidates and personnel related expenses, partially offset by $0.7 million in qualifying reimbursable expenses in connection with the DoD grant.

General and administrative expenses for the years ended December 31, 2021 and 2020 were $7.8 million and $6.6 million, respectively. This increase of $1.2 million for the year ended December 31, 2021 as

compared to the year ended December 31, 2020 was primarily due to increases in personnel related expenses, legal fees and insurance costs.

Cash and cash equivalents as of December 31, 2021 were $18.7 million. Histogen believes that its existing cash and cash equivalents and cash inflow from operations will be sufficient to meet Histogen’s anticipated cash needs into the second quarter of 2023.

On March 10, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported its operational and financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Autolus, MAR 10, 2022, View Source [SID1234609909]).

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"We rounded off the 2021 financial year announcing a collaboration with Blackstone Life Sciences, adding $150M in capital with an additional $100M in potential milestone payments triggered by future development progress of obe-cel, as well as positive clinical data from our pipeline, notably data from the Phase 1b portion of the FELIX study of obe-cel in adult ALL patients. Recruitment is ongoing in the Phase 2 portion of this pivotal study and we look forward to announcing initial Phase 2 data this year, as well as starting preparations for submitting a BLA in 2023," said Dr. Christian Itin, Chief Executive Officer of Autolus. "We are excited about Autolus’ outlook and look forward to updating you on progress with obe-cel, as well as AUTO4 and AUTO1/22 over the coming months."

Key Clinical and Pipeline Updates during 2021:

Obecabtagene autoleucel (obe-cel) in relapsed / refractory (r/r) adult ALL
FELIX Study – Autolus continues to enroll patients in the Phase 2 portion of the FELIX study. As presented at ASH (Free ASH Whitepaper) in December 2021, the data from the Phase 1b portion of the FELIX study show a favorable safety and efficacy profile consistent with our experience in the ALLCAR19 study in adult r/r B-ALL and the CARPALL study in pediatric r/r ALL patients treated with obe-cel. No patient experienced high grade (≥ Grade 3) cytokine release syndrome (CRS) and neurotoxicity (ICANS) of any grade was limited to 13% of patients and only 6% experienced a Grade 3 event.
ALLCAR19 Study – Data were published in the Journal of Clinical Oncology (JCO)(1) in September 2021 from the ALLCAR19 trial in r/r adult ALL patients. Obe-cel demonstrated a manageable adverse events profile, with no patients experiencing high grade CRS, despite the majority of patients enrolled in the study having a high disease burden prior to lymphodepletion. As presented at ASH (Free ASH Whitepaper) in December 2021, duration of response remains highly encouraging with morphological EFS for obe-cel of 46% at 24 months with a median follow-up of 29.3 months and patients approaching up to 42 months of durability, as of the cut-off date of October 15, 2021.

Obe-cel in r/r B-NHL – ALLCAR19 extension
The latest data of obe-cel in relapsed/refractory B-Cell Non-Hodgkin’s Lymphoma (B-NHL) and Chronic Lymphocytic Leukaemia (CLL) were presented by Autolus at ASH (Free ASH Whitepaper) in December 2021. As of the data cut-off date of October 15, 2021, 15 patients with r/r B-NHL and 1 patient with B-CLL had received obe-cel, with 14 patients evaluable for response. 14 of 14 patients responded to obe-cel, of which 13 of 14 patients achieved complete metabolic response per Lugano 2014, with 1 B-CLL patient achieving a partial response. 15 of 16 patients had no disease progression at last follow-up, with 1 of 16 patients having died in CR from COVID-19. Furthermore, long term persistence was demonstrated by quantitative PCR. Across all evaluable patients, obe-cel demonstrated a favorable safety profile with no ICANS or severe Grade ≥ 3 CRS events.
Autolus received preferred regulatory access for obe-cel from UK Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA):
Autolus received an innovative licensing and access pathway (ILAP) designation from the MHRA for obe-cel, which is being investigated in the ongoing FELIX trial in ALL.
Autolus also received PRIority MEdicines (PRIME) designation from the EMA.

AUTO1/22 in pediatric ALL
Pre-clinical data presented at ASH (Free ASH Whitepaper) in December 2021 demonstrated a high level of in vitro and in vivo activity of AUTO1/22 against leukemia cells. AUTO1/22 was shown to control leukemia in a mouse model of CD19 negative escape. AUTO1/22 is currently being tested in a study of r/r pediatric B-ALL. As of the cut-off date of October 21, 2021, 6 patients had received AUTO1/22. All patients showed engraftment of single and double CAR positive populations, pointing to early CAR T cell persistence.
AUTO4 in Peripheral T Cell Lymphoma
Autolus received ILAP designation from the MHRA. The AUTO4 Phase 1 clinical trial is progressing through dose escalation.
Key Operational Updates during 2021

In November 2021, Autolus announced that it had entered into a strategic collaboration and financing agreement under which Blackstone Life Sciences will provide up to $250 million in equity and milestones to support obe-cel, as well as next generation product therapies of obe-cel in B-cell malignancies.

In November 2021, Dr. William D. Young, a Senior Advisor to Blackstone Life Sciences, was appointed as a Non-Executive Director to the Autolus Board of Directors.

In September 2021, Autolus gave an update on its manufacturing strategy, announcing that planning approval had been granted to build the Company’s new 70,000 square foot manufacturing facility in Stevenage, UK. This commercial facility is designed for a capacity of 2,000 batches a year, with an opportunity to expand.

Also in September 2021, Autolus announced the appointment of John H. Johnson as Non-Executive Chairman of the Board of Directors.

In July 2021, Autolus announced its entry into an agreement with Moderna, Inc., granting Moderna an exclusive license to develop and commercialize mRNA-based therapeutics incorporating Autolus’ proprietary binders for up to four immuno-oncology targets.

Updates to Autolus’ executive team over the course of the year:
In July 2021, Autolus announced the appointment of Edgar Braendle M.D., Ph.D., as Chief Development Officer.
In October 2021, Alexander Swan was promoted to Senior Vice President, Human Resources and Dr. Chris Williams was promoted to Senior Vice President, Corporate Development.
Post Period Updates:

In January 2022 Autolus announced the retirement of Andrew J. Oakley as its Chief Financial Officer, effective March 31, 2022. Dr. Lucinda Crabtree, Senior Vice President of Business Strategy & Planning at Autolus, will succeed Mr. Oakley as Autolus’ Chief Financial Officer. Brent Rice was also promoted to Senior Vice President, Chief Commercial Officer.
Key Anticipated Clinical Milestones:

Updates on the FELIX trial, where Autolus is evaluating obe-cel in r/r adult ALL patients. The trial is currently enrolling patients into the Phase 2 portion. The Company expects to report initial clinical data from the Phase 2 study in H2 2022 and full data in H1 2023.

Updates from the ALLCAR19 extension trial in patients with r/r B-NHL and CLL and longer-term follow-up of the fully enrolled r/r adult ALL cohort expected in H1 2022.

Updates on the obe-cel Phase 1 trial, CAROUSEL, in Primary CNS Lymphoma in H1 2022.

Initial clinical data from the AUTO1/22 CARPALL extension trial in pediatric ALL expected to be reported in H1 2022 and with longer follow up in H2 2022.

Initial clinical data from AUTO4 LibraT1 Phase 1 trial in TRBC1+ Peripheral TCL expected to be reported in H1 2022.

AUTO8 in Multiple Myeloma Phase 1 trial expected to be initiated in H1 2022.

AUTO6NG – Neuroblastoma Phase 1 trial expected to start mid 2022.
(1)Roddie et al. "Durable responses and low toxicity after fast off-rate CD19 CAR-T therapy in adults with relapsed/ refractory B-ALL." DOI: 10.1200/JCO.21.00917 Journal of Clinical Oncology – published online before print August 31, 2021

Financial Results for the Quarter and Year Ended December 31, 2021

Cash at December 31, 2021, totaled $310.3 million, as compared to $153.3 million at December 31, 2020. Net total operating expenses for the twelve months ended December 31, 2021 were $165.0 million, net of grant income and license revenue of $2.3 million, as compared to net operating expenses of $168.1 million, net of grant income and license revenue of $1.7 million, for the same period in 2020.

Research and development expenses remained relatively flat at $134.8 million for the year ended December 31, 2021 when compared to $134.9 million for the year ended December 31, 2020. Cash costs, which exclude depreciation and amortization as well as share-based compensation, increased to $121.4 million from $116.9 million. The increase in research and development cash costs of $4.5 million consisted primarily of (i) an increase in compensation and employment related costs of $3.8 million due to a combination of an increase in employee headcount, to support the advancement of our product candidates in clinical development, and to severance payments related to the reduction in workforce that was initiated during the first quarter of 2021, (ii) an increase of $2.5 million in facilities costs related to the continued scaling of manufacturing operations, (iii) an increase of $2.4 million in purchased consumables used in the manufacturing of obe-cel in the FELIX study, (iv) an increase of $0.9 million in IT infrastructure and support for information systems related to the conduct of clinical trials and manufacturing operations, and (v) an increase of $0.1 million related to cell logistics, which is offset by a reduction in clinical trial costs of $5.2 million.

Non-cash costs decreased to $13.4 million for the year ended December 31, 2021 from $18.1 million for the year ended December 31, 2020. The $4.7 million decrease of non-cash costs is related to a decrease of $7.7 million share-based compensation expense as a result of a lower fair value of options recognized during the period and due to the reduction in workforce that was initiated during the first quarter of 2021, offset by a $3.0 million increase in depreciation expense.

General and administrative expenses decreased to $31.9 million for the year ended December 31, 2021 from $35.0 million for the year ended December 31, 2020. Cash costs, which exclude depreciation as well as share-based compensation decreased to $26.7 million from $27.4 million. There were decreases of $0.7 million of costs related to (i) $0.8 million of expenses relating to the Company’s commercial preparation costs, (ii) $0.6 million of employee compensation expense due to the reduction in workforce during the first quarter of 2021 and lower retention costs, (iii) $0.5 million of facilities costs, and (iv) $0.1 million in general administration expenses, offset by increases in director and officer insurance and IT infrastructure and support for information systems of $1.0 million and $0.3 million, respectively.

Non-cash costs decreased to $5.2 million for the year ended December 31, 2021 from $7.6 million for the year ended December 31, 2020. The $2.4 million decrease of non-cash costs is mainly attributed to lower share-based compensation expenses as a result of the lower fair value of options recognized during the period and due to the reduction in workforce that was initiated during the first quarter of 2021.

Interest income decreased to $0.3 million for the year ended December 31, 2021 from $0.5 million for the year ended December 31, 2020. This decrease is due to the lower cash balances held during the year combined with lower interest rates for cash held on deposit.

Interest expense increased to $1.1 million for the year ended December 31, 2021 and relates to the liability relating to the sale of future revenue which arose upon entering into the Collaboration and Financing Agreement with Blackstone.

Income tax benefit decreased to $23.9 million for the year ended December 31, 2021 from $24.2 million for the year ended December 31, 2020 due to small decrease in the research and development expenditures which were qualifying for tax credits for the year. As research and development credits fell at a faster rate than the Company’s net loss before income tax, this led to a lower effective tax rate. Research and development credits are obtained at a maximum rate of 33.35% of the Company’s qualifying research and development expenses, and the decrease in the net credit was primarily attributable to a decrease in its eligible research and development expenses.

Net loss attributable to ordinary shareholders was $142.1 million for the twelve months ended December 31, 2021, compared to $142.1 million for the same period in 2020. The basic and diluted net loss per ordinary share for the twelve months ended December 31, 2021, totaled $(1.97) compared to a basic and diluted net loss per ordinary share of $(2.76) for the twelve months ended December 31, 2020.

Autolus estimates that its current cash on hand and anticipated milestone payments from Blackstone, extends the Company’s runway into 2024.

Conference Call

Management will host a conference call and webcast today at 8:30 am ET/1:30 pm GMT to discuss the Company’s financial results and provide a general business update. To listen to the webcast and view the accompanying slide presentation, please go to the events section of Autolus’ website.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID: 2794888. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID: 2794888.