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.

On March 10, 2022 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Applied Therapeutics, MAR 10, 2022, View Source [SID1234609906]).

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"We remain committed to bringing new treatments to patients in areas of high unmet need," said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. "With three ongoing Phase 3 studies across Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency and Diabetic Cardiomyopathy, we are advancing treatments forward, and expect multiple catalysts and opportunities for value creation in 2022 and 2023."

Recent Highlights

Provided Regulatory Update on Galactosemia Program. In January 2022, the Company provided a regulatory update on the AT-007 Galactosemia program. Following discussions with the FDA at the end of the year, the Company decided to hold on submitting an NDA for AT-007 for treatment of Galactosemia pending additional discussions with the agency. Although the Galactosemia program had previously been discussed in the context of an NDA for Accelerated Approval based on reduction in galactitol, the FDA has now indicated that clinical outcomes data will likely be required for approval. Clinical outcomes are assessed every 6 months by a firewalled committee until the study reaches statistical significance. All children enrolled in the study have completed the 6-month clinical outcomes visits. The Company is in active dialogue with the FDA regarding a potential NDA submission and expects to complete the statistical analysis of the 6-month clinical outcomes in the second quarter. The Company expects to provide an update accordingly.
Announced Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency. In December 2021, the Company initiated the global registrational phase 2/3 study of AT-007 in Sorbitol Dehydrogenase (SORD) Deficiency. The study, termed INSPIRE (INhibition of Sorbitol Production through Inhibition of the Aldose Reductase Enzyme), will investigate biomarker efficacy, clinical outcomes and safety in people living with SORD Deficiency treated with AT-007 vs. placebo.
Financial Results

Cash and cash equivalents and short-term investments totaled $80.8 million as of December 31, 2021, compared with $96.8 million at December 31, 2020.
Research and development expenses for the year ended December 31, 2021 were $62.6 million, compared to $61.8 million for the year ended December 31, 2020. The increase of approximately $0.8 million was primarily related to an increase in clinical and pre-clinical expense of $11.6 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-007 ACTION-Galactosemia Kids pediatric registrational study; a decrease in drug manufacturing and formulation expenses of $12.5 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months ended March 31, 2021; an increase in personnel expenses of $2.2 million due to the increase in headcount in support of our clinical program pipeline; an increase in stock-based compensation of $0.2 million due to new stock option and restricted stock unit grants, offset by forfeitures of stock option and restricted stock unit grants; and a decrease of regulatory and other expenses of $0.6 million primarily related to the UM license fees recognized during the year ended December 31, 2020.
General and administrative expenses were $43.0 million for the year ended December 31, 2021, compared to $32.7 million for the year ended December 31, 2020. The increase of approximately $10.4 million was primarily related to a decrease in professional and legal fees of $2.1 million due to lower external consulting and legal fees; an increase of $5.6 million related to increased commercial expenditures; an increase in personnel expenses of $1.0 million and an increase in stock-based compensation of $3.0 million due to an increase in headcount; an increase of insurance expenses of $0.6 million related to increased directors and officers liability insurance costs; and an increase in other expenses of $2.3 million, primarily relating to increased costs of rent and other office expenses.
Net loss for the year ended December 31, 2021 was $105.6 million, or $4.12 per basic and diluted common share, compared to a net loss of $94.0 million, or $4.28 per basic and diluted common share, for the year ended December 31, 2020.

On March 10, 2022 Sensei Biotherapeutics, Inc. (NASDAQ: SNSE), an immunotherapy company focused on the discovery and development of next generation therapeutics for cancer, reported financial results for 2021 and provided recent corporate updates (Press release, Sensei Biotherapeutics, MAR 10, 2022, View Source [SID1234609905]).

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"In 2021, we established important proof points for our SNS-101 program, which is designed to block the VISTA immune checkpoint," said John Celebi, president and chief executive officer of Sensei Biotherapeutics. "VISTA is widely expressed on myeloid cells, presenting a challenge for drug development, yet plays a decisive role in suppressing the immune system in a broad set of tumor types. SNS-101 is a potent and pH-selective fully human anti-VISTA antibody that is designed to be highly selective for tumors and therefore has potential to overcome historical challenges associated with targeting VISTA."

Mr. Celebi continued, "More broadly, our TMAb platform represents a fundamental advance for a number of important immune targets that are expressed in both normal tissues and tumors, avoiding potential pharmacokinetic sinks and on-target, off-tumor toxicities. Importantly, we are well funded with more than $147 million as of December 31, 2021 to advance our programs."

2021 Highlights and Milestones:

Pipeline

SNS-101 is a monoclonal antibody targeting the immune checkpoint VISTA and is currently in IND-enabling studies. VISTA (V-domain Ig suppressor of T cell activation) is an immune checkpoint that is implicated in resistance to PD-1/PD-L1 and correlates with poor survival across numerous cancers. In 2021, Sensei achieved the following milestones for this program:

In August, Sensei announced it had identified SNS-101, a potent pH-dependent product candidate that selectively blocks the interaction of VISTA with its receptor, PSGL-1, which occurs at low pH, as seen in the tumor microenvironment. The identification of SNS-101 was partly based on nonclinical data from a human VISTA knock-in mouse model, which showed that TMAb antibodies significantly enhanced anti-tumor responses in combination with PD-1 blockade compared to treatment with PD-1 blockade alone.
In November, Sensei presented a poster for SNS-101 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting being held in Washington, D.C. Data highlighted in the poster were the first preclinical data to be presented by Sensei in a scientific forum and highlighted the potency, selectivity, and mechanism of action of SNS-101.
In November, Sensei hosted a virtual science symposium to discuss the potential of the VISTA checkpoint inhibitor to address current limitations of immune checkpoint therapy.
Sensei entered into an agreement with a high-quality CDMO for the manufacture of GMP-grade material to advance toward clinical studies.
Sensei has initiated IND-enabling studies for SNS-101. Key nonclinical studies include the generation of a broader set of in vivo efficacy data from Sensei’s human VISTA knock-in mouse models, nonclinical pharmacokinetic data, and nonclinical safety data. Sensei expects to receive pharmacokinetic and toxicology data from its single dose non-human primate studies in mid-2022 and submit an IND in the first half of 2023.
SNS-102 is a monoclonal antibody targeting VSIG4 (V-Set and Immunoglobulin Domain Containing 4), a B7-family related protein that is frequently overexpressed on tumor-associated macrophages. VSIG4 is a potent inhibitor of T-cell activity and potential driver of immunosuppressive macrophage polarization. Expression of VSIG4 is also found within normal tissues, presenting potential safety challenges, making VSIG4 an ideal candidate for Sensei’s TMAb platform.

Sensei has initiated its TMAb antibody discovery campaign aimed at developing an inhibitory antibody with high selectivity for VSIG4 in the TME versus normal tissue environments.
Sensei plans to select a product candidate and initiate IND-enabling studies in 2023.
SNS-103 is a monoclonal antibody targeting ENTPDase1 (ecto-nucleoside triphosphate diphosphohydrolase-1), also known as CD39. ENTPDase1 is the upstream, rate-limiting enzyme, leading to the breakdown of extracellular ATP. Extracellular ATP represents a potent immunologic "danger signal", which drives immune activation. The ultimate downstream product of this pathway, adenosine, has potent immunosuppressive activity through binding to adenosine receptors. Upregulation of ENTPDase1 by tumors is common and leads to a diminished anti-tumor immune response.

Sensei has initiated a TMAb antibody campaign aimed at developing an inhibitory antibody with high selectivity for ENTPDase1 in the TME versus normal tissue environments.
Sensei expects to select a product candidate in 2023.
SNS-401-NG is a potential first-in-class, multi-antigenic personalized ImmunoPhage candidate being developed in collaboration with the University of Washington designed to treat a broad range of cancers. The first proof-of concept clinical application is directed to the treatment of Merkel cell carcinoma (MCC), an aggressive form of skin cancer commonly driven by the Merkel Cell Polyoma Virus. Sensei plans to conduct a broader study in patients with multiple tumor types, potentially including head and neck cancer, lung cancer, melanoma, and triple negative breast cancer based on the prevalence of Phortress antigens.

Sensei is finalizing the genetic design of its next generation ImmunoPhage, which will serve as the backbone for delivery of anti-tumor antigens to the immune system.
Sensei intends to initiate IND-enabling studies for this product candidate in the second half of 2022.
Corporate

Sensei completed an upsized initial public offering of its common stock in February 2021. The gross proceeds to Sensei, before deducting underwriting discounts and commissions and other offering expenses payable by Sensei, were approximately $152.6 million.
Sensei Biotherapeutics completed the move of its corporate headquarters to the Seaport Innovation District in Boston, MA. The new space more than doubles the size of the company’s current research and development footprint. The company’s manufacturing operations remain in Rockville, MD.
Sensei strengthened its board of directors with the appointments of Deneen Vojta, Kristian Humer, and Jessie English.
In January 2022, Sensei announced the promotions of Erin Colgan as Chief Financial Officer and Robert Pierce, M.D., as Chief R&D Officer.
On March 9, 2022, Sensei announced the appointment William Ringo as the Chair of the Board of Directors.
Year End 2021 Financial Results

Cash Position – Cash, cash equivalents and marketable securities were $147.6 million as of December 31, 2021, as compared to $16.6 as of December 31, 2020. Sensei expects the current cash balance to fund operations at least into the first half of 2024.

Research and Development (R&D) Expenses – R&D expenses were $21.7 million for the year ended December 31, 2021, compared to $11.9 million for the year ended December 31, 2020, including Alvaxa IPR+D. The increase in R&D expenses was primarily attributable to increased headcount to support Sensei’s research, development, and manufacturing activities.

General and Administrative (G&A) Expenses – G&A expenses were $15.8 million for the year ended December 31, 2021, compared to $7.5 million for the year ended December 31, 2020. The increase in G&A expenses was primarily attributable to higher personnel costs, including stock-based compensation expense, and costs associated with operating as a public company.

Net Loss – Net loss was $36.8 million, for the year ended December 31, 2021, compared to $20.1 million for the year ended December 31, 2020.

On March 10, 2022 Theseus Pharmaceuticals, Inc. (NASDAQ: THRX) (Theseus or the Company), a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients through the discovery, development and commercialization of transformative targeted therapies, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Theseus Pharmaceuticals, MAR 10, 2022, View Source [SID1234609904]).

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"2021 was a transformative year for Theseus—we evolved into a clinical stage company, strengthened our team, and completed a successful IPO," said Tim Clackson, Ph.D., President and Chief Executive Officer of Theseus. "In January 2022 we initiated a Phase 1/2 clinical trial to evaluate our lead candidate, THE-630, in patients with advanced GIST and continue to drive towards development candidate nomination for our fourth-generation EGFR program in non-small cell lung cancer. Over the course of this year, we look forward to executing across our pipeline, which includes advancing THE-630 in the clinic, initiating IND-enabling studies of our second development candidate targeting EGFR, and expanding our pipeline with one or more additional kinase targets."

Recent Business Highlights and Anticipated Milestones:

Initiated Phase 1/2 clinical trial to evaluate THE-630 in patients with GIST. First patient treated in January 2022 in a Phase 1/2 dose escalation and expansion clinical trial of THE-630 in patients with advanced GIST. THE-630 is a pan-variant inhibitor of the receptor tyrosine kinase KIT designed for patients with advanced GIST whose cancer has developed resistance to earlier lines of therapy. Initial data from the Phase 1 portion of the clinical trial is expected to be presented at a scientific meeting in the first half of 2023.
Nomination of development candidate for fourth-generation EGFR program expected in the third quarter of 2022. Theseus expects to nominate an EGFR candidate designed to inhibit the full range of single-, double-, and triple-mutant EGFR variants found in the tumors of patients with EGFR-mutant NSCLC that have developed resistance to osimertinib, including the C797S mutation. Preclinical data is expected to be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April and at a scientific conference in the second half of 2022. IND submission is expected in 2023.
One or more additional kinase targets expected to be nominated by the end of 2022.
Successfully completed initial public offering (IPO). In October 2021, the Company completed a successful IPO, raising $178.8 million in aggregate gross proceeds, and listed on the Nasdaq Global Select Market.
Fourth Quarter and Full Year Financial Results:

Cash Position: As of December 31, 2021, Theseus had cash and cash equivalents of $244.7 million. Theseus expects its cash and cash equivalents, including proceeds from the IPO, to fund operations and capital expenditures into the second half of 2024 based on its current operating plan.
R&D Expenses: Research and development expenses were $5.0 million for the fourth quarter of 2021, as compared to $2.5 million for the fourth quarter of 2020. This increase was primarily due to $1.6 million of increased employee-related costs, and $0.9 million in increased expenses for clinical and preclinical studies. Research and development expenses were $18.3 million for the full year 2021, as compared to $6.0 million for the full year 2020.
G&A Expenses: General and administrative expenses were $3.6 million for the fourth quarter of 2021, as compared to $0.4 million for the fourth quarter of 2020. This increase was primarily due to $1.6 million of increased employee-related costs, primarily due to increases in our headcount, as well as $1.7 million of increased general expenses, primarily driven by public company-related costs. General and administrative expenses were $9.0 million for the full year 2021, as compared to $0.9 million for the full year 2020.
Net Loss: Net loss was $8.7 million for the fourth quarter of 2021, as compared to a net loss of $2.9 million for the fourth quarter of 2020. Net loss was $27.3 million for the full year 2021, or a net loss per share of $2.84, as compared to a net loss of $12.0 million for the full year 2020, or a net loss per share of $21.10.

On March 10, 2022 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming platform, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Omega Therapeutics, MAR 10, 2022, View Source [SID1234609903]).

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"In 2021, we made significant strides across all aspects of our business highlighted by the successful completion of our initial public offering, the nomination of OTX-2002 as the industry’s first programmable epigenetic medicine to be developed for the treatment of c-Myc (MYC)-driven hepatocellular carcinoma (HCC), and the continued development of our groundbreaking platform and pipeline," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "With our funding in 2021 and recent key additions to our team, we are well positioned to steadily advance a broad portfolio of Omega Epigenomic Controllers (OECs). Looking ahead, we are targeting to submit an Investigational New Drug application (IND) for OTX-2002 and nominate two OEC candidates in the first half of 2022. We are also planning for an additional IND in the second half of 2022 or early 2023 and several scientific presentations and publications throughout the year."

Recent Business Highlights and Corporate Update

Development Pipeline and Platform

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022: An abstract titled, "Epigenetic Modulation of the MYC oncogene as a potential novel therapy for HCC" was selected for a poster presentation at the upcoming AACR (Free AACR Whitepaper) 2022 Annual Meeting. The presentation will highlight the mechanism of action of OTX-2002 and its potential as a differentiated and viable approach to the treatment of HCC.
OTX-2002: IND-enabling studies are ongoing for Omega’s lead OEC candidate OTX-2002, a novel, engineered, and programmable mRNA therapeutic targeting the MYC oncogene in patients with HCC. In preclinical studies, OTX-2002 demonstrated its ability to potently downregulate MYC oncogene expression. The Company continues to be on track to file an IND for OTX-2002 in the first half of 2022.
Additional OEC Development: The Company is working on multiple programs in pre-clinical studies, including acute respiratory distress syndrome (ARDS) with CXCL1-3/IL8, non-small cell lung cancer (NSCLC) with MYC, alopecia with SFRP1, and liver disease with HNF4a.
OMEGA Epigenomic Programming Platform: Omega is creating a new generation of programmable epigenetic mRNA medicines that are designed to control the fundamental epigenetic processes to correct the root cause of disease by restoring aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega has developed a highly rational and deterministic approach to drug design that enables the Company to rapidly develop and optimize novel OECs with high target specificity to durably tune the expression of single or multiple genes. Omega is advancing multiple pre-clinical development programs in oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
Corporate

In January 2022, Yan Moore, M.D., was appointed Chief Medical Officer of Omega. Dr. Moore has extensive management, research, translational drug development and medical affairs experience across various pharmaceutical and biotechnology companies.
Anticipated Milestones and Key Priorities for 2022

Complete IND-enabling studies for OTX-2002 and file the Company’s first IND to the U.S. Food and Drug Administration (FDA) during the first half of 2022.
Declare two OEC development candidates in the first half of 2022.
Target submission of an additional IND application in the second half of 2022 or early 2023.
Continue to develop the OMEGA Epigenomic Programming platform and investigate additional development programs to expand pipeline.
Publish and present relevant preclinical and early clinical data supporting programs and platform development.
Fourth Quarter and Full Year 2021 Financial Results

As of December 31, 2021, the Company had cash, cash equivalents and marketable securities totaling $225.3 million.

Research and development (R&D) expenses for the fourth quarter of 2021 were $14.7 million, compared with $7.1 million for the fourth quarter of 2020. R&D expenses for 2021 were $47.9 million, compared to $21.1 million in 2020. The $26.8 million increase in R&D expenses in 2021 compared to 2020 was primarily due to an increase in discovery and preclinical development costs and personnel and related expenses as the Company continues to advance its pipeline and discovery portfolio.

General and administrative expenses (G&A) for the fourth quarter of 2021 were $5.7 million, compared with $1.9 million for the fourth quarter of 2020. G&A expenses for 2021 were $16.6 million, compared to $6.2 million in 2020. The $10.4 million increase in G&A expenses in 2021 compared to 2020 was primarily due to higher personnel and related expense and increased costs to operate as a public company, in addition to the higher professional fees to support business growth.

Net loss for the fourth quarter of 2021 was $20.9 million, compared with $9.5 million for the fourth quarter of 2020. Net loss for the year ended December 31, 2021 was $68.3 million, compared to a net loss of $29.4 million for the year ended December 31, 2020. The increase in net loss for 2021 compared to 2020 was primarily due to increased R&D and G&A expenses to support the Company’s growth and operations as a public company.