On March 27, 2023 GSK plc (LSE/NYSE: GSK) reported interim results from Part 1 of the RUBY/ENGOT-EN6/GOG3031/NSGO phase III trial investigating Jemperli (dostarlimab) plus standard-of-care chemotherapy (carboplatin-paclitaxel) followed by dostarlimab compared to chemotherapy plus placebo followed by placebo in adult patients with primary advanced or recurrent endometrial cancer (Press release, GlaxoSmithKline, MAR 27, 2023, View Source [SID1234629373]).

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Hesham Abdullah, Senior Vice President, Global Head of Oncology Development, GSK said: "These positive results from the RUBY trial bring us one step closer to addressing the significant unmet needs of endometrial cancer patients and add to the growing body of evidence on dostarlimab, strengthening our belief in its potential to transform cancer treatment as a backbone immuno-oncology therapy."

A statistically significant and clinically meaningful improvement in progression free survival (PFS) was observed for dostarlimab plus carboplatin-paclitaxel in the mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) population (n=118) and in the overall population (n=494) versus placebo plus chemotherapy. The separation of the lines in the Kaplan-Meier curve below illustrates the significant reduction in risk of disease progression or death in patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer in the dostarlimab plus chemotherapy treatment arm compared to the placebo plus chemotherapy treatment arm.

Dr Mansoor Raza Mirza, Chief Oncologist, Copenhagen University Hospital, Denmark and RUBY principal investigator, said: "Clinical practice has been waiting decades for a meaningful advancement in the standard of care for primary advanced or recurrent endometrial cancer. The results from the RUBY clinical trial, especially given the difficult-to-treat histologies included in the trial, demonstrate support for a new treatment standard with the addition of dostarlimab to current standard-of-care chemotherapy."

Additionally, at this first interim analysis, there was a clinically meaningful overall survival (OS) trend in the overall population among patients receiving dostarlimab plus chemotherapy followed by dostarlimab. The analysis was done at 33% maturity and statistical significance was not reached. OS follow-up continues and further analysis is planned. PFS and OS summaries are listed below.

dostarlimab + chemotherapy placebo + chemotherapy
PFS dMMR/MSI-H population
Number of patients evaluated 53 65
HR (95% CI)
0.28
(0.162-0.495)

P-value P<0.0001
At 24 months (95% CI)
61.4%
(46.3–73.4)

18.1%
(13.0%–23.9%)

PFS overall patient population
Number of patients evaluated 245 249
HR (95% CI) 0.64
(0.507–0.800)
P-value P<0.0001
At 24 months (95% CI)
36.1%
(29.3%–42.9%)

18.1%
(13.0%–23.9%)

PFS mismatch repair proficient (MMRp)/microsatellite stable (MSS) populationa
Number of patients evaluated 192 184
HR (95% CI) 0.76
(0.592–0.981)
P-value N/A
At 24 months (95% CI)
28.4%
(21.2–36.0)

18.8%
(12.8–25.7)

OS overall patient populationb
HR (95% CI) 0.64
(0.46­4–0.870)
P-value
P=0.0021c

At 24 months (95% CI)
71.3%
(64.5–77.1)

56.0%
(48.9–62.5)
OS dMMR/MSI-H populationa,d
HR (95% CI)
0.30
(0.127–0.699)

P-value N/A
At 24 months (95% CI)
83.3%
(66.8–92.0)

58.7%
(43.4–71.2)

OS MMRp/MSS populationa,e
HR (95% CI) 0.73
(0.515–1.024)
P-value N/A
At 24 months (95% CI)
67.7%
(59.8–74.4)

55.1%
(46.8–62.5)

aExploratory analyses of PFS in MMRp/MSS, OS in dMMR/MSI-H, and OS in MMRp/MSS populations were pre-specified with no planned hypothesis testing. bMaturity ≈33%. cP-value of ≤0.00177 was required for statistical significance at this OS interim analysis. dMaturity ≈26%. eMaturity ≈36%.

The safety and tolerability profile of dostarlimab in combination with carboplatin-paclitaxel in the RUBY phase III trial was generally consistent with the known safety profiles of the individual agents. The most common (>45%) treatment-emergent adverse events (TEAEs) in both treatment arms in the dMMR/MSI-H and overall populations were nausea, alopecia and fatigue, as well as anaemia in the placebo plus chemotherapy arm in the dMMR/MSI-H population. Severe and serious TEAEs were approximately 10% higher in the dostarlimab plus carboplatin-paclitaxel arm compared with the placebo plus carboplatin-paclitaxel arm in the overall population. The nature and types of immune-related adverse events (irAEs) in the dostarlimab plus chemotherapy safety profile were consistent with the mechanism of action of dostarlimab and similar to those reported for other PD-(L)1 inhibitors. In the overall population, 38.2% of participants in the dostarlimab plus carboplatin-paclitaxel arm and 15.4% of participants in the placebo plus carboplatin-paclitaxel arm had irAEs assessed by the investigator as related to dostarlimab or placebo, respectively. The most frequently reported dostarlimab-related irAE categories were endocrinopathies (15.8% dostarlimab-related versus 3.3% placebo-related) and skin adverse reactions (14.1% dostarlimab-related versus 3.7% placebo-related). Discontinuation of dostarlimab or placebo due to a TEAE occurred in 17.4% of patients in the dostarlimab plus chemotherapy treatment arm and 9.3% of patients in the placebo plus chemotherapy treatment arm in the overall population.

These data were shared in a European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Plenary, presented in a late breaking session at the Society of Gynecologic Oncology (SGO) Annual Meeting on Women’s Cancer (25-28 March) and published simultaneously in The New England Journal of Medicine.

RUBY is part of an international collaboration between the European Network of Gynaecological Oncological Trial groups (ENGOT), a research network of the European Society of Gynaecological Oncology (ESGO) that consists of 22 trial groups from 31 European countries that perform cooperative clinical trials; the GOG Foundation, a non-profit organisation dedicated to transforming the standard of care in gynaecologic oncology; and the Nordic Society of Gynaecological Oncology – Clinical Trial Unit (NSGO-CTU), a non-profit organisation aiming to improve the practice of prevention, diagnosis and treatment for gynaecological cancers by supporting research and conducting clinical trials across countries.

GSK’s ambition is for dostarlimab to become the backbone of the Company’s ongoing immuno-oncology-based research and development programme when used alone and in combination with standard of care and future novel cancer therapies, particularly for patients who currently have limited treatment options. Dostarlimab is being investigated in registrational enabling studies as monotherapy and as part of combination regimens, including in patients with primary advanced or recurrent endometrial cancer, patients with Stage III or IV non-mucinous epithelial ovarian cancer, and patients with other advanced solid tumours or metastatic cancers.

About endometrial cancer
Endometrial cancer is found in the inner lining of the uterus, known as the endometrium. Endometrial cancer is the most common gynaecologic cancer in developed countries, with approximately 417,000 new cases reported each year worldwide[i], and incidence rates are expected to rise by almost 40% by 2040.[ii][iii] Approximately 15-20% of patients with endometrial cancer will be diagnosed with advanced disease at the time of diagnosis.[iv]

About RUBY
RUBY is a two-part global, randomised, double-blind, multicentre phase III trial of patients with primary advanced or recurrent endometrial cancer. Part 1 is evaluating dostarlimab plus carboplatin-paclitaxel followed by dostarlimab versus carboplatin-paclitaxel plus placebo followed by placebo. Part 2 is evaluating dostarlimab plus carboplatin-paclitaxel followed by dostarlimab plus niraparib versus placebo plus carboplatin-paclitaxel followed by placebo. The primary endpoints in Part 1 are investigator-assessed PFS based on the Response Evaluation Criteria in Solid Tumours v1.1 and OS. The statistical analysis plan included pre-specified analyses of PFS in the dMMR/MSI-H and ITT populations and OS in the overall population. Pre-specified exploratory analyses of PFS in the MMRp/MSS population and OS in the dMMR/MSI-H populations were also performed. RUBY Part 1 included a broad population, including histologies often excluded from clinical trials and had approximately 10% of patients with carcinosarcoma and 20% with serous carcinoma. In Part 2, the primary endpoint is investigator-assessed PFS. Secondary endpoints in Part 1 and Part 2 include PFS per blinded independent central review, overall response rate, duration of response, disease control rate, patient-reported outcomes, and safety and tolerability.

About Jemperli (dostarlimab)
Jemperli is a programmed death receptor-1 (PD-1)-blocking antibody that binds to the PD-1 receptor and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2.[v]

Jemperli is not approved anywhere in the world for use in combination with standard-of-care chemotherapy (carboplatin-paclitaxel) followed by dostarlimab for primary advanced or recurrent endometrial cancer. In the US, Jemperli is indicated for adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. Jemperli is also indicated in the US for patients with dMMR recurrent or advanced solid tumours, as determined by a US FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. The latter indication is approved in the US under accelerated approval based on tumour response rate and durability of response. Continued approval for this indication in solid tumours may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Jemperli was discovered by AnaptysBio, Inc. and licensed to TESARO, Inc., under a collaboration and exclusive license agreement signed in March 2014. The collaboration has resulted in three monospecific antibody therapies that have progressed into the clinic. These are: Jemperli (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialisation, and manufacturing of each of these medicines under the agreement.

Important Information for Jemperli in the EU

Indication 
Dostarlimab is indicated as monotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum-containing regimen. 

Refer to the Jemperli EMA Reference Information for a full list of adverse events and the complete important safety information in the EU.

On March 27, 2023 Genmab A/S (Nasdaq: GMAB) reported the initiation of a share buy-back program to honor our commitments under our Restricted Stock Units program (Press release, Genmab, MAR 27, 2023, View Source [SID1234629372]).

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The share buy-back program is expected to be completed no later than March 31, 2023, and comprises up to 220,000 shares.

The following transactions were executed under the program from March 20, 2023, to March 24, 2023:

No. of shares Average price (DKK) Total value (DKK)
Accumulated through last announcement 167,000 427,631,620
March 20, 2023 6,000 2,504.24 15,025,440
March 21, 2023 5,000 2,541.77 12,708,850
March 22,2023 5,000 2,541.44 12,707,200
March 23, 2023 7,000 2,529.99 17,709,930
March 24, 2023 7,000 2,564.91 17,954,370

Total 30,000 76,105,790
Accumulated under the program 197,000 503,737,410
Details of each transaction are included as an appendix to this announcement.

Following these transactions, Genmab holds 740,416 shares as treasury shares, corresponding to 1.12% of the total share capital and voting rights.

The share buy-back program is undertaken in accordance with Regulation (EU) No. 596/2014 (‘MAR’) and the Commission Delegated Regulation (EU) 2016/1052, also referred to as the "Safe Harbour Regulation." Further details on the terms of the share buy-back program can be found in our company announcement no. 06 dated February 22, 2023.

On March 27, 2023 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated, localized biologics, reported full year 2022 financial results and provided a business update (Press release, CytomX Therapeutics, MAR 27, 2023, View Source [SID1234629369]).

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"2022 was an important year of transition for CytomX as we proactively restructured our organization to maximize long-term success and value creation. We entered 2023 with considerable momentum across our therapeutic pipeline including our first Probody T-cell bispecific in the clinic and preparing for two new, wholly-owned IND filings anticipated this year. We continue to optimize and leverage our leading Probody platform to create high impact therapeutic candidates with the potential to maximize overall benefit for patients. With our recently announced collaborations with Moderna and Regeneron, we are further extending the reach of our science, broadening our pipeline, and bringing important non-dilutive capital into the company. These accomplishments underscore our leadership in conditionally activated, localized biologic therapies and we look forward to a year of strong execution in 2023 that will also include a reassessment of our CX-2029 and CD71 strategy following AbbVie’s decision not to further advance the program," said Sean McCarthy, D.Phil., chief executive officer and chairman of CytomX Therapeutics.

Fourth Quarter Business Highlights and Recent Developments

CX-904, T-cell-engaging bispecific (TCB) EGFRxCD3, Phase 1 clinical study ongoing – CX-904 is a conditionally activated TCB designed to target the epidermal growth factor receptor (EGFR) on cancer cells and the CD3 receptor on T cells within the tumor microenvironment. CX-904 is being evaluated by CytomX in an ongoing Phase 1 study in patients with advanced solid tumors. The first patient was dosed in May 2022 and the dose escalation portion of the study continues to advance and is now in the 3+3 stage having successfully completed initial single patient cohorts. The continued progression of CX-904 is a key priority for the company in 2023 with the primary goal of assessing safety and determining a recommended dose, or doses, for subsequent expansions in select tumor types. This program is partnered with Amgen in a global co-development alliance with CytomX retaining the right to opt into a profit share in the U.S.

IND filings anticipated in 2H 2023 for Wholly Owned CX-801, Interferon (IFN) alpha-2b, and CX-2051, EpCAM-directed ADC – For CytomX’s next generation molecules, the company has selected the previously validated anti-cancer targets, EpCAM and IFNa2b, respectively, that have been limited in their potential due to systemic toxicities. In the molecular design of CX-2051 and CX-801, we have incorporated our platform expertise and clinical learnings to optimize predicted therapeutic index in order to potentially broaden the clinical utility of these promising agents through localization to the tumor microenvironment. CytomX anticipates filing INDs for both programs in the second half of 2023.

BMS advancement of BMS-986288 to Phase 2 – In February 2023, BMS published pipeline updates that included moving the Anti-CTLA-4 non-fucosylated Probody, BMS-986288, from Phase 1 to Phase 2. BMS prioritized the BMS-986288 Probody program over the other two molecules in its CTLA-4 pipeline – the Probody, BMS-986249, and the antibody, BMS-986218.

CX-2029: Updated Phase 2 data and next steps – CX-2029 is a conditionally activated ADC directed toward CD71, the transferrin receptor. In January 2023, updated Phase 2 results were announced from the cohort expansion study which included squamous esophageal cancer, squamous non-small cell lung cancer (sqNSCLC), and head and neck squamous cell carcinoma (HNSCC). The Phase 2 data demonstrated encouraging clinical activity in unselected, heavily pre-treated patients with tumors of squamous histology including a 21% objective response rate (ORR) in squamous esophageal cancer and a 10% ORR in squamous non-small cell lung cancer (sqNSCLC). The adverse event (AE) profile was consistent with Phase 1 observations with anemia (82.6% all grade, 76.1% grade 3+) being the most common treatment related adverse event (TRAE). On March 21, 2023, AbbVie notified the company that it would not advance CX-2029 into additional clinical studies and provided notice of termination of the 2016 CD71 License and Collaboration Agreement. As a result of the termination, CytomX will regain full rights to CD71 and has an exclusive option to re-acquire full rights to CX-2029. CytomX will evaluate potential next steps for CX-2029 as well as pursue next-generation strategies for targeting CD71. CytomX and AbbVie have also concluded their research activities under a 2016 Discovery License and Collaboration Agreement.

Clinical candidate milestone achievement in Astellas TCB collaboration – In January 2023, Astellas nominated a collaboration clinical candidate, the first Probody TCB molecule to progress in the alliance, triggering a $5 million dollar milestone payment to CytomX. CytomX and Astellas are collaborating on additional conditionally activated TCB programs, and CytomX is eligible to receive future preclinical, clinical, and commercial milestones. CytomX retains a cost share and co-commercialization option on a select number of targets.

New strategic research collaboration with Moderna – In December 2022, CytomX entered a collaboration and licensing agreement to create investigational mRNA-based conditionally activated therapies utilizing Moderna’s mRNA technologies and CytomX’s Probody therapeutic platform. The research collaboration will leverage core scientific advances at Moderna and CytomX with the strategy of encoding potent, masked biologics with mRNA, for the potential treatment of oncology and non-oncology conditions. CytomX received an upfront payment of $35 million, including $5 million of pre-paid research funding, and is eligible to receive up to approximately $1.2 billion in future development, regulatory, and commercial milestone payments, along with tiered royalties on global net sales of any products that are commercialized under the agreement. Moderna has the option to participate in a future CytomX equity financing, subject to certain terms, conditions and regulatory requirements.

New strategic research collaboration with Regeneron – In November 2022, CytomX entered its collaboration with Regeneron to enable the development of investigational next-generation bispecific immunotherapies using CytomX’s Probody and Regeneron’s Veloci-Bi platforms. The Probody platform has the potential to widen the therapeutic window and minimize on-target, off-tumor effects for these next-generation T-cell engaging therapies, potentially addressing tumor types that have historically been unresponsive to immunotherapy. CytomX received a $30 million upfront payment in December 2022 and is eligible for up to approximately $2 billion in research, development, regulatory and sales-based milestones.
Priorities for 2023

CX-904 (EGFRxCD3): Continue patient enrollment and dose escalation in ongoing Phase 1 study
File 2 New INDs (wholly-owned): CX-801 (IFNa2b) and CX-2051 (EpCAM) projected in the second half of 2023
CX-2029 (CD71 ADC): Determine next steps for CD71 program, including CX-2029
Next-Generation CTLA-4 Program: Continued clinical progress for BMS-986288
Collaborations: Initiation of R&D activities with our newest collaborators, Regeneron and Moderna, and ongoing activities with Astellas, Amgen and BMS
Full Year 2022 Financial Results

Cash, cash equivalents and investments totaled $194 million as of December 31, 2022, compared to $305 million as of December 31, 2021 and $194 million as of September 30, 2022. The cash balance at December 31, 2022 includes the $30 million upfront payment received under the Regeneron agreement, but excludes the $35 million cash payment received under the Moderna agreement in the first quarter of 2023.

Total revenue was $53.2 million for the year ended December 31, 2022, compared to $37.3 million in 2021. The increase in revenue was driven by higher estimated percentages of completion for the research and development programs in the company’s collaborations with AbbVie, Astellas and Bristol Myers Squibb, partially offset by decreased revenue under the Amgen Agreement driven by a lower estimated percentage of completion for the CX-904 program due to an increase in the projected hours-to-completion.

Research and development expenses decreased by $2.5 million during year ended December 31, 2022, to $111.6 million compared to $114.2 million in 2021. The decrease in research and development expenses was driven by a decrease in clinical trial and lab contract services for CX-2009, CX-072, CX-2029, CX-904 and pre-clinical programs, offset by $5.3 million of restructuring expenses.

General and administrative expenses increased by $3.6 million during the year ended December 31, 2022 to $42.8 million compared to $39.2 million in 2021 primarily driven by $2.4 million of restructuring expenses and a $1.0 million increase in professional expenses related to new collaboration agreements.

Overall expenses related to the company restructuring announced in July 2022 were $7.7 million consisting primarily of employee-related expenses and severance benefits. The restructuring is substantially complete as of December 31, 2022.

On March 27, 2023, CytomX Therapeutics, Inc. filed an amended 2021 Annual Report on Form 10-K/A which included restated financial statements for the years ended December 31, 2019, 2020, and 2021 and the quarterly periods for 2020 and 2021. CytomX’s 2022 Annual Report on Form 10-K includes restated interim information for the 2022 quarterly periods. Please refer to the 2021 Form 10-K/A and 2022 Form 10-K for a full description of the restatement and the corresponding financials.

The financial results contained in this press release reflect the restated financial statements in CytomX’s most recent SEC filings.

Conference Call & Webcast
CytomX management will host a conference call and simultaneous webcast today at 5 p.m. ET (2 p.m. PT) to discuss the financial results and provide a business update. Participants may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at View Source Participants may register for the conference call here and are advised to do so at least 10 minutes prior to joining the call. An archived replay of the webcast will be available on the Company’s website.

On March 27, 2023 Exicure, Inc. (Nasdaq: XCUR), historically an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets, reported financial results for the year ended December 31, 2022 and provided an update on its business strategy and corporate progress (Press release, Exicure, MAR 27, 2023, View Source [SID1234629362]).

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Corporate Update

As previously reported, in September 2022, we announced a significant reduction in force, suspension of preclinical activities and halting of all research and development, and that we were exploring strategic alternatives to maximize stockholder value. With respect to our historical assets, this includes continuing to explore out-licensing opportunities for cavrotolimod, our clinical-stage asset in immuno-oncology, as well as for our preclinical candidate associated with the SCN9A program for neuropathic pain.

While the foregoing efforts with respect to our historical assets are continuing, we do not expect they will generate significant value for stockholders, at least in the near term. Therefore, we are engaging in a broader exploration of strategic alternatives. This effort involves exploring growth through transactions with potential partners that see an opportunity in joining an existing, publicly-traded organization. We are exploring transactions both within our historical biotechnology and life science industry, as well as in other industries unrelated to our historical operations.

On February 24, 2023, we closed our private placement (the "Private Placement") to CBI USA, Inc. Following the closing of the Private Placement, CBI USA is the beneficial owner of approximately 50.4% of the Company’s outstanding shares. At closing, CBI USA designated three members to the Company’s board of directors effective as of February 24, 2023. Additional directors were subsequently appointed by the board, and our board of directors currently includes 6 members, only one of which (Matthias Schroff, Exicure’s Chief Executive Officer) was a director prior to the closing of the Private Placement. The Company is currently relying on Nasdaq’s "controlled company" exemption from the requirements that a majority of its board be independent and that it has an independent compensation committee and an independent nominating committee or function.

The Company currently expects to focus its efforts on the following:

•Continue to implement its previously announced restructuring plan and efforts to maximize stockholder value that can be derived from historical biotechnology assets. The Company expects to evaluate on an ongoing basis whether the resources dedicated to these activities are sustainable and commensurate with the potential value that can be derived from them.

•Explore growth through transactions with potential partners that see opportunity in joining an existing, publicly-traded organization. The board of directors will consider any promising transactions that it believes can create value for stockholders. We are exploring transactions both within our historical biotechnology and life science industry and in other industries unrelated to our historical operations. The Company expects these efforts may be focused in Asia where CBI USA’s affiliates have relationships and business connections, although domestic transactions are also being considered. Transactions that may be explored could include reverse mergers or share

exchanges, as well as acquisitions of other businesses or investments. There can be no assurance that any agreement, arrangement or understanding with respect to such a transaction will be reached, or the potential structure or financial and other terms of any agreement, arrangement or understanding that may be reached.

•Seek additional financing for the Company as needed to support these activities. Without a current source of revenue or committed financing, the Company believes that it will be necessary to obtain substantial additional financing in the next few months in order to provide sufficient runway to continue operating and pursue these activities. There can be no assurance that such financing, or financing in sufficient amounts or on acceptable terms, will be received.

"On behalf of the board of directors, I look forward to working with Exicure and CBI USA, now the majority controlling shareholder, to continue to explore strategic transactions and alternatives to maximize stockholder value," said Seung Soo Shin, Chairman of the Board of Directors of Exicure.

2022 Financial Results

Cash Position: Cash, cash equivalents, and restricted cash were $9.8 million as of December 31, 2022. Subsequent to December 31, 2022, the Company raised gross proceeds of $5.4 million on the closing of the Private Placement (or net proceeds of approximately $4.6 million after transaction expenses) and the Company expects to use the net proceeds for general working capital purposes as it pursues strategic alternatives as well as for the payout for warrant put rights that were exercised as a result of the change of control. The Company believes that its existing cash and cash equivalents (including the proceeds received in February 2023 in connection with the closing of the Private Placement) could enable the Company to fund its operating expenses into the beginning of the fourth quarter of 2023. However, this estimate is based on assumptions about how the Company can limit spending that may prove to be wrong and it is very difficult to project the Company’s current cash burn rate given the transitional status of the Company as it explores strategic alternatives and this estimate may prove inaccurate and the Company may expend its limited resources sooner.

Revenue: Revenue was $28.8 million for the year ended December 31, 2022, reflecting an increase of $29.3 million from revenue of $(0.5) million for the year ended December 31, 2021. The increase in collaboration revenue of $29.3 million is due to the recognition of the remaining deferred revenue related to the AbbVie Collaboration Agreement of $13.9 million and the Ipsen Collaboration Agreement of $15.4 million in connection with the terminations of those collaboration agreements in December 2022. This revenue resulted from an accounting adjustment, did not reflect any new cash proceeds to the Company and will not recur. Following these terminations, the Company currently has no source of revenues.

Research and Development (R&D) Expense: Research and development expense was $19.8 million for the year ended December 31, 2022, reflecting a decrease of $29.2 million, or 60%, from research and development expense of $49.0 million for the year ended December 31, 2021. The decrease in research and development expense for the year ended December 31, 2022 of $29.2 million reflects fewer clinical, preclinical, and discovery program activities and a reduction in headcount resulting from the restructuring activities that were announced in December 2021 and September 2022.

General and Administrative (G&A) Expense: General and administrative expense was $10.9 million for the year ended December 31, 2022, representing a decrease of $2.2 million, or 17%, from $13.1 million for the year ended December 31, 2021. The decrease for the year ended December 31, 2022 is mostly due to lower compensation and related costs in connection with a lower headcount during the period resulting from the restructuring activities that were announced in December 2021, as well as lower costs for accounting, director fees, and investor relations. These lower costs in the current year period were partially offset by higher retention award expense, as well as higher consultant and advisory costs.

Net Loss: The Company had a net loss of $2.6 million for the year ended December 31, 2022, as compared to a net loss of $64.1 million for the year ended December 31, 2021. The decrease in net loss was primarily driven by higher non-cash revenue and lower R&D expense during the period.

Going Concern: Given the Company’s current cash position, operating plans and forecasted negative cash flows from operating activities over the next twelve months, management believes there is substantial doubt regarding the Company’s ability to continue as a going concern within one year after the date that its consolidated financial statements for the year ended December 31, 2022 are issued. As a result, substantial additional financing will be needed by the Company within the next few months to pay its expenses, fund its ongoing exploration of strategic alternatives and pursue any alternatives that it identifies.

On March 27, 2023 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Delcath Systems, MAR 27, 2023, View Source [SID1234629361]).

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Recent Business Highlights

During and since the fourth quarter, Delcath Systems, Inc. (Delcath or the Company):

Received an acceptance of the NDA resubmission from the U.S. Food and Drug Administration (FDA) for Hepzato Kit (melphalan hydrochloride for injection/Hepatic Delivery System) with a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2023,

Priced a financing that is expected to provide an initial upfront funding of $25 million, with up to an additional $60 million tied to satisfaction of milestones, in gross proceeds to Delcath through a private placement. The financing was led by Vivo Capital with participation from Logos Capital, BVF Partners LP, Stonepine Capital Management, LLC, Serrado Capital LLC and existing investor, Rosalind Advisors,

Completed in December a private placement with existing investors priced at market for a total of $11.2 million funds raised in 2022,

Announced the rotation of its Board of Directors with John R. Sylvester appointed as Chairman, and

Reached terms of settlement to end its dispute with medac, its former distributor in Europe.
In addition, during and since the fourth quarter, independent investigators published:

Updated results from the CHOPIN phase 1B trial in which seven patients with advanced uveal melanoma treated with CHEMOSAT and ipilimumab plus nivolumab show a median PFS of 29.1 months at a median follow-up of 29.1 months, and

Results of a Single Center Study in the treatment of Cholangiocarcinoma (CCA) in which the authors concluded that percutaneous hepatic perfusion (PHP) with CHEMOSAT is an effective and safe treatment option for patients with advanced CCA and has the potential to prolong life in patients with inoperable, treatment-refractory liver metastases. The authors highlighted the increasing importance of locoregional forms of therapy in the treatment of CCA and that the new edition of the German S3 cancer guideline "Diagnostics and Therapy of Hepatocellular Carcinoma and Biliary Carcinomas" now includes PHP with melphalan for the treatment of inoperable iCCA or eCCA liver metastases.
"With the FDA setting an August 14, 2023, PDUFA date we have crossed a significant milestone for the Company." said Gerard Michel, Chief Executive Officer of Delcath. Mr. Michel added, "We are gratified with the support from both our existing investors and our new investors, all of whom are highly regarded healthcare-focused funds. Their support, potentially totaling up to $85 million, subject to satisfaction of milestones, we believe validates the clinical relevance of and the commercial opportunity for Hepzato in metastatic ocular melanoma. Further, it positions Delcath to execute on HEPZATO commercialization plans upon potential FDA approval. Finally, we eagerly await the publication of interim results from the phase II portion of the CHOPIN study which should provide critical additional information about the potential utility of CHEMOSAT used in sequence with immune checkpoint inhibitors."

Income Statement Highlights.

Fourth Quarter 2022 Results

Product revenue for the three months ended December 31, 2022 was approximately $0.6 million, compared to $0.2 million for the prior year period, from our sales of CHEMOSAT in Europe. This increase in product revenue is primarily due to direct product sales for the fourth quarter of 2022 compared to the revenue share arrangement with our distribution partner in Europe during the fourth quarter of 2021. Other income for the three months ended December 31, 2022 was $1.9 million due to the acceleration of deferred revenue caused by the termination of the medac license agreement.

Research and development expenses for the three months ended December 31, 2022 were $4.4 million, compared to $3.6 million in the prior year quarter. The growth in R&D expense is primarily due to increased activity related to the expenses incurred for the preparation of our NDA resubmission which occurred on February 14, 2023. Selling, general and administrative expenses for the three months ended December 31, 2022 were approximately $3.8 million, compared to $3.0 million in the prior year quarter. The increase in general and administrative expenses was primarily due to higher headcount related costs such as higher share-based compensation expense.

The Company recorded a net loss for the three months ended December 31, 2022 of $8.5 million, $0.86 per share (basic and diluted), compared to a net loss of $5.3 million, $0.69 per share (basic and diluted), for the same period in 2021.

Full Year 2022 Results

Product revenue for the year ended December 31, 2022 was approximately $2.5 million, compared to $1.3 million for the prior year from sales of CHEMOSAT in Europe. Other income for the year ended December 31, 2022 was $0.2 million compared to $2.3 million in the prior year primarily due to the termination of the medac license agreement in December last year.

Research and development expenses for the year ended December 31, 2022 were $18.6 million compared to $13.8 million in the prior year with the increase due to preparation for the pre-NDA meeting in April 2022 and expenses related to the NDA resubmission. Selling, general and administrative expenses for the year ended December 31, 2022 were approximately $17.3 million compared to $13.6 million in the prior year with the increase primarily due to the pending launch of HEPZATO in the United States and the accrual for the settlement of the medac litigation.

The Company recorded a net loss for the year ended December 31, 2022, of $36.5 million, $4.12 per share (basic and diluted), compared to a net loss of $25.6 million $3.59 per share (basic and diluted) for the year ended December 31, 2021.

Balance Sheet Highlights

On December 31, 2022, the Company had cash, cash equivalents and restricted cash totaling $11.8 million, as compared to cash, cash equivalents and restricted cash totaling $26.9 million on December 31, 2021. During the years ended December 31, 2022, and December 31, 2021, the Company used $25.0 million and $22.6 million, respectively, of cash in our operating activities. The use of cash in operating activities was partially offset by two private placements during 2022 resulting in net proceeds of $10.9 million. On March 15, 2023, the Company returned to Avenue Venture Opportunity Fund L.P. the $4.0 million held in the restricted cash to paydown a portion of the outstanding loan balance.

On December 13, 2022, the Company closed a private placement for the issuance and sale of 1,448,889 shares of common stock and 692,042 pre-funded warrants to purchase common stock to certain investors. Each share of common stock was sold at a price per share of $2.90 and the pre-funded warrants were sold at a price of $2.89 per pre-funded warrant. The pre-funded warrants have an exercise price of $0.01 per share of common stock and are immediately exercisable. The Company received gross proceeds from the private placement of approximately $6.2 million before deducting offering expenses.

Conference Call Information

Delcath will host a conference call today, on March 27, 2023, at 4:30 PM Eastern Time to discuss results for its fourth quarter and full year ended December 31, 2022 and provide a business update.

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

Event Date: Monday March 27, 2023

Time: 4:30 PM Eastern Time

Participant Numbers: Toll Free: 1-833-630-1960
International: 1-412-317-1841
Webcast: View Source

CONFERENCE REPLAY

US Toll Free: 1-877-344-7529

International Toll: 1-412-317-0088

Replay Access Code: 7305121

End Date: April 03, 2023