On February 23, 2023 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year of 2022 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 23, 2023, View Source [SID1234627594]).

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"The past year for Aclaris was marked by tremendous execution across our organization notably highlighted by our clinical development programs and our proprietary kinase-focused drug discovery platform," stated Doug Manion, M.D., Chief Executive Officer of Aclaris. "We enter 2023 well positioned to capitalize on that momentum with multiple data read-outs expected, beginning with top line data from the Phase 2a trial of zunsemetinib in hidradenitis suppurativa next month."

Continued Dr. Manion, "In addition to the enthusiasm related to our upcoming development-stage milestones, we also continue to benefit from the output of our KINect discovery engine, and the ability to identify novel development candidates, which we believe will not only position us to continue to build long-term shareholder value, but also enable us to further fulfill our mission of delivering new therapeutic options for patients."

Research and Development Highlights:

Clinical Development Programs:

• Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
Currently being developed as a potential treatment for immuno-inflammatory diseases

Rheumatoid Arthritis (ATI-450-RA-202): This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe rheumatoid arthritis (RA) is ongoing. Aclaris expects topline data in the second half of 2023.

Hidradenitis Suppurativa (ATI-450-HS-201): This Phase 2a trial to investigate the efficacy, safety, tolerability, PK and PD of zunsemetinib (50 mg twice daily) over 12 weeks in subjects with moderate to severe hidradenitis suppurativa (HS) has completed enrollment with 95 patients randomized and is ongoing. Aclaris expects topline data in March of 2023.

Psoriatic Arthritis (ATI-450-PsA-201): This Phase 2a trial to investigate the efficacy, safety, tolerability, PK and PD of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis (PsA) is ongoing. Aclaris expects topline data by year end 2023.

• ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developing as a potential treatment for moderate to severe atopic dermatitis (AD)

Atopic Dermatitis (ATI-1777-AD-202): This Phase 2b trial to determine the efficacy, safety, tolerability, and PK of multiple doses and application regimens of ATI-1777 in subjects with moderate to severe AD is ongoing. Aclaris expects topline data mid-year 2023.
• ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor:
Currently being developed as a potential treatment for T cell-mediated autoimmune diseases

Aclaris has selected ulcerative colitis as the intended first clinical development target for ATI-2138. Aclaris is also exploring additional indications that are relevant to the mechanism of action.

Aclaris initiated a Phase 1 MAD (multiple ascending dose) trial of ATI-2138 in healthy volunteers in December of 2022. Aclaris expects topline data from the MAD trial in the second half of 2023.
Preclinical Development Program

• ATI-2231, an investigational oral MK2 inhibitor compound:
Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer

Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long plasma half-life.

Aclaris expects clinical development activities to be initiated in 2023, which is expected to advance as a collaboration with an academic third party.
Financial Highlights:

Liquidity and Capital Resources

As of December 31, 2022, Aclaris had aggregate cash, cash equivalents and marketable securities of $229.8 million compared to $225.7 million as of December 31, 2021. Aggregate cash, cash equivalents and marketable securities as of December 31, 2022 included proceeds received during the fourth quarter under a license agreement with Pediatrix Therapeutics, Inc. (Pediatrix).

Aclaris continues to anticipate that its cash, cash equivalents and marketable securities as of December 31, 2022 will be sufficient to fund its operations through the end of 2025, without giving effect to any potential business development transactions or financing activities.

Financial Results

Fourth Quarter 2022

Net loss was $27.6 million for the fourth quarter of 2022 compared to $22.8 million for the fourth quarter of 2021.

Total revenue was $7.8 million for the fourth quarter of 2022 compared to $1.5 million for the fourth quarter of 2021. The increase was driven by $6.7 million of licensing revenue in the quarter, including $5 million from the license agreement with Pediatrix.

Research and development (R&D) expenses were $21.1 million for the quarter ended December 31, 2022 compared to $14.1 million for the prior year period.
The $7.0 million increase was primarily the result of higher:
Zunsemetinib development expenses, including costs associated with clinical activities for a Phase 2b trial for RA, a Phase 2a trial for HS, and a Phase 2a trial for PsA.
ATI-1777 development expenses related to drug candidate manufacturing and other preclinical activities and costs associated with a Phase 2b clinical trial for AD.
ATI-2138 development expenses, including costs associated with a Phase 1 SAD trial, a Phase 1 MAD trial, and other preclinical activities.
Compensation-related expenses due to an increase in headcount.
General and administrative (G&A) expenses were $7.1 million for the quarter ended December 31, 2022 compared to $6.9 million for the prior year period.

Licensing expenses were $0.6 million for the quarter ended December 31, 2022 resulting primarily from obligations to the former Confluence equity holders from revenue generated from the Pediatrix license agreement. There were no licensing expenses for the quarter ended December 31, 2021.

Revaluation of contingent consideration was $7.1 million for the quarter ended December 31, 2022, compared to a revaluation of contingent consideration expense of $2.2 million for the prior year period.
Full Year 2022

Net loss was $86.9 million for the year ended December 31, 2022 compared to $90.9 million for the year ended December 31, 2021.

Total revenue was $29.8 million for the year ended December 31, 2022 compared to $6.8 million for the year ended December 31, 2021.
The $23.0 million increase was driven by a $24.3 million increase in licensing revenue during 2022, which included $17.6 million of upfront and milestone payments received under the non-exclusive patent license agreement with Eli Lilly and a $5.0 million upfront payment received under the Pediatrix license agreement. The increase in licensing revenue was offset by a decrease in contract research revenues.
R&D expenses were $77.8 million for the year ended December 31, 2022 compared to $43.8 million for the prior year period.
The $34.0 million increase was primarily the result of higher:
Zunsemetinib development expenses, including costs associated with clinical activities for a Phase 2b trial for RA, a Phase 2a trial for HS, and a Phase 2a trial for PsA.
ATI-1777 development expenses related to drug candidate manufacturing and other preclinical activities and costs associated with a Phase 2b clinical trial for AD.
ATI-2138 development expenses, including costs associated with a Phase 1 SAD trial, a Phase 1 MAD trial, and other preclinical activities.
ATI-2231 preclinical development expenses associated with preclinical activities and IND-enabling studies.
Compensation-related expenses due to an increase in headcount.

Licensing expenses were $7.9 million for the year ended December 31, 2022 resulting from separate third-party contractual obligations primarily related to the non-exclusive patent license agreement with Eli Lilly. There were no licensing expenses for the year ended December 31, 2021.

G&A expenses were $25.1 million for the year ended December 31, 2022 compared to $23.6 million for the prior year period.
The $1.5 million increase was primarily the result of higher stock-based compensation and personnel costs due to an increase in headcount.
Revaluation of contingent consideration charges related to the Confluence acquisition was $4.7 million for the year ended December 31, 2022 compared to $24.3 million for the prior year period.

On February 23, 2023 AbbVie (NYSE: ABBV) and Capsida Biotherapeutics Inc. ("Capsida") reported an expanded strategic collaboration to develop genetic medicines for eye diseases with high unmet need (Press release, AbbVie, FEB 23, 2023, View Source [SID1234627593]). AbbVie’s extensive capabilities will be paired with Capsida’s novel adeno-associated virus (AAV) engineering platform and manufacturing capability to identify and advance three programs. The collaboration builds upon the neurodegenerative disease partnership announced in 2021.

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"This expanded collaboration with Capsida has the potential to develop transformative therapies for patients with serious eye diseases," said Jonathon Sedgwick, Ph.D., vice president and global head of discovery research, AbbVie. "In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner."

"AbbVie has been an excellent partner, and we are excited to expand our collaboration into ophthalmology with the world leader in this therapeutic area," said Peter Anastasiou, chief executive officer of Capsida. "Combining AbbVie’s expertise in eye disease drug development and commercialization with Capsida’s fully integrated next-generation AAV engineering platform and manufacturing capabilities offers the potential to provide novel therapies enabling unprecedented benefit to patients with serious eye diseases."

Under the terms of the expanded agreement, Capsida will receive $70 million, consisting of upfront payments and a potential equity investment. For the three programs, Capsida may be eligible to receive up to $595 million in option fees and research and development milestones, with potential for further commercial milestones. Capsida is also eligible to receive mid-to-high single-digit royalty payments on future product sales. Capsida will lead capsid discovery efforts for all programs using its high throughput AAV engineering platform and will be responsible for process development and early clinical manufacturing. AbbVie will lead innovative therapeutic cargo approaches and be responsible for development and commercialization.

On February 22, 2023 Redx Pharma (AIM: REDX) and Jounce Therapeutics, Inc. (Nasdaq: JNCE) reported an unanimously recommended Business Combination of the two companies via a proposed all share merger transaction (Press release, Jounce Therapeutics, FEB 23, 2023, View Source [SID1234627546]). Redx is a clinical-stage biotechnology company focused on the discovery and development of novel, small molecule, targeted therapeutics for the treatment of cancer and fibrotic diseases and the emerging area of cancer-associated fibrosis. Jounce is a clinical-stage immunotherapy company, dedicated to transforming the treatment of cancer by developing therapies that enable the immune system to attack tumors and provide long-lasting benefits to patients through a biomarker approach. The Business Combination will create a transatlantic organization with proven expertise in both small molecule drugs and biologics, and a clinical pipeline with multiple value inflection points in the near and medium term.

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The combined group’s highest priority will be the development of RXC007, a next-generation selective ROCK2 inhibitor, which is currently being assessed in a Phase 2a study in idiopathic pulmonary fibrosis (IPF), with topline data expected in Q1 2024. ROCK2 inhibition is now a commercially validated target with potential in multiple disease areas, following the recent US FDA approval and launch of the first drug with this mechanism of action. In addition to the ongoing clinical development plan in IPF, Redx has also generated supportive preclinical data that highlights the broad potential of next-generation ROCK2 inhibitors across a number of fibrotic indications where there remains a significant unmet need, which supports potential development opportunities in other interstitial lung diseases and cancer-associated fibrosis. Additionally, RXC004 is being developed as a targeted treatment for Wnt-ligand dependent cancers and is progressing through Phase 2 trials, and RXC008, a GI-targeted ROCK inhibitor for fibrostenotic Crohn’s disease, is expected to enter clinical development in H1 2024.

Lisa Anson, Chief Executive Officer, Redx Pharma commented: "By combining Redx’s proven track record in small molecule drug discovery and development with Jounce’s expertise in biologics and immunotherapy, we will establish a world-class biotech company with a robust pipeline, aimed at developing therapeutics for cancer and fibrotic disease. RXC007, our next-generation ROCK2 inhibitor, will lead the clinical pipeline, and we will now further investigate opportunities for this asset in cancer-associated fibrosis and other interstitial lung diseases. While the majority of the combined group’s operations, and its headquarters, will be at Alderley Park in the UK, we believe that listing solely on Nasdaq is the most efficient way to enable us to access a deep pool of risk capital and engage with specialist global investors to support our future growth."

Richard Murray, Ph.D., Chief Executive Officer and President of Jounce Therapeutics commented: "I am very proud of the work we have achieved at Jounce and today’s news is testament to the accomplishments of our team’s efforts. We are pleased that many of our employees will join the combined group and continue to focus on bringing much-needed alternatives to patients. Additionally, we are pleased that Redx will continue to advance certain of our early discovery programs. With a focus on cancer and fibrosis, and a strong scientific basis for discovery and development programs in those areas, we believe that the new company will be well positioned to succeed."

At the time of the completion of the Business Combination, Jounce is expected to have around $155m of cash and cash equivalents, which net of any tail and closing costs results in at least $130m in cash and cash equivalents available to the combined group. Together with Redx’s expected cash at completion would provide the combined group with cash runway into H2 2025. Based on Redx’s fully diluted market capitalization of £244m ($294m) as at the Last Practicable Date and Jounce’s expected cash and cash equivalents at the time of completion, this implies a market value for the combined group of $425m, before taking into account the value of Jounce’s existing clinical and non-clinical stage programs.

This announcement follows the earlier news1 that Jounce plans to reduce its workforce by approximately 57%. After completion of the transaction, around 47 Jounce employees will be retained by the combined group at a research and development base in Massachusetts, USA and will bring complementary expertise in biologics and immuno-oncology. Jounce’s clinical programs will not be pursued in-house beyond the currently ongoing studies.

Management and Organization

Named Redx Inc., the combined group will be solely listed on Nasdaq in the US under the ticker symbol REDX. Led by current Redx CEO Lisa Anson, Dr Jane Griffiths current Redx Chair will become the non-executive Chair of the combined group; with the Board including representatives from both Redx and Jounce, in line with the relative shareholding percentages. The combined group will be headquartered at Alderley Park, UK, with a drug discovery and clinical development team in Massachusetts, USA.

About the transaction

Under the terms of the Business Combination, Redx Shareholders shall be entitled to receive 0.2105 Jounce Shares in exchange for each Redx Share (the "Exchange Ratio"). Jounce intends to conduct a reverse stock split of Jounce Shares in conjunction with the Business Combination, with a ratio of one new share for every five outstanding shares of Jounce. A reverse split is a share exchange transaction, without any impact on the amount of the share capital: only the number of outstanding shares is modified. If the Reverse Stock Split is approved by Jounce Shareholders, the Exchange Ratio will be adjusted to 0.0421 Jounce Shares in exchange for each Redx Share.

Immediately following completion of the Business Combination, including conversion of the Redx Convertible Loan Notes, Redx Shareholders are expected to own approximately 63% and Jounce shareholders approximately 37% of the share capital of the combined group.

The Business Combination is expected to be implemented by way of a Scheme of Arrangement ("Scheme") of Redx under Part 26 of the UK Companies Act, immediately preceded by a merger transaction between RM Special Holdings 3, LLC, an entity controlled by Redmile, and Jounce and its affiliates (the "Redmile Merger"), which together will result in Jounce owning the entire issued and to be issued ordinary share capital of Redx. Further details of the Redmile Merger and the ability for eligible Redx Shareholders to request that their Redx Shares be transferred to Jounce via a similar merger structure are set out in the Rule 2.7 announcement.

In connection with the Business Combination, a non-transferrable Contingent Value Right ("CVR") is expected to be distributed to Jounce Shareholders that held Jounce Shares prior to completion of the Business Combination and holders of Jounce Share Awards immediately prior to completion of the Business Combination comprising vested options, relating to certain existing Jounce clinical and non-clinical stage programs. The CVRs shall entitle the relevant Jounce Shareholders to receive, on a pro rata basis, subject to certain terms and conditions, 80% of the net proceeds resulting from any sale, transfer, disposition, spin-off, or license of certain assets relating to such programs that is consummated within one year following the Business Combination, subject to one six-month extension term in certain limited circumstances, as set forth in the CVR Agreement.

The Boards of both Redx and Jounce have unanimously recommended that shareholders vote in favor of the Business Combination. In connection with the Business Combination, Jounce has received irrevocable undertakings from Redx Shareholders who together own 76.6% of Redx’s share capital as of 22 February 2023 (excluding the Redx shares held by RM3) and RM3 has entered into the Redmile Merger Agreement conditional on the Court sanctioning the Scheme and will receive the same Exchange Ratio. Both Redmile and Sofinnova have agreed to convert all the Redx Convertible Loan Notes held by them respectively as part of the transaction. Redx has also received voting and support agreements in respect of Jounce shares, representing, in aggregate, approximately 21.3% of the issued and outstanding share capital of Jounce. Full details of the undertakings and voting and support agreements are contained in the Rule 2.7 announcement.

It is expected that the Business Combination will complete during the second quarter of 2023, subject to satisfaction or waiver of the Conditions including approval of the Scheme by Redx Shareholders, Jounce Shareholder approvals, and customary regulatory approvals.

Defined terms have the meaning given to them in the Rule 2.7 announcement. Additional details on the Business Combination are available in the Rule 2.7 announcement which is available at www.redxpharma.com/investor-centre/ and in the Investors and Media section of Jounce’s website at www.jouncetx.com.

Conference Call and Webcast

Jounce and Redx will host a live conference call and webcast today at 08:00 ET/13:00 GMT. To access the conference call, please register here and please be advised to do so at least 10 minutes prior to joining the call. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com and on the investor section of Redx’s website at www.redxpharma.com/investor-centre/presentations-analyst-reports-documents-and-videos/. The webcast will be archived and made available for replay on Jounce and Redx’s websites approximately two hours after the call and will be available throughout the offer period thereafter.

On February 23, 2023 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a biopharmaceutical company focused on developing novel synthetic lethality-based cancer therapeutics targeting DNA damage response (DDR) pathways, reported the pricing of its previously announced underwritten public offering of 1,050,000 shares of its common stock at a public offering price of $5.25 per share (Press release, Aprea, FEB 23, 2023, View Source [SID1234627541]). Gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be approximately $5.5 million before deducting underwriting discounts and commissions and estimated offering expenses. In addition, Aprea has granted the underwriter a 30-day option to purchase up to an additional 157,500 shares of common stock.

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The net proceeds received from the public offering will enable the Company to continue developing its clinical asset, ATRN-119, its pre-clinical asset ATRN-W1051 and for general corporate purposes.

Maxim Group LLC is acting as sole book-running manager for the offering.

The public offering is being made pursuant to an effective shelf registration statement on Form S-3, as amended (File No. 333-250041), previously filed with the U.S. Securities and Exchange Commission (SEC) on November 12, 2020 and declared effective on November 30, 2020. The securities may be offered only by means of a prospectus. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the public offering have been filed with the SEC, will form a part of the effective registration statement and will be available on the SEC’s website at www.sec.gov. Before you invest, you should read the preliminary prospectus supplement and accompanying prospectus, together with the information incorporated therein, for more complete information about Aprea and the proposed offering. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC. When available, copies of the final prospectus supplement and accompanying prospectus relating to the public offering may also be obtained by contacting Maxim Group LLC, at 300 Park Avenue, 16th Floor, New York, NY 10022, Attention: Prospectus Department, or by telephone at (212) 895-3745 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On February 22, 2023 OSE Immunotherapeutics SA reported the online publication in the peer-reviewed ‘The Journal of Immunology’ of positive Phase 1 clinical results with OSE-127/S95011, an antiIL-7 receptor (IL-7R) antagonist, and provides an overall update on the product, which is being developed in immuno-inflammation through two ongoing Phase 2 clinical trials in Ulcerative Colitis (sponsor OSE) and in primary Sjögren’s Syndrome (sponsor Servier), and in hematology where promising preclinical data in Acute Lymphoblastic Leukemia (ALL) has already been reported (Press release, OSE Immunotherapeutics, FEB 22, 2023, View Source [SID1234646943]).

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OSE-127/S95011 is being developed in partnership with Servier* via a collaboration agreement. Two clinical studies evaluating OSE-127/S95011 are ongoing: a Phase 2a study conducted by Servier in primary Sjögren’s syndrome and a Phase 2 study conducted by OSE Immunotherapeutics in ulcerative colitis (UC).

Nicolas Poirier, Chief Executive Officer of OSE Immunotherapeutics, comments: "We are very pleased to have this peer-reviewed data on OSE-127 published in ‘The Journal of Immunology’. It recognizes the value of our Phase 1 data which marked an important step towards understanding the safety and efficacy of OSE-127. It confirms the interest in the novel and differentiated mechanism of action of the only full-antagonist of IL-7R for the treatment of chronic autoimmune diseases. OSE-127 is the only compound targeting IL-7R under clinical development in the primary Sjögren’s syndrome and ulcerative colitis.

Based on this strong scientific rationale, we are confident that the ongoing Phase 2 trials conducted by OSE in ulcerative colitis and Servier in primary Sjögren’s syndrome will start to establish OSE-127 as a potential new best-in-class treatment for these disabling chronic inflammatory diseases.

Patient enrolment is completed in the Phase 2a trial in primary Sjögren’s syndrome, and we are looking forward to the results that are expected in H1 2023.

Besides autoimmune diseases, OSE-127 has also demonstrated great therapeutic potential in Acute Lymphoblastic Leukemia (ALL), a very aggressive tumor arising from B or T cell precursors. Our collaboration with the University of Kiel aims at evaluating the therapeutic potential of OSE-127 in targeting and blocking the high and dysregulated IL-7 receptor-expression observed in more than 80% of B- or T-ALL patients. Relapse remains a clinical challenge in B-ALL in high-risk patients and treatment options for T-ALL remain very limited. Novel targeted immunotherapy approaches are urgently needed to meet these patients’ high medical need."

An article, selected as ‘Top Read’ for the March 15th issue, was published online in ‘The Journal of Immunology’ (online). The publication, entitled "First-in-Human Study in Healthy Subjects with the Non-Cytotoxic 1 Monoclonal Antibody OSE-127, a Strict Antagonist of the IL-7Rα" reports on the Phase 1 positive results. These showed a good safety and tolerability profile for OSE-127/S95011, with no signs of significant lymphopenia, cytokine release syndrome or T-cell compartment alterations. All pharmacokinetic and pharmacodynamic parameters were consistent and demonstrated a doseproportionality across the several dose-levels up to 10 mg/kg. A decreased IL-7 pathway gene signature in human peripheral blood cells has been demonstrated confirming the efficient blockade of the target.

Enrollment completed in Phase 2a clinical trial in primary Sjögren’s syndrome (Servier)
Patient enrollment in the Phase 2a clinical trial of OSE-127/S95011 in primary Sjögren’s syndrome conducted by Servier was completed in October 2022.
This international, randomized, double-blind, placebo-controlled, Phase 2a study is designed to evaluate the efficacy and tolerance of the monoclonal antibody OSE-127/S95011 in primary Sjögren’s syndrome. The multicenter study in the United States, Australia and Europe includes 48 patients. Results are expected in H1 2023.
The estimated prevalence of Sjögren’s varies from 2.5 million to 4 million patients (Sjögren’s Syndrome Foundation) in the US alone, with a worldwide estimate of up to 7.7 million in the key markets (US, France, Germany, Italy, Spain, UK and Japan) by the year 2024 (Global Data Research).
To date there is no treatment approved in altering the course of the disease for primary Sjögren’s syndrome, a disease with significant unmet medical need, and only a few molecules are being evaluated in Phase 2/3 clinical development.

Phase 2 clinical trial in ulcerative colitis (UC) with interim analysis (OSE Immunotherapeutics)
The randomized, double-blind Phase 2 clinical trial aims to assess the efficacy and safety of OSE-127/S95011 versus placebo in patients with moderate to severe active UC who have previously failed or lost response or are intolerant to previous treatment(s).

An interim futility analysis was conducted on the prespecified first 50 patients (i.e., 33% of the total patient enrollment in the study) having completed the Induction Phase. After completion of the planned safety and efficacy assessment for futility, the trial’s Independent Data Monitoring Committee (IDMC) recommended the continuation of the study.

UC is a debilitating and chronic inflammatory bowel disease which affects 3.3 million patients in US, Europe and Japan (1) representing 12.2 per 100,000 people by year (2) . Despite broad options, remission rates are only 25-30% (3) leaving most patients without satisfactory treatments.

UC is characterized by a heavy burden on patients’ lives with a strong medical need for new therapeutic options.

Positive preclinical efficacy data in B- and T-Cell Acute Lymphoblastic Leukemia (B- and T-ALL)
The research program of IL-7R antagonist OSE-127 in Acute Lymphoblastic Leukemia (ALL) is being conducted through a collaborative research program between OSE Immunotherapeutics and the University Medical Center Schleswig-Holstein in Kiel (Germany). This collaboration is using patient-derived samples and in-vivo xenograft models to evaluate the therapeutic potential of OSE-127 in targeting and blocking the high and dysregulated IL-7R-expression observed in 84% of B- or T-ALL patients.

The latest preclinical data on the use of OSE-127 for the treatment of B- and T-Cell ALL (B- and TALL) were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 11, 2022. This oral presentation has received the merit-based "Abstract Achievement Award" from the peer-review committee. The presentation, entitled "The IL7R-Antagonist OSE-127 Blocks Acute Lymphoblastic Leukemia Development Via a Dual Mode of Action", reported on the preclinical efficacy of OSE-127 in ALL and on the mechanism of action underlying its anti-leukemic efficacy:

In a large prospective ALL patient cohort, IL-7R cell positivity was detected in more than 84% of cases.
OSE-127 demonstrated preclinical in vivo efficacy as monotherapy in 96% of tested B- and T-ALL Patient Derived Xenografts (PDXs), including samples from relapse and refractory patients.

The American Cancer Society estimated that almost 6,660 new cases of ALL will have been diagnosed in the United States in 2022(4) . In Europe, 7,000 cases of ALL are diagnosed each year(5) . The number of patients in Japan was reported to be about 5,000 in a survey by the Japanese MHLW (Ministry of Health, Labour and Welfare) in 2017. The number of diagnosed incident cases of ALL in Europe, US, Japan and China is estimated to achieve 26,482 cases in 2029(6) .

A global patent protection
Given the potential of OSE-127, OSE Immunotherapeutics has strengthened its global intellectual property until 2037. This has been achieved through the grant of a large number of patents worldwide, notably in major territories including Europe, the US, China and Japan
These patents protect anti-IL-7R antagonist OSE-127 and its therapeutic applications, in particular in autoimmune and inflammatory diseases.

ABOUT OSE-127/S95011
OSE-127/S95011 is a monoclonal immunomodulatory antibody targeting the CD127 receptor, the alpha chain of the interleukin-7 receptor (IL-7R) that induces a powerful antagonist effect on effector T lymphocytes. Interleukin-7 is a cytokine which specifically regulates the tissue migration of human effector T lymphocytes. The blockage of IL-7R prevents the migration of pathogenic T lymphocytes while preserving regulator T lymphocytes which have a positive impact in autoimmune diseases.