Orca Bio to Present Positive Clinical Data on its Novel High-Precision Cell Therapies at the 65th American Society of Hematology Annual Meeting

On November 2, 2023 Orca Bio, a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders, reported that new data will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting from December 9-12 in San Diego, CA (Press release, Orca Bio, NOV 2, 2023, View Source;utm_medium=rss&utm_campaign=orca-bio-to-present-positive-clinical-data-on-its-novel-high-precision-cell-therapies-at-the-65th-american-society-of-hematology-annual-meeting [SID1234636782]). Specifically, an oral presentation will highlight the outcomes of myeloablative Orca-T, Orca Bio’s lead investigational high-precision cell therapy, in patients over the age of 55. A second oral presentation will share updated results in an expanded group of patients with haploidentical donors treated with Orca Bio’s second investigational high-precision cell therapy, Orca-Q.

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Orca Bio will also present two posters highlighting the results of patients with high-risk myelodysplastic syndrome who were treated with Orca-T, and the outcomes of patients treated with Orca-T who received reduced intensity conditioning.

The ASH (Free ASH Whitepaper) abstracts are now available at www.hematology.org. Details of the Orca Bio presentations follow:

Oral Session: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities: Optimizing Conditioning Regimens for Lymphoid Malignancies and Fanconi Anemia

Title: Optimizing Outcomes with Myeloablative Conditioning in Older Patients: Efficacy and Safety of Precision Engineered Orca-T in Patients > 55 Years Old with Hematologic Malignancies

Abstract Number: 230

Date and Time: Saturday, December 9, 2023 at 2:15 PM PT

Location: Marriott Marquis, Marriott Grand Ballroom 5-6

Oral Session: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Novel Approaches to Enhance Cellular Therapies and Immune Responses in Leukemias and Lymphomas

Title: Safety and Efficacy of Orca-Q with Haploidentical Donors for the Treatment of Advanced Hematologic Malignancies Without the Use of Post-Transplant Cyclophosphamide

Abstract Number: 773

Date and Time: Monday, December 11, 2023 at 11:30 AM PT

Location: San Diego Convention Center, Room 6CF

Poster Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies

Title: High Disease-Free Survival in Patients with High-Risk MDS Treated with Orca-T

Abstract Number: 2230

Date and Time: Saturday, December 9, 2023 at 5:30 PM PT

Location: San Diego Convention Center, Halls G-H

Poster Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution

Title: Phase 1 Trial Results for Patients with Advanced Hematologic Malignancies Undergoing Reduced Intensity Allogeneic HCT with Orca-T Donor Cell Therapy Product and Single Agent Tacrolimus

Abstract Number: 3560

Date and Time: Sunday, December 10, 2023 at 6:00 PM PT

Location: San Diego Convention Center, Halls G-H

About Orca-T
Orca-T is an investigational high-precision allogeneic cell therapy for the treatment of multiple hematological malignancies. Orca-T includes infusions containing regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca-Q
Orca-Q is an investigational high-precision allogeneic cell therapy designed to treat hematological malignancies in patients with haploidentical donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform. Orca-Q has the potential to improve patient outcomes and reduce the risks of toxicities without the use of post-transplant cyclophosphamide (PTCy) in patients unable to identify a full human leukocyte antigen (HLA) match.

NGM Bio Reports Third Quarter 2023 Financial Results and Provides Business Highlights

On November 2, 2023 NGM Biopharmaceuticals, Inc. (NGM Bio) (Nasdaq: NGM), a clinical-stage biotechnology company focused on discovering and developing transformative therapeutics for patients, reported financial results for the quarterly period ended September 30, 2023 and provided business highlights (Press release, NGM Biopharmaceuticals, NOV 2, 2023, View Source [SID1234636781]).

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"In the third quarter, we continued to advance our efforts to develop novel medicines for cancer and other grievous diseases. This includes steady progress across our clinical-stage solid tumor oncology portfolio," said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM Bio. "We also announced that comprehensive data from our completed Phase 2b 48-week ALPINE 4 trial of aldafermin in compensated cirrhosis, or F4, patients due to NASH was selected for an oral plenary presentation at the upcoming AASLD The Liver Meeting in November. We are pleased that the ALPINE 4 trial achieved its primary endpoint with positive, statistically significant results across multiple measures, demonstrating clinical impact in this very advanced, difficult-to-treat patient population. This is an important milestone as we seek to partner aldafermin for continued, late-stage development."

Key Third Quarter and Recent Highlights

Solid Tumor Oncology

•In the Phase 1/2 trial evaluating NGM707, an ILT2/ILT4 antagonist antibody product candidate, as a monotherapy and in combination with KEYTRUDA (pembrolizumab) for the treatment of patients with advanced or metastatic solid tumors, completed enrollment in the Phase 1, Part 1a (monotherapy dose escalation) cohort. Enrollment in the Phase 1, Part 1b (combination dose escalation with pembrolizumab) cohort is ongoing.
•In the Phase 1/1b trial evaluating NGM438, a LAIR1 antagonist antibody product candidate, as a monotherapy and in combination with pembrolizumab for the treatment of patients with advanced or metastatic solid tumors, completed enrollment in the Phase 1, Part 1a (monotherapy dose escalation) cohort and the Phase 1, Part 1b (combination dose escalation with pembrolizumab) cohort is nearing completion of enrollment. NGM438, alone and in combination with pembrolizumab, has been well-tolerated and there have been no dose limiting toxicities to date.
•In the Phase 1/1b trial evaluating NGM831, an ILT3 antagonist antibody product candidate, as a monotherapy and in combination with pembrolizumab for the treatment of patients with advanced or metastatic solid tumors, completed enrollment in both a Phase 1, Part 1a and a Phase 1, Part 1b cohort. NGM831, alone and in combination with pembrolizumab, has been well-tolerated and there have been no dose limiting toxicities to date.

Aldafermin

•Upcoming oral plenary presentation of data from Phase 2b ALPINE 4 trial of aldafermin in compensated cirrhosis (F4) due to NASH to be presented at AASLD The Liver Meeting taking place November 10–14, 2023, in Boston, MA.
Corporate
•Jean-Frédéric Viret, Ph.D., has been appointed as Chief Financial Officer (CFO) effective November 3, 2023. Dr. Viret brings a wealth of experience to this role, having previously served for seven years (2014–2021) as CFO of Coherus BioSciences, Inc., a commercial-stage, publicly traded, biopharmaceutical company focused on the research, development and commercialization of biosimilars and biologics to treat cancer, and more recently as CFO of Shasqi, Inc. and Blade Therapeutics, Inc., two privately held biotechnology companies focused on oncology and fibrotic disease, respectively. Earlier in his career, Dr. Viret was CFO at diaDexus, Inc., XDx, Inc. (now CareDx, Inc.) and Anesiva, Inc. and worked in a variety of finance roles at Tularik Inc. and PricewaterhouseCoopers LLP (now PwC).
Third Quarter 2023 Financial Results
•NGM Bio reported a net loss of $28.8 million for the quarter ended September 30, 2023, compared to a net loss of $47.3 million for the same period in 2022.
•Related party revenue from our collaboration with Merck Sharp & Dohme LLC, or Merck, was $0.6 million for the quarter ended September 30, 2023, compared to $7.9 million for the same period in 2022. Our related party revenue from Merck will continue to decrease in 2023 and we expect minimal funding from Merck from October 1, 2023 through March 31, 2024.
•Research and development expenses were $22.9 million for the quarter ended September 30, 2023, compared to $46.1 million for the same period in 2022.
•General and administrative expenses were $8.7 million for the quarter ended September 30, 2023, compared to $10.1 million for the same period in 2022.
•Cash, cash equivalents and short-term marketable securities were $166.0 million as of September 30, 2023, compared to $271.5 million as of December 31, 2022. NGM Bio expects its cash, cash equivalents and marketable securities will be sufficient to fund its planned operations into mid-2025. NGM Bio has based this estimate on plans and assumptions that may prove to be insufficient or inaccurate (for example, with respect to anticipated costs, timing or success of certain activities), and NGM Bio could utilize its available financial resources sooner than it currently expects.

NextCure Provides Business Update and Reports Third Quarter 2023 Financial Results

On November 2, 2023 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases reported a business update and reported third quarter 2023 financial results (Press release, NextCure, NOV 2, 2023, View Source [SID1234636780]).

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"We plan to provide updates on our clinical programs, including the Phase 1b trial of NC410 in combination with pembrolizumab in patients with solid tumors, the Phase 1b trial of NC762 targeting tumor types with high B7-H4 expression, and a Phase 1a operational update for NC525 treating patients with acute myeloid leukemia (AML) by the end of the year," said Michael Richman, NextCure’s president and chief executive officer. "Our cash position and mid-2025 runway will support advancement of our most promising programs."

Q3 2023 Business Highlights and Updates

● Phase 1b updates for NC410 (LAIR-2 Fusion) and NC762 (B7-H4 mAb) by year end.
● Operational update for NC525 (LAIR-1 mAb) Phase 1a by year end.
● Selected LNCB74 (B7-H4 ADC), the first antibody drug conjugate (ADC) candidate from our collaboration with LegoChem Biosciences, Inc. Under the terms of the collaboration, both parties equally share the costs of developing the molecules and profits on commercialized products.
● Presented data on NC605, a novel anti-Siglec-15 antibody, at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, demonstrating reduced bone loss and enhanced bone quality in mice with osteogenesis imperfecta.
● Presented data on NC181, a novel humanized antibody targeting ApoE4, at the 2023 Cambridge Health Tech Institute’s 2nd Annual Neurodegeneration Targets Conference, demonstrating amyloid clearance, prevention of amyloid deposition, plaque clearance and other important findings for the treatment of Alzheimer’s disease.
Financial Guidance

● NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditures into mid-2025.

Financial Results for Quarter Ended September 30, 2023

● Cash, cash equivalents, and marketable securities as of September 30, 2023, were $118.2 million as compared to $159.9 million as of December 31, 2022. The decrease of $41.7 million was primarily related to cash used to fund operations, and cash used to purchase fixed assets.
● Research and development expenses were $11.0 million for the quarter ended September 30, 2023, as compared to $13.5 million for the quarter ended September 30, 2022. The decrease of $2.5 million was primarily related to lower costs related to our clinical programs.
● General and administrative expenses were $4.6 million for the quarter ended September 30, 2023, as compared to $5.7 million for the quarter ended September 30, 2022. The decrease of $1.1 million was primarily related to lower payroll related costs, lower stock compensation, lower insurance costs and lower legal costs.
● Net loss was $14.3 million for the quarter ended September 30, 2023, as compared with a net loss of $18.9 million for the quarter ended September 30, 2022. Lower research and development expenses, lower general and administrative expenses and higher other income contributed to the lower net loss.

NANOBIOTIX Announces Pricing of a Capital Increase Raising Total Gross Proceeds of $55M

On November 2, 2023 NANOBIOTIX (Euronext: NANO – NASDAQ: NBTX – "Nanobiotix" or the "Company"), a late-clinical stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported the pricing of its previously announced global follow-on offering reserved to specified categories of investors, consisting of (i) an offering of 3,106,907 American Depositary Shares ("ADSs"), each representing one ordinary share, €0.03 nominal value per share (each an "Ordinary Share"), of the Company, in the United States (the "U.S. Offering") at an offering price of $5.36 per ADS, and (ii) an offering of 2,492,223 Ordinary Shares, exclusively to "qualified investors" in Europe (including France) within the meaning of Article 2(e) of Regulation (EU) 2017/1129, as amended (the "Prospectus Regulation") and certain other countries (excluding the United States and Canada) (the "European Offering") at an offering price of €5.07 per Ordinary Share (Press release, Nanobiotix, NOV 2, 2023, View Source [SID1234636779]). The U.S. Offering and the European Offering are referred to, together, as the "Global Offering."

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The offering price of €5.07 per Ordinary Share, corresponding to the offering price of $5.36 per ADS based on an exchange rate of €1.00 = $1.0568, is equal to the volume weighted average price of the Ordinary Shares on the regulated market of Euronext in Paris ("Euronext") over the last three trading sessions preceding the pricing of the Global Offering (i.e. October 30, 31 and November 1, 2023), less a discount of 15% and has been determined by the Company pursuant to the 24th and 25th resolutions of the Company’s combined shareholders’ meeting held on June 27, 2023.

Pursuant to an existing securities purchase agreement, Johnson & Johnson Innovation – JJDC, Inc. ("JJDC") is obligated to subscribe, subject to any required regulatory approvals, for $25.0 million of Ordinary Shares in the form of restricted ADSs (the "Placement Amount"), at a price per ADS equal to the offering price in the U.S. Offering in a concurrent private placement (the "Concurrent Private Placement"), exempt from the registration requirements of the Securities Act of 1933, as amended (the "Securities Act"). Pursuant to French foreign investment control rules, the Placement Amount is required to be reduced, such that JJDC will initially subscribe for 3,670,294 restricted ADSs (representing 9.99% of the outstanding voting rights of the Company’s capital stock (the "Regulatory Cap")) for $19.7 million. Upon, and subject to, the approval of the French Ministry of Economy, JJDC will subscribe for additional restricted ADSs (corresponding to the portion of the Placement Amount in excess of the Regulatory Cap) for $5.3 million. The closing of the Global Offering is not conditioned on the closing of the Concurrent Private Placement.

The aggregate gross proceeds of the Global Offering and the Concurrent Private Placement are expected to be approximately $55 million, equivalent to approximately €51.5 million, before deduction of underwriting commissions in respect of the Global Offering and estimated expenses payable by the Company, assuming no exercise of the Over-Allotment Option (as defined below) in respect of the Global Offering.

Jefferies LLC, Leerink Partners and Guggenheim are acting as global coordinators and joint bookrunners for the Global Offering.

The Global Offering is subject to an underwriting agreement, which was entered into on November 2, 2023. The underwriting agreement does not constitute a performance guarantee (garantie de bonne fin) within the meaning of Article L. 225-145 of the French Commercial Code (Code de commerce).

Type of Offering

The 9,269,424 Ordinary Shares issued in the Global Offering and the Concurrent Private Placement (including in the form of ADSs and in the case of JJDC, restricted ADSs) are issued without preferential subscription rights for existing shareholders by way of:

a first capital increase of 5,599,130 Ordinary Shares subscribed in the Global Offering, decided on the date hereof by the Company’s Executive Board pursuant to the delegation granted to it by the Company’s combined shareholders’ meeting held on June 27, 2023 in its 24th resolution in accordance with Article L. 225-138 of the French Commercial Code (Code de commerce) reserved to specific investors meeting the criteria defined by the shareholders’ meeting in the 24th resolution – i.e., natural or legal entities (including companies), trusts and investment funds or other investment vehicles that invest on a regular basis, or have invested at least €1 million within the last 36 months, in the healthcare or biotechnology sectors; and
a second capital increase of 3,670,294 Ordinary Shares (in the form of restricted ADSs) subscribed by JJDC in the Concurrent Private Placement, decided on the date hereof by the Company’s Executive Board pursuant to the delegation granted to it by the Company’s combined shareholders’ meeting held on June 27, 2023 in its 25th resolution in accordance with Article L. 225-138 of the French Commercial Code (Code de commerce) reserved to a specific investor meeting the criteria defined by the shareholders’ meeting in the 25th resolution – i.e., an industrial company, institution or entity operating in the healthcare or biotechnology sector, either directly or through a controlled company or a company by which they are controlled, where applicable when entering into a commercial agreement, financing contract or partnership with the Company.
Expected Closing

The Global Offering and Concurrent Private Placement are expected to close on November 6, and November 9, 2023, respectively, subject to the satisfaction of customary closing conditions.

Option to Purchase Additional Shares

In connection with the Global Offering, the Company has granted the underwriters for the Global Offering a 30-day option to purchase additional ADSs on the same terms and conditions as in the Global Offering, in accordance with delegation granted by the Company’s combined shareholders’ meeting held on June 27, 2023 in its 26th resolution (the "Over-Allotment Option"). The Company will announce the exercise of the Over-Allotment Option and the number ADSs to be issued in connection therewith, if any, as soon as practicable thereafter in a subsequent press release.

Stabilization

In connection with the Global Offering, Jefferies LLC, acting as stabilization agent, may effect transactions with a view to supporting, stabilizing, or maintaining the market price of such securities at a level higher than which might otherwise prevail in the Company’s ADS market. However, there is no assurance that the stabilization agent will take any stabilization action and, if begun, such stabilization action may be ended at any time without prior notice. Any stabilization action or over-allotment shall be carried out in accordance with all applicable rules and regulations and may be undertaken on the Nasdaq Global Select Market.

Estimated Proceeds from the Global Offering and the Concurrent Private Placement

The aggregate gross proceeds from the Global Offering and the Concurrent Private Placement are expected to be approximately $55 million, equivalent to approximately €51.5 million, before deducting underwriting commissions in respect of the Global Offering and estimated offering expenses payable by the Company, assuming no exercise of the Over-Allotment Option in connection with the Global Offering. If the Company issues additional ADSs pursuant to the exercise in full of the Over-Allotment Option in connection with the Global Offering, the estimated gross proceeds received by the Company from the Global Offering and the Concurrent Private Placement would be expected to be approximately $59.5 million, equivalent to approximately €56.3 million, before deducting underwriting commissions in respect of the Global Offering and estimated offering expenses payable by the Company.

The Company intends to use the net proceeds from the Global Offering, together with the proceeds from the Concurrent Private Placement, as follows:

approximately 32% of the net proceeds to advance the Company’s NANORAY-312 global randomized Phase 3 clinical trial in the United States and the EU for the treatment of locally advanced head and neck cancers, and approximately 17% of the proceeds for the related increase of production capacity and supply of NBTXR3 required for this study;
approximately 27% to advance the research and development of the Company’s other preclinical and clinical programs and related regulatory and medical activities; and
approximately 24% to other operating expenses funding and other general corporate purposes, including financing expenses (including a payment to the EIB).
The expected use of proceeds represents the Company’s intentions based upon its current plans and business conditions. The Company cannot predict with certainty all of the particular uses for the net proceeds to be received upon the completion of Global Offering and Concurrent Private Placement or the amounts that the Company will actually spend on the uses set forth above. The amounts and timing of the Company’s actual expenditures and the extent of clinical development may vary significantly depending on numerous factors, including the progress of the development efforts, the status of and results from preclinical studies and any ongoing clinical trials or clinical trials the Company may commence in the future, as well as any collaborations that the Company may enter into with third parties for its product candidates and any unforeseen cash needs. As a result, the Company’s management will retain broad discretion over the allocation of the net proceeds.

As of September 30, 2023, the Company had cash and cash equivalents of €38.7 million (unaudited). The Company believes that the net proceeds from the Global Offering and the Concurrent Private Placement, together with its cash and cash equivalents, will be sufficient to meet its working capital requirements for operations until the end of the first quarter 2025, and, assuming the receipt from Janssen Pharmaceutica NV (‘‘Janssen’’) of the first milestone payment under the Company’s License Agreement with Janssen dated July 7, 2023, until the end of the second quarter 2025.

The Company’s estimates of the period of time through which its financial resources are expected to be adequate to meet its working capital requirements are forward-looking statements and involve risks and uncertainties, and actual results could vary materially and negatively as a result of a number of factors, as described under "Special Note Regarding Forward-Looking Statements."

Lock-up

In connection with the Global Offering, the Company’s executive board members and supervisory board members are subject to a contractual lock-up for a period of 90 days after the date hereof, subject to customary exceptions, including an exception for the purpose of financing the exercise price of stock options and/or satisfying any applicable taxes due in connection with such exercise. The Company has also agreed to be bound by a contractual lock-up for a period of 90 days after the date hereof, subject to customary exceptions.

Dilution

The 9,269,424Ordinary Shares (including in the form of ADSs) issued in the Global Offering, to be mainly subscribed by existing investors, and in the Concurrent Private Placement will represent dilution of approximately 20.4% of the share capital of the Company (on a non-diluted basis) (21.8%, if the Over-Allotment is exercised in full). On an illustrative basis, a shareholder holding 1% of Nanobiotix’ share capital before the Global Offering and Concurrent Private Placement would hold a stake of 0.83% after completion of the Global Offering and the Concurrent Private Placement (0.82%% if the Over-Allotment is exercised in full).

Certain entities affiliated with Invus Public Equities Advisors, LLC, Baillie Gifford & Co and Qatar Holding LLC, existing shareholders of the Company not represented at its supervisory board, have agreed to purchase an aggregate of approximately 94% of the total number of Ordinary Shares (including in the form of ADSs) to be sold in the Global Offering.

Risk Factors

Potential investors should carefully consider the risks described under "Risk Factors" in the Preliminary Prospectus Supplement, including the following risks:

shareholders not participating in the Global Offering and Concurrent Private Placement may see their participation in the Company’s share capital diluted due to the issuance of new securities;
the volatility and liquidity of the Company’s Ordinary Shares and ADSs may experience significant fluctuation (mainly downwards), and there may be differences on Nasdaq and Euronext; and
sales of the Company’s Ordinary Shares and ADSs, in particular by its significant shareholders, could occur on the market and have an adverse impact on the Company’s trading prices.
Settlement and Delivery – Documentation

The Company’s ADSs are listed on the Nasdaq Global Select Market under the ticker symbol "NBTX". The Company’s Ordinary Shares are listed on Euronext under the symbol "NANO."

The Ordinary Share are expected to be admitted to trading on Euronext on November 6, 2023 with respect to the Global Offering and on November 9, 2023 with respect to the Concurrent Private Placement.

Ordinary Shares (including those underlying ADS) issued in the Global Offering and the Concurrent Private Placement will be subject to an application for admission to trading on Euronext on the same trading line as the existing Ordinary Shares of the Company currently listed on Euronext, under the same ISIN code FR0011341205. The trading of Nanobiotix’s Ordinary Shares on Euronext is suspended today until the opening of trading of Nanobiotix’s ADSs on the Nasdaq Global Select Market at approximately 2:30 pm (Paris time) / 9:30 a.m. (New York time) today.

The ADSs and Ordinary Shares being offered in the Global Offering are being offered pursuant to an effective shelf registration statement on Form F-3 (Registration No. 333-262545), which was filed with the Securities and Exchange Commission (the "SEC") on February 4, 2022 and subsequently declared effective on February 16, 2022. The Global Offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the Global Offering has been filed with the SEC on November 1, 2023 and is available on the SEC’s website at www.sec.gov. The final prospectus supplement relating to the Global Offering will be filed with the SEC. When available, copies of the final prospectus supplement (and accompanying prospectus) relating to the Global Offering may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, or by telephone at (877) 821-7388 or by email at [email protected]; from Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525 ext. 6105, or by email at [email protected]; or from Guggenheim Securities, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

Mustang Bio Announces MB-106 CD20-Targeted CAR-T Data Selected for Presentation at 65th American Society of Hematology (ASH) Annual Meeting

On November 2, 2023 Mustang Bio, Inc. ("Mustang") (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, reported that interim Phase 1/2 data from Mustang’s multicenter clinical trial of MB-106, a CD20-targeted, autologous CAR-T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), have been selected for a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting, taking place December 9-12, 2023 in San Diego (Press release, Mustang Bio, NOV 2, 2023, View Source [SID1234636778]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center ("Fred Hutch").

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The abstract posted today on the ASH (Free ASH Whitepaper) Annual Meeting website reported on four patients who received MB-106 at dose level 1 ("DL1"), 3.3×106 cells/kg: two patients with follicular lymphoma ("FL") who achieved complete response as demonstrated by both PET-CT and bone marrow biopsy, one patient with Waldenstrom macroglobulinemia ("WM") who achieved a very good partial response ("VGPR"), and one patient with transfusion-dependent hairy cell leukemia variant ("HCL-v") who continued to have stable disease with decreased bone marrow disease and who achieved complete transfusion independence which is ongoing at 6 months. All patients displayed MB-106 expansion, with peak levels between 7-14 days post-infusion, and CAR-T cell persistence is ongoing at 6 months. From a safety perspective, 3 patients experienced Grade 1 cytokine release syndrome and no occurrences of immune effector cell-associated neurotoxicity syndrome were reported. Dose-limiting toxicities ("DLT") were monitored through day 28, and no DLTs were observed at DL1. Because of this favorable safety profile, MB-106 is infused in the outpatient setting if allowed by the institution, except for the first patient in each DL cohort, who is kept for overnight observation after MB-106 administration.

All four patients were heavily pre-treated, with a median of 5.5 prior lines of treatment. The WM patient in particular had nine prior treatments, including autologous stem cell transplant, and a high disease burden, and the patient’s VGPR was notable for complete metabolic response by PET-CT, morphologic clearance of lymphoma in bone marrow and resolution of the immunoglobulin M paraprotein. Other high-risk features were observed in each of the two FL patients: progression of disease within 24 months of first-line treatment in one patient and prior CD19-targeted CAR-T therapy in the other. Finally, the HCL-v patient received non-conforming material following FDA authorization.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We’re encouraged by the promising initial safety and efficacy data from our ongoing Phase 1/2 multicenter MB-106 clinical trial, which align with the data from the ongoing Phase 1/2 single-institution clinical trial at Fred Hutch. MB-106 continues

to demonstrate a favorable safety and efficacy profile, including complete responses from both trials in patients previously treated with CD19-targeted CAR-T cell therapy. In particular, we are excited about the very good partial response in WM, our lead indication for MB-106 and a disease where complete responses are extremely rare. We look forward to the upcoming presentation at the ASH (Free ASH Whitepaper) Annual Meeting, during which we anticipate sharing additional data from the Phase 1 indolent NHL arm, including all patients treated at the second and final dose level, 1×107 cells/kg, who have had at least 28-day follow-up. Looking beyond this meeting, we expect to treat the first patient in the pivotal Phase 2 WM trial in mid-2024, which could enable disclosure of top-line data from this trial as early as mid-2026."

Details of the presentation are as follows:

Title: Efficacy and Safety of a Third Generation CD20 CAR-T (MB-106) for Treatment of Relapsed/Refractory Indolent B-Cell Non-Hodgkin Lymphoma: Phase-1 Results from a Multicenter Trial

Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster I

Date and Time: Saturday, December 9, 2023, 5:30 pm – 7:30 pm PT

Location: San Diego Convention Center, Halls G-H

Abstract Number: 2102

Presenter: Mazyar Shadman, M.D., M.P.H., Study Chair, Innovators Network Endowed Chair at Fred Hutch, Associate Professor and physician at Fred Hutch and University of Washington

For more information, please visit the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition website at View Source