On November 4, 2021 Akoya Biosciences Inc., (Nasdaq: AKYA) The Spatial Biology Company, reported that new data generated with its CODEX and Phenoptics platforms will be presented at the 35th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), taking place November 10-14 virtually and at the Walter E. Washington Convention Center in Washington, DC (Press release, Akoya Biosciences, NOV 4, 2021, View Source [SID1234594572]).

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In recent years, immunotherapy, which utilizes the patient’s immune system to fight cancer, has significantly advanced the care of cancer patients. However, it has delivered durable benefits only to some subsets of people with advanced disease, creating an urgent need for accurate, predictive biomarkers to stratify responders and non-responders.

Recent studies have demonstrated that spatial phenotypic signatures offer higher predictive power than traditional immunohistochemistry and genomic biomarkers because they preserve the spatial context of tumor samples and measure cellular proximity and interactions in the tumor microenvironment. Akoya’s CODEX and Phenoptics Solutions for discovery, translational and clinical research can image multiple biomarkers across whole tumor sections at single-cell and sub-cellular resolution, allowing researchers to discover novel signatures to predict immunotherapy response.

A sponsored dinner symposium entitled "Leading and Managing Spatial Biomarker Innovations in Immuno-Oncology: Multistakeholder Perspectives," will feature experts from a pharmaceutical company, an academic medical center, and a clinical research organization (CRO), who will discuss how they are using spatial biomarkers to transform oncology drug development and testing. The speakers are: Qingyan (Sandy) Au, PhD, principal scientist, director of multiplexing operations, NeoGenomics; Michael Surace, PhD, associate director, AstraZeneca; and Houssein A. Sater, MD, Lead Physician Scientist, Hematology Oncology, Cleveland Clinic Martin Health. The dinner will take place on Thursday, November 11, at the Marriott Marquis (next to the Convention Center).

The company and its collaborators will also highlight novel applications and data through the following poster presentations:

P#49: Highly Multiplexed Detection of Critical Immune Checkpoints and Immune Cell Subtypes in Cancerous FFPE Tissues Using CODEX.
P#51: A Novel Cross-Site Analysis of Vectra Polaris Multiplex Fluorescence PD-1/PD-L1 Immunohistochemistry on Colorectal Cancer with High and Low Microsatellite Instability.
P#309: Visualizing the Immunotherapy-Induced Spatial Reorganization of the Tumor-Immune Microenvironment by CODEX Multiplex Imaging.
P#937: Advanced Understanding of the Tumor Microenvironment with Multiplex Analysis: An Automated 7-color Multiplex Assay Using Akoya’s Opal Technology.
In addition, conference attendees can get hands-on experience with the CODEX and Phenoptics systems by visiting the Akoya booth (#4). In-booth presentations will take place on Friday, November 12 and on Saturday, November 13 covering the following topics:

Visiopharm: Analysis of a Spatial Signature Data Set
Expanding into Multiplex Immunofluorescence: Complementing Your Research
Paving the Path for Spatial Biomarker Discovery
Brian McKelligon, Akoya’s Chief Executive Officer, commented: "We at Akoya look forward to SITC (Free SITC Whitepaper) 2021, where we will demonstrate how complete workflow solutions like CODEX and Phenoptics can simplify the discovery and translation of spatial biomarkers into advanced clinical tools, leading to more optimal cancer treatments that produce better outcomes for greater numbers of patients."

For more information about Akoya’s activities at SITC (Free SITC Whitepaper) 2021, please visit akoyabio.com/sitc2021.

On November 4, 2021 Merus N.V. (Nasdaq: MRUS) ("Merus", the "Company," "we" and "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported the pricing of an underwritten public offering of 3,859,650 common shares, at a public offering price of $28.50 per share (the "Offer Shares") (Press release, Merus, NOV 4, 2021, View Source [SID1234594603]). Merus also granted the underwriters a 30-day option to purchase up to an additional 578,947 common shares (the "Option Shares" and together with the Offer Shares, the "Shares"). The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses and excluding the underwriters’ option to purchase the Option Shares, are approximately $110.0 million. All of the shares in the offering are to be sold by Merus.

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The offering is expected to close on or about November 9, 2021, subject to customary closing conditions.

Merus intends to use the net proceeds from the offering to advance the clinical development of its product candidates, for preclinical research and technology development, and for working capital and general corporate purposes.

Jefferies LLC and SVB Leerink LLC are acting as joint book-running managers for the offering. Kempen & Co is acting as lead manager for the offering. H.C. Wainwright & Co. and Roth Capital Partners are acting as co-managers for the offering.

The offering is being made pursuant to a shelf registration statement on Form S-3 that was filed with the Securities and Exchange Commission (SEC) on May 7, 2021 and was effective upon filing. The offering will be made only by means of a written prospectus and prospectus supplement that form a part of the registration statement, which, for the avoidance of doubt, will not constitute a "prospectus" for the purposes of (i) Regulation (EU) 2017/1129 (the "Prospectus Regulation") and has not been reviewed by any competent authority in any member state in the European Economic Area (the "EEA") and (ii) the Prospectus Regulation as it forms part of domestic law in the United Kingdom by virtue of the European Union (Withdrawal) Act 2018 (the "UK Prospectus Regulation") and has not been reviewed by the Financial Conduct Authority in the United Kingdom. A preliminary prospectus supplement to the prospectus describing the terms of the offering was filed with the SEC on November 4, 2021, and a final prospectus supplement will be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained, when available, from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by phone at (877) 821-7388, or by email at [email protected] or SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected].

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

This press release is an advertisement and not a prospectus within the meaning of either the Prospectus Regulation or the UK Prospectus Regulation.

EEA:

In relation to each member state of the EEA (each, a "Relevant State"), no Shares have been offered or will be offered pursuant to the offering to the public in that Relevant State, other than:

to any legal entity which is a qualified investor as defined under Article 2 of the Prospectus Regulation;
to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the Prospectus Regulation), subject to obtaining the prior consent of the underwriters for any such offer; and
in any other circumstances falling within Article 1(4) of the Prospectus Regulation,
provided that no such offer of the Company’s Shares shall require us or any of our representatives to publish a prospectus pursuant to Article 3 of the Prospectus Regulation or supplement a prospectus pursuant to Article 23 of the Prospectus Regulation and each person who initially acquires any Shares or to whom any offer is made will be deemed to have represented, acknowledged and agreed to and with each of the representatives and us that it is a "qualified investor" as defined in the Prospectus Regulation.

For the purposes of the above, the expression an "offer of shares to the public" in relation to any Shares in any Relevant State means the communication in any form and by means of sufficient information on the terms of the offer and the Shares to be offered so as to enable an investor to decide to purchase Shares.

United Kingdom:

No Shares have been offered or will be offered pursuant to this offering to the public in the United Kingdom prior to the publication of a prospectus in relation to the Shares which has been approved by the Financial Conduct Authority, except that the Shares may be offered to the public in the United Kingdom at any time:

a) to any legal entity which is a qualified investor as defined under Article 2 of the UK Prospectus Regulation;
b) to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the UK Prospectus Regulation), subject to obtaining the prior consent of the representatives for any such offer; or
c) in any other circumstances falling within Section 86 of the Financial Services and Markets Act 2000 (the "FSMA")

provided that no such offer of the Shares shall require us or any of our representatives to publish a prospectus pursuant to Section 85 of the FSMA or supplement a prospectus pursuant to Article 23 of the UK Prospectus Regulation.

For the purposes of this provision, the expression an "offer to the public" in relation to the Shares in the United Kingdom means the communication in any form and by any means of sufficient information on the terms of the offer and any Shares to be offered so as to enable an investor to decide to purchase or subscribe for any Shares.

In addition, in the United Kingdom, the transaction to which this press release relates will only be available to, and will be engaged in only with persons who are "qualified investors" (as defined in the UK Prospectus Regulation) (i) who have professional experience in matters relating to investments falling within Article 19(5) of the FSMA (Financial Promotion) Order 2005, as amended (the Order), and/or (ii) who are high net worth entities (or persons to whom it may otherwise be lawfully communicated) falling within Article 49(2)(a) to (d) of the Order (all such persons together being referred to as "relevant persons"). In the United Kingdom, the securities referred to herein are only available to, and any invitation, offer or agreement to subscribe, purchase or otherwise acquire such securities will be engaged in only with relevant persons. Any person in the United Kingdom who is not a relevant person should not act or rely on this communication or any of its contents.

On November 4, 2021 Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure Hepatitis Delta Virus (HDV) and other serious rare diseases, reported its third quarter 2021 financial results and provided a business update (Press release, Eiger Biopharmaceuticals, NOV 4, 2021, View Source [SID1234594620]).

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"The recent completion of enrollment in our Phase 3 HDV D-LIVR study of Lonafarnib-based regimens sets up pivotal topline data by the end of 2022," said David Cory, President and CEO. "Additionally, our Phase 3 HDV LIMT-2 study of Peginterferon Lambda is now activating sites and screening patients. Eiger is focused on the development of treatments and a cure for HDV. We are positioned to be a leader in this space with two first-in-class therapies for HDV, offering hope for the over 12 million patients around the globe with this devastating disease."

Program Updates and Upcoming Milestones

HDV Platform

Lonafarnib for HDV

First-in-class prenylation inhibitor and only oral agent in development
D-LIVR, largest Phase 3 global study conducted in HDV
Fully enrolled with over 400 patients
Opportunity for approval of two Lonafarnib-based regimens:
All-oral and combination with peginterferon alfa
Pivotal topline data planned by end of 2022
Peginterferon Lambda for HDV

Well-tolerated interferon administered as a weekly subcutaneous injection
LIMT-2 (N=150), pivotal study of Peginterferon Lambda monotherapy
Now activating sites and screening patients
Avexitide for Rare Metabolic Disorders

Phase 3 studies for post-bariatric hypoglycemia and congenital hyperinsulinism could begin as early as 2022
Zokinvy for Progeria and Processing-Deficient Progeroid Laminopathies

MAA is under EMA review, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected around end of 2021
Cohort ATU program (Temporary Use Authorization) approved in France
First ATU shipment completed
Peginterferon Lambda for COVID-19

Phase 3 TOGETHER study enrolling patients across clinical sites in Brazil
DSMB interim futility analysis (n=453) recommended study continuation
Next interim futility data analysis by end of 2021
Positive data could support emergency use authorization package
Corporate

Appointed Erik Atkisson General Counsel and Chief Compliance Officer
Cash and investments of $120.4 million at the end of third quarter 2021 expected to fund planned operations into fourth quarter 2023
Third Quarter Financial Results

Net revenues from Zokinvy product sales were $3.0 million for third quarter 2021, as compared to $2.1 million for second quarter 2021. The increase was primarily driven by modestly higher inventory on-hand at the specialty pharmacy. The company commercially launched Zokinvy in the U.S. in January 2021 and has reported September year-to-date net sales of $8.8 million.

Cost of Sales were $0.3 million for third quarter 2021 and is related to certain costs associated with Zokinvy that were incurred after FDA approval.

Research and Development expenses were $18.1 million for third quarter 2021, as compared to $9.8 million for the same period in 2020. The increase was primarily due to clinical trial related expenses, including contract manufacturing and headcount related expenses, including stock-based compensation expense.

Selling, General and Administrative expenses were $6.5 million for the third quarter of 2021, as compared to $5.0 million for the same period in 2020. The increase was primarily due to outside consulting and advisory services and headcount related expenses, including stock-based compensation expense.

Total operating expenses include non-cash expenses of $3.0 million for the third quarter of 2021, as compared to $1.9 million for the same period in 2020.

Eiger reported a third quarter 2021 net loss of $22.2 million, or $0.65 on a per share basis. This compares to a net loss of $15.7 million, or $0.52 on a per share basis, for the third quarter of 2020.

Cash, cash equivalents, and investments as of September 30, 2021, totaled $120.4 million compared to $139.8 million as of June 30, 2021.

As of September 30, 2021, the company had 33,975,800 common shares outstanding.

Conference Call
At 4:30 PM Eastern Time today, November 4, 2021, Eiger will host a conference call to discuss its financial results and provide a business update. The live and replayed webcast of the call will be available through the company’s website at www.eigerbio.com. To participate in the live call by phone, dial (844) 743-2495 (U.S.) or (661) 378-9529 (International) and enter conference ID 9874006. The webcast will be archived and available for replay for at least 90 days after the event.

On November 4, 2021 Galapagos NV (Euronext & NASDAQ: GLPG) reported on its commercial launch of filgotinib in Europe (Press release, Galapagos, NOV 4, 2021, View Source [SID1234594786]). The company is moving forward with its revised R&D strategy and operational restructuring announced in May, resulting in a downward adjustment of the cash burn by €50 million. The unaudited Q3 financial and operational results are further detailed in the Q3 2021 report available on the website, www.glpg.com.

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"This quarter we achieved key steps in our growing commercial business in Europe, while moving earlier-stage R&D programs forward. We continue to deliver on our revised strategy, accelerating the savings program announced at the first quarter results. We are focused on nominating a successor to lead our company going forward following my eventual retirement as CEO," said Onno van de Stolpe, CEO of Galapagos. "Supported by our strong balance sheet and long-term R&D collaboration with Gilead, we believe that Galapagos remains well-positioned for future growth."

"After years of hard work by so many, we are very excited to bring Jyseleca to market as a new treatment option for people living with rheumatoid arthritis (RA). As per 30 September 2021, we booked €6.1 million in net sales for Jyseleca, for a total of €15.8 million together with Gilead. Encouraged by these sales of our first commercial product and its positioning in the growing JAK market in Europe, we are confident in the commercial potential of our Jyseleca franchise in Europe. Following the positive CHMP opinion for filgotinib for the treatment of patients with ulcerative colitis (UC), we expect a decision by the European Commission (EC) before year-end, and if granted, we are ready to go full steam ahead with the commercial roll-out in a second indication," added Bart Filius, President and COO of Galapagos. "Following our strategic operational review in March 2021, we implemented a cost savings program of €150 million on a full year basis. As a result of an acceleration of this program, we revise our guidance for full year 2021 operational cash burni from €580 to €620 million to €530 to €570 million."

Details of the financial results

Due to the sale of our fee-for-service business (Fidelta) to Selvita on 4 January 2021 for a total consideration of €37.1 million (including customary adjustments for net cash and working capital), the results of Fidelta are presented as "Net profit from discontinued operations" in our unaudited condensed consolidated income statements for the nine months ended 30 September 2021 and 30 September 2020.

Revenues from continuing operations

Our revenues from continuing operations for the first nine months of 2021 amounted to €317.9 million compared to €321.9 million in the first nine months of 2020.

We reported net sales of Jyseleca for the first nine months of 2021 amounting to €6.1 million (€5.7 million in the third quarter of 2021), which reflects the sales booked by Galapagos after the transition from Gilead. Total sales of Jyseleca in Europe by both companies for the first nine months of 2021 is €15.8 million.

2

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Collaboration revenues amounted to €311.7 million for the first nine months of 2021, compared to €321.9 million for the same period last year. This was mainly driven by the recognition of upfront consideration and milestone payments received in the scope of the collaboration with Gilead for filgotinib, amounting to €136.4 million for the first nine months of 2021 (€145.9 million for the same period last year). The decrease in revenue recognition was primarily due to a negative cumulative catch up of revenue triggered by the recent agreement under which Galapagos will assume operational and financial responsibility for the ongoing DIVERSITY clinical study. This decrease was partly compensated by additional consideration from Gilead related to the renegotiated collaboration, when compared to the same period last year. The revenue recognition related to the exclusive access rights for Gilead to our drug discovery platform amounted to €173.3 million for the first nine months of 2021 (€170.7 million for the same period last year).

Our deferred income balance on 30 September 2021 includes €1.8 billion allocated to our drug discovery platform that is recognized linearly over 10 years, and €0.7 billion allocated to the filgotinib development that is recognized over time until the end of the development period.

Results from continuing operations

We realized a net loss from continuing operations of €141.8 million for the first nine months of 2021, compared to a net loss of €251.8 million for the first nine months of 2020.

We reported an operating loss amounting to €175.7 million for the first nine months of 2021, compared to an operating loss of €167.7 million for the same period last year.

Cost of sales related to Jyseleca net sales in the first nine months of 2021 amounted to €0.7 million.

Our R&D expenditure in the first nine months of 2021 amounted to €378.0 million, compared to €392.2 million for the first nine months of 2020. This decrease was primarily explained by winding down of our ziritaxestat (IPF), MOR106 (atopic dermatitis), and GLPG1972 (OA) programs and by reduced spend on our other programs. This was partly offset by costs increases for our filgotinib and Toledo (SIKi) programs, on a nine months comparison basis. Personnel costs increased primarily because of an increased average headcount compared to the same period last year, and increased costs of our subscription right plans.

Our S&M and G&A expenses were respectively €46.6 million and €104.7 million in the first nine months of 2021, compared to respectively €44.1 million and €88.3 million in the first nine months of 2020. This increase was primarily due to an increase in personnel costs and other operating expenses mainly driven by the commercial launch of filgotinib in Europe. This increase was partly compensated by higher cost recharges from us to Gilead in the scope of our commercial cost sharing for filgotinib in Europe.

Other income (€36.3 million vs €35.0 million for the same period last year) increased, mainly driven by higher grant income.

We reported a non-cash fair value gain from the re-measurement of initial warrant B issued to Gilead, amounting to €3.0 million, mainly due to the decreased implied volatility of the Galapagos share price and its evolution between 31 December 2020 and 30 September 2021.

Net other financial income in the first nine months of 2021 amounted to €30.6 million, compared to net other financial loss of €75.3 million for the first nine months of 2020, which was primarily attributable to €54.9 million of currency exchange gain on our cash and cash equivalents and current financial investments in U.S. dollars, to €10.1 million of negative changes in (fair) value of current financial investments and financial assets and to €8.5 million of interest expenses. The other financial expenses also contained the effect of discounting our long term deferred income of €7.2 million.

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Results from discontinued operations

The net profit from discontinued operations for the nine months ended 30 September 2021 consisted of the gain on the sale of Fidelta, our fee-for-services business, for €22.2 million.

Group net results

We reported a group net loss for the first nine months of 2021 of €119.6 million, compared to a group net loss of €247.6 million for the first nine months of 2020.

Cash position

Current financial investments and cash and cash equivalents totaled €4,874.2 million on 30 September 2021, as compared to €5,169.3 million on 31 December 2020.

Total net decrease in cash and cash equivalents and current financial investments amounted to €295.2 million during the first nine months of 2021, compared to a net decrease of €472.2 million during the first nine months of 2020. This net decrease was composed of (i) €376.7 million of operational cash burn, (ii) offset by €2.7 million of cash proceeds from capital and share premium increase from exercise of subscription rights in the first nine months of 2021, (iii) €7.2 million negative changes in (fair) value of current financial investments and €57.3 million of mainly positive exchange rate differences, (iv) €28.7 million cash in from disposal of subsidiaries, net of cash disposed.

Our balance sheet on 30 September 2021 also held a receivable from the French government (Crédit d’Impôt Rechercheiv) and a receivable from the Belgian Government for R&D incentives, for a total of both receivables of €149.3 million.

Outlook 2021

Going forward, we continue to build our filgotinib franchise throughout Europe, and remain on track to complete the transition of the full European commercial operations for filgotinib from our collaboration partner Gilead to us by year-end. We anticipate an approval decision from the EC and Great Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) for filgotinib for the treatment of UC, which, if approved, would add a second indication to our growing commercial footprint in Europe.

Following the positive topline Phase 1b data from our TYK2 inhibitor GLPG3667, we are running an extended dose escalation study in healthy volunteers, and we are preparing to launch a Phase 2b trial in Psoriasis and a Phase 2 trial in UC in 2022.

We are advancing our SIK3 inhibitor GLPG4399 in healthy volunteers this year, and we aim to move a follow-up SIK2/3 preclinical candidate into the clinic in 2022.

By year-end we also intend to finalize recruitment into the GLPG2737 Phase 2a trial in autosomal dominant polycystic kidney disease (ADPKD), an indication with important unmet medical need.

Meanwhile we continue to apply lessons learned from the strategic exercise announced at Q1 to the development of our deep pipeline, and we diligently evaluate business development opportunities in our core therapeutic areas of inflammation and fibrosis.

Following our strategic review of operations in March 2021, we implemented a cost savings program of €150 million on a full year basis. As a result of an acceleration of this program, we revise our guidance for full year 2021 operational cash burn from €580 to €620 million to €530 to €570 million.

Third quarter report 2021

Galapagos’ financial report for the first nine months ended 30 September 2021, including details of the unaudited consolidated results, is accessible via www.glpg.com/financial-reports.

Conference call and webcast presentation

Galapagos will conduct a conference call open to the public tomorrow, 5 November 2021, at 13:00 CET / 8 AM ET, which will also be webcasted. To participate in the conference call, please call one of the following numbers ten minutes prior to commencement:

A question and answer session will follow the presentation of the results. Go to www.glpg.com to access the live audio webcast. The archived webcast will also be available for replay shortly after the close of the call.

Financial calendar

24 February 2022 Full year 2021 results (webcast 25 February 2022)

On November 4, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company using its ARCUS genome editing platform to develop allogeneic CAR T and in vivo gene editing therapies, reported that investigators involved with the Phase 1/2a study of PBCAR0191 in Relapsed/Refractory (R/R) non-Hodgkin’s lymphoma (NHL) and B-cell acute lymphoblastic leukemia (B-ALL), will present new data during two oral presentations at the 63rd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) taking place December 11-14, 2021 (Press release, Precision Biosciences, NOV 4, 2021, View Source [SID1234594365]).

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"We are encouraged by the response rates seen in this heavily pre-treated patient population, and that a treatment strategy with enhanced lymphodepletion mitigated PBCAR0191 rejection and improved peak CAR T cell expansion and persistence, compared to standard lymphodepletion, with predictable toxicity," said Alan List, MD, Chief Medical Officer of Precision BioSciences. "We look forward to sharing additional patient outcome, durability, and safety data for PBCAR0191 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting."

The abstracts accepted by the ASH (Free ASH Whitepaper) are now available at www.hematology.org, and will be presented during the following oral presentation sessions:

Session Name: 626, Abstract #302. Aggressive Lymphomas Prospective Therapeutic Trials: Challenging Populations
Oral Presentation Title: Allogeneic CAR-T PBCAR0191 with Intensified Lymphodepletion is Highly Active in Subjects with Relapsed/Refractory B-cell Malignancies
Presenting Author: Bijal Shah, M.D., Moffitt Cancer Center
Date/Time: Saturday, December 11, 2021 at 4:15 PM ET
Location: Georgia World Congress Center, B401-B402

Session Name: 704, Abstract #650 Cellular Immunotherapies: Allogeneic CARs and CARs for T Cell Lymphomas
Oral Presentation Title: Preliminary Safety and Efficacy of PBCAR0191, an Allogeneic ‘Off-the-Shelf’ CD19-Directed CAR-T for Patients with Relapsed/Refractory (R/R) CD19+ B-ALL
Presenting Author: Nitin Jain, M.D., The University of Texas MD Anderson Cancer Center
Date/Time: Monday, December 13, 2021 at 10:45 AM ET
Location: Georgia World Congress Center, Sidney Marcus Auditorium

Published abstracts report on key interim clinical evaluations of CD19+ NHL or B-ALL subjects treated with PBCAR0191.

Abstract #302: For 21 subjects with Relapsed/Refractory (R/R) B-cell malignancies (16 NHL, 5 B-ALL) who received PBCAR0191 following enhanced lymphodepletion1 as of July 1, 2021:

PBCAR0191 demonstrated a safety profile with no Grade 3 CRS, one Grade 3 self-limited ICANS, no evidence of GvHD, and one infectious death at Day 54, deemed possibly related to treatment.
83% (15/18) of evaluable subjects experienced a complete response (CR) rate or complete remission with incomplete marrow recovery (CRi); 62% (8/13) of NHL subjects and 80% (4/5) of B-ALL subjects, respectively.
20% (3/15) of responders demonstrated durability of response greater than 6 months, with 3 additional responders not yet having reached a 6-month evaluation threshold.
Compared to standard lymphodepletion2, enhanced lymphodepletion mitigated PBCAR0191 rejection to markedly improve peak CAR T cell expansion and persistence with area under the curve increasing 80-fold.
Among 6 subjects who progressed following prior CD19 CAR therapy (5 NHL, 1 B-ALL), the overall response rate was 83% (5/6) with 67% (4/6) achieving a CR, including an ongoing MRD negative CR in a B-ALL subject of >6 months.
Abstract #650: For 15 subjects with R/R B-cell acute lymphoblastic leukemia including 11 subjects who received PBCAR0191 Dose Level 3/4a3 and 4 subjects who received PBCAR0191 Dose Level 4b4 as of August 2, 2021:

PBCAR0191 demonstrated a safety profile with no cases of GvHD, no Grade ≥3 CRS, and one case of Grade 3 ICANS, which resolved within 48 hours.
For subjects who received either Dose Level 3/4a following enhanced lymphodepletion or Dose Level 4b following standard lymphodepletion, 78% (7/9) achieved a high CR or CRi rate; 56% (5/9) maintained the CR at day 28 or later potentially securing an adequate window to bridge to allogeneic stem cell transplant.
Use of enhanced lymphodepletion or higher doses of PBCAR0191 resulted in substantial improvements in peak CAR T cell expansion and area under the curve.