On May 2, 2019 Veracyte, Inc. (Nasdaq: VCYT) (the "Company") reported the pricing of an underwritten public offering of 5,500,000 shares of its common stock at a public offering price of $23.25 per share (Press release, Veracyte, MAY 2, 2019, View Source [SID1234535624]). The gross proceeds to the Company from this offering, before deducting underwriting discounts and commissions and offering expenses payable by Veracyte, are expected to be $127.9 million. All of the shares are being offered by the Company. The offering is expected to close on or about May 7, 2019, subject to customary closing conditions. In addition, the Company granted to the underwriters participating in the offering a 30-day option to purchase up to an additional 825,000 shares of its common stock at the public offering price, less underwriting discounts and commissions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Veracyte intends to use the net proceeds from the offering for working capital and other general corporate purposes. The Company also intends to use a portion of the net proceeds to repay approximately $12.4 million of its term loan. Veracyte may also use a portion of the net proceeds from the offering to acquire or invest in complementary businesses, technologies or other assets, although it has no present commitments or agreements to do so.

Morgan Stanley and SVB Leerink are acting as lead book-running managers for the offering. William Blair is acting as a book-running manager and BTIG and Janney Montgomery Scott are acting as co-managers.

The shares will be issued pursuant to an effective shelf registration statement filed with the Securities and Exchange Commission (SEC) on May 2, 2019. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. A copy of the final prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained by contacting Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 01220, by email at [email protected] or by telephone at (800) 808-7525, ext. 6132.

This press release does not and shall not constitute an offer to sell or the solicitation of an offer to buy any 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 registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement.

Veracyte, Afirma, Percepta, Envisia, Know by Design, the Veracyte logo and the Afirma logo are trademarks of Veracyte, Inc.

On May 2, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the first quarter ended March 31, 2019 (Press release, Agios Pharmaceuticals, MAY 2, 2019, View Source [SID1234535657]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continued to deliver significant progress across our oncology and rare genetic disease programs during the first quarter. Importantly, our U.S. commercial team is driving TIBSOVO toward solid performance for its first full year on the market, and we are on track to achieve our internal forecast," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We made progress on expansion opportunities for TIBSOVO across the frontline AML setting. FDA accepted our sNDA in newly diagnosed AML, the HOVON/AMLSG Phase 3 intensive chemotherapy combination study initiated, and we received Breakthrough Therapy Designation for the combination of TIBSOVO and azacitadine. We also advanced our mitapivat program. Our two pivotal trials in pyruvate kinase deficiency remain on track to complete enrollment this year, we have dosed the first patient in our Phase 2 thalassemia study, and we now expect a NIH-sponsored study in sickle cell disease to initiate this year."

FIRST QUARTER 2019 HIGHLIGHTS & RECENT PROGRESS

Received FDA acceptance and Priority Review for the supplemental new drug application (sNDA) for single agent TIBSOVO for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation who are not eligible for standard therapy. The Prescription Drug User Fee Act (PDUFA) action date was set for June 21, 2019.
Presented updated data from the ongoing Phase 1 combination trial of TIBSOVO with azacitidine in patients with newly diagnosed AML with an IDH1 mutation at the 17th International Symposium on Acute Leukemias.
Received Breakthrough Therapy designation from FDA for TIBSOVO in combination with azacitidine for the treatment of newly diagnosed AML patients with an IDH1 mutation who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Supported the cooperative groups HOVON and AMLSG on the initiation of the Phase 3 randomized, placebo-controlled study of TIBSOVO or IDHIFA in combination with induction therapy and consolidation therapy followed by maintenance therapy in patients with newly diagnosed AML with an IDH1 mutation.
Presented preclinical data for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting.
Dosed the first patient in a Phase 2 proof-of-concept study for mitapivat in thalassemia.
Announced two newly created commercial leadership roles to support the commercialization of the company’s medicines in the U.S. and Europe. Darrin Miles, who has been with the company since 2015, most recently as vice president, oncology program leadership, was promoted to senior vice president, U.S. commercial and global marketing. In addition, a search is underway for the role of senior vice president, international.
KEY UPCOMING MILESTONES

The company plans to achieve the following key milestones in the remainder of 2019:

Cancer:

Potential U.S. approval and launch of single agent TIBSOVO for newly diagnosed AML with an IDH1 mutation not eligible for standard therapy by June 21, 2019.
Submit an sNDA to the FDA for TIBSOVO for second line or later IDH1 mutant cholangiocarcinoma by year-end.
Initiate a registration-enabling Phase 3 study of vorasidenib in low-grade glioma with an IDH1 mutation by year-end.
Determine recommended dose of AG-270 in methylthioadenosine phosphorylase (MTAP)-deleted tumors; initiate expansion arms, including a single-agent arm in a variety of MTAP-deleted tumors and two combination arms combining AG-270 and standard-of-care in non-small cell lung cancer and pancreatic ductal adenocarcinoma in the third quarter.
Begin dosing patients in the Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma in the first half of 2019.
Rare Genetic Diseases:

Complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019:
ACTIVATE-T: A single-arm trial of up to 40 regularly transfused patients; enrollment was increased from 20 to 40 based on demand
ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions
Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
ANTICIPATED 2019 DATA PRESENTATIONS

The following abstracts have been accepted for presentation at 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting:
Updated data from a Phase 1 study of single agent TIBSOVO in IDH1 mutant newly diagnosed AML ineligible for standard therapies.
Updated data from the Phase 1 combination study of TIBSOVO and azacitidine in newly diagnosed AML with an IDH1 mutation.
Results from the first cohort in a Phase 1 perioperative study of TIBSOVO and vorasidenib in recurrent IDH1 mutant low-grade glioma.
Topline data from the Phase 3 ClarIDHy study of TIBSOVO in IDH1 mutant second line or later cholangiocarcinoma to be reported in the first half and full data to be presented in the second half of 2019.
Data from the dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors expected in the second half of 2019.
FIRST QUARTER 2019 FINANCIAL RESULTS

Revenue: Total revenue for the first quarter of 2019 was $30.2 million, which includes $17.9 million in collaboration revenue and $2.2 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene, and $9.1 million of net product revenue from U.S. sales of TIBSOVO. This compares to revenue of $8.8 million for the first quarter of 2018, which included $7.3 million in collaboration revenue and $1.4 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene.

Cost of Sales: We began U.S. sales of TIBSOVO in the third quarter of 2018. Cost of sales were $0.3 million for the first quarter of 2019.

Research and Development (R&D) Expenses: R&D expenses were $95.6 million for the first quarter of 2019 compared to $78.2 million for the first quarter of 2018. The increase in R&D expense was primarily attributable to clinical trial activity related to TIBSOVO frontline trials, the mitapivat pivotal program in PK deficiency and Phase 2 study in thalassemia, and start-up activities for AG-636.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $31.8 million for the first quarter of 2019 compared to $24.6 million for the first quarter of 2018. The increase in SG&A expense was primarily attributable to costs to support commercialization of TIBSOVO and personnel costs related to increased headcount.

Net Loss: Net loss was $93.1 million for the first quarter of 2019 compared to $90.8 million for the first quarter of 2018.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2019 were $707.8 million compared to $805.4 million as of December 31, 2018. The net decrease of $97.6 million in cash position was primarily driven by net expenditures to fund operations, including a onetime cash expense of $19.2 million for bonus payouts during the first quarter. The company expects that its cash, cash equivalents and marketable securities as of March 31, 2019, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss first quarter 2019 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 9573049. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On May 2, 2019 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2019 and provided an operating forecast and program updates (Press release, Ligand, MAY 2, 2019, View Source [SID1234535543]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The first quarter of 2019 was a great start to the year for Ligand. We saw an important new drug approved that will generate royalties for Ligand, we completed six new OmniAb and Captisol licensing deals, invested in a new technology company called Dianomi and saw our first OmniAb partnered program enter Phase 3 testing. This past quarter we divested Promacta for $827 million, converting the remaining years of royalty cash to be generated into capital today that can be reinvested into growing our business. Our partnered programs experienced significant value-enhancing events in the first quarter with a calendar for the rest of the year stacked with several substantial events," said John Higgins, Chief Executive Officer of Ligand. "At our analyst day we laid out our strategic priorities and cash investment agenda, which centers on M&A, project-financing royalty purchases, seed investments into technology companies and share repurchases. We have a highly diversified portfolio and robust financial outlook with attractive growth of top and bottom lines projected and meaningful expansion of our operating margins anticipated."

First Quarter 2019 Financial Results

Total revenues for the first quarter of 2019 were $43.5 million, compared with $56.2 million for the same period in 2018. Royalties were $19.5 million, compared with $20.8 million for the same period in 2018 and primarily consisted of royalties from Promacta, Kyprolis and EVOMELA. Royalties reflect Ligand’s sale of Promacta to Royalty Pharma as of March 6, 2019, resulting in a partial quarter of Promacta royalties, and Ligand will not receive Promacta royalties going forward. Material sales were $9.0 million, compared with $4.4 million for the same period in 2018 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $15.0 million, compared with $30.9 million for the same period in 2018, with the prior-year quarter including an upfront milestone payment upon the out-license of Ligand’s Glucagon Receptor Antagonist program to Roivant Sciences.

Cost of material sales was $3.9 million for the first quarter of 2019, compared with $0.8 million for the same period in 2018. Amortization of intangibles was $3.5 million, compared with $3.3 million for the same period in 2018. Research and development expense was $11.3 million, compared with $7.4 million for the same period of 2018, due to costs associated with recent acquisitions. General and administrative expense was $11.1 million, compared with $7.6 million for the same period in 2018, due to costs associated with recent acquisitions and non-cash stock-based compensation expense.

Net income for the first quarter of 2019 was $666.3 million, or $31.32 per diluted share, compared with net income of $45.3 million, or $1.83 per diluted share, for the same period in 2018. Net income for the first quarter of 2019 was impacted by an after-tax gain of just over $640 million on the sale of Ligand’s assets and royalty on Promacta to Royalty Pharma. Adjusted net income for the first quarter of 2019 was $24.8 million, or $1.16 per diluted share excluding the impact of the gain recognized on the sale of Promacta, compared with adjusted net income of $35.7 million, or $1.55 per diluted share, for the same period in 2018.

As of March 31, 2019, Ligand had cash, cash equivalents and short-term investments of approximately $1.4 billion. Cash generated from operations during the first quarter of 2019 was $45.3 million.

2019 Financial Guidance

Ligand is affirming existing revenue guidance for 2019 with total revenues expected to be approximately $118 million including royalties of approximately $48 million, material sales of approximately $27 million and license fees and milestones of approximately $43 million. Ligand is also affirming its existing adjusted earnings per share guidance of approximately $3.20, which excludes the impact of the gain recognized on the sale of Promacta of $30.09 per share compared to the initial estimate of the impact of the gain of $29.05 per share.

First Quarter 2019 and Recent Business Highlights

Promacta

In March 2019, Ligand announced the sale of Promacta to Royalty Pharma for $827 million in cash. As of March 6, 2019 Ligand will not receive any royalty on sales of Promacta.
Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

On April 30, 2019, Amgen reported first quarter 2019 net sales of Kyprolis of $245 million, a $23 million or 10% increase over the same period in 2018.
Recent Acquisitions, Targeted Investments and Partner Funding Events

Ligand announced a $3 million investment in Dianomi Therapeutics in exchange for 1) a tiered royalty of 2% or 3% based on level of net sales for the first five products to be approved using Dianomi’s patented Mineral Coated Microparticle (MCM) technology, and 2) a loan convertible into $1 million of equity at the next qualified financing.
Seelos Therapeutics completed a reverse merger with Apricus Biosciences and is now publicly traded on the Nasdaq Capital Market under the symbol "SEEL". In conjunction with the transaction, Seelos issued common stock and warrants in a private round led by a group of leading venture capital investors, for gross proceeds of $18 million.
CStone Pharmaceuticals listed shares on the Hong Kong Stock Exchange and raised $266 million in an equity offering that, in part, will be used to fund the company’s OmniAb-derived Phase 2/3 program CS1001.
Internal R&D

Ligand announced completion of enrollment of the Company’s Phase 1 clinical trial of its internal Captisol-enabled Iohexol program and announced that top-line data from the trial is expected in the third quarter of 2019.
Additional Pipeline and Partner Developments

Ligand’s portfolio of partnerships now includes more than 200 Shots on Goal driven by business development and licensing activity, and the expansion and advancement of OmniAb partnerships.
Sage Therapeutics announced U.S. Food and Drug Administration (FDA) approval of ZULRESSO (brexanolone) injection for the treatment of postpartum depression. Ligand received a $3 million milestone payment as a result of the approval. Sage Therapeutics plans to launch ZULRESSO in late June 2019.
Daiichi Sankyo announced receipt of marketing approval in Japan for MINNEBRO (esaxerenone) for the treatment of hypertension.
Melinta Therapeutics announced preparation of a supplemental new drug application (sNDA) for Baxdela in community-acquired bacterial pneumonia with the sNDA expected to be filed in the second quarter of 2019.
CStone Pharmaceuticals announced dosing of the first patient in a Phase 3 clinical trial assessing OmniAb-derived CS1001 in combination with chemotherapy for the treatment of gastric adenocarcinoma or gastro-esophageal junction adenocarcinoma.
Viking Therapeutics announced that additional VK2809 Phase 2 data was presented at the 2019 annual meeting of the European Association for the Study of Liver, and that results demonstrated promising efficacy at doses as low as 5 mg daily. Viking also announced plans to initiate a Phase 2b study of VK2809 in biopsy-confirmed NASH in the second half of 2019.
Metavant updated Ligand that they no longer plan to initiate a clinical proof-of-concept trial this year for RVT-1502 in Type 1 diabetes following requests from FDA for additional non-clinical studies. Metavant is evaluating its development plans for the program, and we expect them to provide an update on the status of the program.
Verona Pharma announced positive interim efficacy and safety data from part one of a two-part Phase 2 clinical trial of a dry powder inhaler formulation of ensifentrine in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD).
Verona Pharma also announced the European Patent Office granted an additional key patent relating to the company’s lead development candidate ensifentrine.
Sermonix Pharmaceuticals announced a poster presentation on the performance of its lead investigational drug, lasofoxifene, at ENDO 2019.
Opthea Limited announced that the independent Data and Safety Monitoring Board for the company’s ongoing Phase 2b study of OPT-302 in wet age-related macular degeneration reaffirmed its positive recommendation that the trial continue without modification.
Aptevo Therapeutics provided an update on OmniAb-derived APVO436 and announced that Phase 1 data is anticipated in the fourth quarter of 2019. New preclinical data for APVO436 was also presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting.
OmniAb partner xCella Biosciences presented high-throughput functional screening of antibody libraries, highlighting OmniRat and OmniChicken, at the 2019 Protein Engineering Summit (PEGS).
Business Development

Ligand announced a worldwide license agreement with Genagon Therapeutics AB to use the OmniAb platform technologies to discover fully human antibodies. Genagon is an immuno-oncology biotechnology company located in Sweden. Ligand is eligible to receive development milestone payments and tiered royalties on future product sales.
Ligand disclosed a worldwide license agreement with a San Francisco Bay Area venture-stage biotechnology company to use the OmniAb platform technologies to discover fully human antibodies. Ligand is eligible to receive development and commercial milestone payments and royalties on future product sales.
Ligand entered into new Captisol clinical use or commercial license and supply agreements with Merck KGaA, reVision Therapeutics, Takeda and SQ Innovation.
Publications and Presentations

At PEGS 2019 Ligand scientists announced the launch of OmniClic, a novel next-generation common light chain OmniChicken-based antibody discovery technology focused on bispecific antibodies.
Preclinical data of the combination of the BCL-2 inhibitor S55746 and the MCL1 inhibitor S63845, compounds originating from the Servier collaboration and now partnered with Novartis, in models of AML were recently published in the journal Leukemia, demonstrating potential to rapidly suppress leukemia with limited toxicity to normal human bone marrow cells compared to chemotherapy.
Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include stock-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, unissued shares relating to the Senior Convertible Notes, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, other than with respect to total revenues, the Company only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 591-4752 from the U.S. or (720) 405-1612 from outside the U.S., using the conference ID 6375579. To participate via live or replay webcast, a link is available at www.ligand.com.

On May 2, 2019 Incyte Corporation (Nasdaq:INCY) reported that it will present at the following investor conferences during the month of May (Press release, Incyte, MAY 2, 2019, View Source [SID1234535576]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Bank of America Merrill Lynch Health Care Conference on Tuesday, May 14, 2019 at 8:40 am (PDT) in Las Vegas;
UBS Global Healthcare Conference on Monday, May 20, 2019 at 3:00 pm (EDT) in New York; and
RBC Capital Markets Healthcare Conference on Tuesday, May 21, 2019 at 10:30 am (EDT) in New York
The presentations will be webcast live and can be accessed at www.incyte.com in the Investors section under "Events and Presentations." Investors interested in listening to the live webcast should log on before the start time in order to download any software required.

On May 2, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it will present new and compelling data during an oral presentation about its off-the-shelf SPEAR T-cell program at the annual American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) meeting (Press release, Adaptimmune, MAY 2, 2019, View Source;p=RssLanding&cat=news&id=2396799 [SID1234535607]). Data indicate that T-cells can be generated from Human Induced Pluripotent Stem Cells (hiPSC) in vitro and that these T-cells respond to cancer targets via engineered SPEAR TCRs. This process will be used to investigate the ability of this off-the-shelf SPEAR T-cell product to fight cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our three autologous SPEAR T-cell therapies are already in the clinic and have the potential to treat cancer in multiple solid tumor indications," said Rafael Amado, Adaptimmune’s President of R&D. "Beyond the promise of our current autologous therapies, this allogeneic approach – or more simply ‘off-the-shelf’ product – could offer treatment to more patients more quickly, and provide a homogeneous and unlimited source of therapeutic cells. We are thrilled with the progress we have made, both in the gene editing space with our partners at Universal Cells and with our internal T-cell differentiation program."

During an oral presentation scheduled for 11:30 AM ET today at the ASGCT (Free ASGCT Whitepaper) Annual Meeting, Dr. Jo Brewer, Adaptimmune’s Vice President of Allogeneic Research, will present progress to-date with Adaptimmune’s off‑the-shelf SPEAR T-cell program:

Adaptimmune has demonstrated hiPSC differentiation in a serum-free process without the addition of mouse stromal cells, which is designed to enable scale-up and GMP manufacture of a gene-edited off-the-shelf SPEAR T-cell product
Lentiviral transduction of T-cells derived from hiPSCs with a SPEAR TCR produces differentiated T-cells that can respond to cancer targets in vitro
The process starts with an hiPSC line, before the cells are rendered invisible to the host immune system with a series of Recombinant Adeno-Associated Virus (rAAV)-based gene-editing steps (the editing will be performed in association with Universal Cells, an Astellas Company)
The process has been shown to promote differentiation of cells from a pluripotent state (SSEA4+Oct4+TRA-160+) through various intermediate stages: CD34+CD45+ hematopoietic progenitor cells (HPC), pro-/pre-T CD7+CD5+cells, and CD4+CD8+ double positive cells towards CD3+CD8+TCR+ single positive T-cells