On November 5, 2021 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported that it plans to host a conference call on Tuesday, November 9, 2021, at 8:30 a.m. ET to discuss its financial results for the quarter ended September 30, 2021 and provide a business update (Press release, Selecta Biosciences, NOV 5, 2021, https://selectabio.gcs-web.com/news-releases/news-release-details/selecta-biosciences-host-conference-call-and-webcast-discuss-6 [SID1234594589]).

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Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10147803. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On November 5, 2021Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company with a mission to deliver targeted therapies that treat rare forms of cancer, reported its financial and operating results for the third quarter ended September 30, 2021 (Press release, Sierra Oncology, NOV 5, 2021, View Source [SID1234594588])."The past several months have been quite productive for Sierra with the addition of SRA515 to our pipeline and preparing for momelotinib pivotal data," said Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology. "We remain focused on the completion of the MOMENTUM study and look forward to sharing topline results by February next year."

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Key Business Highlights

The pivotal, Phase 3 MOMENTUM study remains on track to report topline data by February 2022. Assuming positive results, the company plans to submit a New Drug Application with the US Food & Drug Administration in the second quarter of 2022.
An exclusive global in-licensing agreement for SRA515 (formerly AZD5153), a potent and selective BRD4 BET inhibitor with a novel bivalent binding mode, was announced on August 5. The initiation of a Phase 2 study examining momelotinib in combination with SRA515 for the treatment of myelofibrosis is planned for the first half of 2022. We continue to evaluate additional studies and may initiate other new trials for SRA515 and SRA737 in 2022.
Sierra raised $37.0 million in gross proceeds through the use of its at-the-market (ATM) offering during the third quarter of 2021 and ended the quarter with $97.1 million in cash and cash equivalents. Subsequent to the close of the third quarter, the company has raised an additional $12.6 million in gross proceeds via the ATM. The company also has the potential to obtain up to $33.3 million in cash from Series B warrants that must be exercised within 75 days of the announcement of MOMENTUM topline data.
On November 4, the company announced the acceptance of a momelotinib abstract for poster presentation at the 2021 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper). The poster, "Baseline Serum Ferritin Differentially Predicts W24 Transfusion Independence Response for Momelotinib and Ruxolitinib in Patients with Myelofibrosis" will be presented on Monday, December 13, 2021, from 6:00 – 8:00 pm ET, Georgia World Congress Center, Hall B5. The abstract has been published online View Source and a pre-recorded presentation of the poster will be made available for participants attending virtually.
Third Quarter 2021 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $21.6 million for the three months ended September 30, 2021 compared with $10.4 million for the three months ended September 30, 2020. The increase primarily related to an upfront cash payment of $8.0 million that was made to AstraZeneca for the exclusive global license of SRA515. Also attributing to the increase was a $2.2 million increase in personnel-related and allocated overhead costs, of which $0.6 million pertained to an increase in non-cash stock-based compensation, a $1.0 million increase in third-party manufacturing costs for momelotinib, and a $0.3 million increase in other research and support costs. These increases were partially offset by a $0.3 million decrease in clinical trial and development costs for momelotinib. Research and development expenses included non-cash stock-based compensation of $1.8 million and $1.2 million for the three months ended September 30, 2021 and 2020, respectively.

Research and development expenses were $49.7 million for the nine months ended September 30, 2021, compared with $32.2 million for the nine months ended September 30, 2020. The increase was due to an upfront cash payment of $8.0 million that was made to AstraZeneca for the exclusive global license of SRA515, a $5.8 million increase in personnel-related and allocated overhead costs, of which $2.6 million pertained to an increase in non-cash stock-based compensation, and costs for momelotinib including a $2.8 million increase in clinical trial and development costs and a $2.4 million increase in third-party manufacturing costs. These increases were partially offset by a $1.5 million non-cash charge incurred in 2020 to recognize the change in fair value of an obligation to issue securities to Gilead until the issuance of the securities in January 2020. Research and development expenses included non-cash stock-based compensation of $5.3 million and $2.7 million for the nine months ended September 30, 2021 and 2020, respectively.

General and administrative expenses were $7.0 million for the three months ended September 30, 2021, compared to $4.1 million for the three months ended September 30, 2020. The increase was due to a $1.7 million increase in personnel-related and allocated overhead costs, of which $0.6 million pertained to an increase in non-cash stock-based compensation, and a $1.2 million increase in professional fees primarily relating to pre-commercial costs for momelotinib. General and administrative expenses included non-cash stock-based compensation of $1.5 million and $0.9 million for the three months ended September 30, 2021 and 2020, respectively.

General and administrative expenses were $19.3 million for the nine months ended September 30, 2021, compared to $14.9 million for the nine months ended September 30, 2020. The increase was due to a $2.7 million increase in personnel-related and allocated overhead costs, of which $0.2 million pertained to an increase in non-cash stock-based compensation, and an increase of $1.7 million in professional fees primarily relating to pre-commercial costs for momelotinib. General and administrative expenses included non-cash stock-based compensation of $4.2 million and $4.0 million nine months ended September 30, 2021 and 2020, respectively.

Total other expense (income), net was $0.1 million of total other expense, net for the nine months ended September 30, 2021, compared to $15.7 million of total other expense, net for the nine months ended September 30, 2020. The difference was primarily attributable to a non-cash charge of $16.2 million incurred during the nine months ended September 30, 2020 related to the change in fair value of warrant liabilities until the reclassification to equity in January 2020.

For the three months ended September 30, 2021, Sierra incurred a Generally Accepted Accounting Principles (GAAP) net loss of $28.6 million compared to a GAAP net loss of $14.5 million for the three months ended September 30, 2020. For the nine months ended September 30, 2021 Sierra incurred a GAAP net loss of $69.2 million compared to a GAAP net loss of $62.9 million for the nine months ended September 30, 2020. The GAAP net loss for the three and nine months ended September 30, 2021 includes an upfront cash payment of $8.0 million that was made to AstraZeneca for the exclusive global license of SRA515. The GAAP net loss for the nine months ended September 30, 2020 includes a non-cash charge of $16.2 million related to the change in fair value of warrant liabilities included in total other expense (income), net and a $1.5 million non-cash charge pertaining to the obligation to issue securities to Gilead included in research and development expenses as mentioned above.

Non-GAAP adjusted net loss was $25.4 million for the three months ended September 30, 2021, compared with a non-GAAP adjusted net loss of $12.4 million for the three months ended September 30, 2020. Non-GAAP adjusted net loss for the three months ended September 30, 2021 and 2020 excludes expenses related to stock-based compensation. For the nine months ended September 30, 2021, Sierra incurred a non-GAAP adjusted net loss of $59.8 million compared to a non-GAAP adjusted net loss of $38.5 million for the nine months ended September 30, 2020. Non-GAAP adjusted net loss for the nine months ended September 30, 2021 excludes expenses related to stock-based compensation. Non-GAAP adjusted net loss for the nine months ended September 30, 2020 excludes expenses related to the change in fair value of warrant liabilities, the change in fair value of the securities issuance obligation, and stock-based compensation. See "Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below for a reconciliation of this GAAP measure to non-GAAP financial measure.

Cash and cash equivalents totaled $97.1 million as of September 30, 2021, compared to $104.1 million as of December 31, 2020.

As of September 30, 2021, there were 14,498,953 total shares of common stock outstanding and warrants to purchase 11,040,894 shares of common stock, with an exercise price equal to $13.20 per share. There were 4,880,267 shares issuable upon exercise of stock options and an additional warrant to purchase 1,839 shares.

On November 5, 2021 Filtricine reported that researchers leading a Filtricine-sponsored prostate cancer clinical study at Stanford University School of Medicine presented results from the first 10 participants at the 2021 Annual Prostate Cancer Foundation Scientific Retreat (Press release, Filtricine, NOV 5, 2021, View Source [SID1234594587]).

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The poster presented featured early results from "A Feasibility Study to Deplete Non-Essential Amino Acids in Patients with Prostate Cancer," which used Tality, the medical food program developed by Filtricine for the dietary management of cancer. All 10 participants are instructed to use Tality as their sole nutritional intake for 28 days. The poster stated the researchers’ conclusion: "so far, we can conclude from the first ten participants that Tality is tolerable for the study period of 28 days, can preferentially reduce non-essential amino acids, and may reduce PSA for patients with high baseline PSA values." PSA, or prostate-specific antigen, is a biomarker of prostate cancer. The poster also stated that, while the study was designed to test Tality’s tolerability and adherence, "three participants experienced decrease in PSA; one participant experienced 32% decrease from baseline level of 107.9."

The principal investigators of the study are Randall S. Stafford, MD, PhD, Professor of Medicine at the Stanford School of Medicine and the Director of the Program on Prevention Outcomes and Practices, and Alice C. Fan, MD, Assistant Professor of Medicine (Oncology) at Stanford University School of Medicine.

The Stanford investigators presented these results at the 2021 Annual Prostate Cancer Foundation Scientific Retreat:

link to the poster on PCF website; download the poster in pdf

Researchers are seeking additional participants for this prostate cancer clinical study:

View Source

On November 5, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported the presentation of data from its ex vivo research and development efforts in two poster presentations at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place in Atlanta, GA and virtually from December 11-14, 2021 (Press release, Intellia Therapeutics, NOV 5, 2021, View Source [SID1234594586]).

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"As we continue to advance our full-spectrum strategy, we look forward to sharing preclinical data from our ex vivo platform with the research community at this year’s ASH (Free ASH Whitepaper) Annual Meeting," said Intellia Chief Scientific Officer Laura Sepp-Lorenzino, Ph.D. "The data will feature our novel allogeneic technology designed to overcome rejection by host T and NK cells without the need for host immune suppression, as well as highlight our clinical-scale manufacturing process developed for NTLA-5001, our TCR-based T cell therapeutic candidate for the treatment of acute myeloid leukemia. Together, the data support our progress toward developing engineered cell therapies with the potential to transform the lives of people living with life-threatening diseases."

ASH Annual Meeting Poster Presentations

Title: A Novel Strategy for Off-the-shelf T Cell Therapies Evading Host T Cell and NK Cell Rejection
Abstract number: 1711
Date/Time: Saturday, December 11, 2021, 5:30 p.m. – 7:30 p.m. ET
Location: Georgia World Congress Center, Hall B5
Presenting Author: Yong Zhang, Ph.D., associate director, Cell Therapy

Title: Clinical-scale Production and Characterization of NTLA-5001 – a Novel Approach to Manufacturing CRISPR/Cas9 Engineered T cell Therapies
Abstract number: 3881
Date/Time: Monday, December 13, 2021, 6:00 p.m. – 8:00 p.m. ET
Location: Georgia World Congress Center, Hall B5

Presenting Author: Daniel Cosette, senior scientist, Process Development

Additional data collected will be included in final meeting presentations. All abstracts for the ASH (Free ASH Whitepaper) Annual Meeting will be available on ASH (Free ASH Whitepaper)’s website here.

On November 5, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the third quarter ended September 30, 2021 (Press release, bluebird bio, NOV 5, 2021, View Source [SID1234594585]).

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"This quarter was about preparing for the completion of the separation of bluebird bio and 2seventy bio and realizing the value of two independent companies," said Andrew Obenshain, chief executive officer, bluebird bio. "Notably this quarter, we secured additional capital through the close of a private financing and completion of the sale of our manufacturing facility in North Carolina and continued to make meaningful progress with our product pipeline, including filing the US biologics licensing application for beti-cel for beta-thalassemia. I am excited for what lies ahead for both bluebird and 2seventy bio, and the impact that both companies will have for patients and their families."

BUSINESS SEPARATION RECENT HIGHLIGHTS

COMPLETION OF SEPARATION – On November 4, 2021, bluebird bio completed the tax-free spin-off of its oncology business, 2seventy bio, Inc. bluebird bio will continue its work focused on severe genetic diseases, with three near-term opportunities to bring transformative gene therapies to patients and their families in the U.S. 2seventy began regular-way trading on the NASDAQ under the stock ticker symbol "TSVT" on November 5, 2021. bluebird bio will continue to trade under the stock ticker symbol "BLUE".
PRIVATE FINANCING – Prior to the separation on September 8, 2021, bluebird bio announced that it has entered into an agreement for a $75 million private placement of common stock and common stock equivalents with a healthcare investment fund selected as part of a competitive process.
STARTING CASH POSITION – As of completion of the separation, bluebird’s restricted cash, cash and cash equivalents and marketable securities balance is approximately $518.5M. Increased fiscal discipline, including through projected real estate savings with the move of the Company’s headquarters to Assembly Row in Somerville, Massachusetts, and the wind down of European operations, together with the potential sale of priority review vouchers that would be issued with anticipated U.S. regulatory approvals of biologics licensing applications for beti-cel and eli-cel will be sufficient to fund operations for bluebird bio into 2023 under current business plans.
RECENT HIGHLIGHTS

β-THALASSEMIA

BETI-CEL SUBMISSION – On September 21, 2021, bluebird bio announced it completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell transfusions, across all genotypes. If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the United States.
COMPANY

NEW HEADQUARTERS – Today, bluebird bio announced its new headquarters in Assembly Row, designed to reflect modern ways of working and estimated to result in more than $120 million in cost savings over the next six years for the company. bluebird signed a long-term lease with Federal Realty Investment Trust (FRIT) for the 61,000 square foot facility located at 455 Grand Union in Somerville, MA.
BOARD OF DIRECTORS – This quarter, bluebird bio announced the appointment of Najoh Tita-Reid (Logitech) and Lis Leiderman, M.D. (Decibel Therapeutics) to its board of directors. They are joined on the bluebird bio board of directors by Mark Vachon (chairman – formerly of GE), John Agwunobi, M.D. (Herbalife Nutrition), Wendy Dixon, Ph.D. (formerly of Bristol-Myers Squibb), Nick Leschly (2seventy bio) and Andrew Obenshain (bluebird bio).
EUROPE WIND DOWN – Following the August 9, 2021 announcement that it intended to wind down operations in Europe, on October 21, bluebird bio announced that it will withdraw its regulatory marketing authorization for SKYSONA from the European Union, and its marketing application for SKYSONA from the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (UK). bluebird bio, Inc. also anticipates withdrawing marketing authorizations for ZYNTEGLO from both the EU and the UK by early 2022. The company expects to continue activities for the long-term follow-up of patients previously enrolled within the European clinical trial programs as planned, but does not intend to initiate any new clinical trials in Europe for the beta-thalassemia, cerebral adrenoleukodystrophy or sickle cell disease programs.
MANAGEMENT APPOINTMENT – On November 4, 2021, bluebird bio announced the appointment of Gina Consylman as Chief Financial Officer, effective upon the completion of the spin-off transaction of 2seventy bio.
UPCOMING ANTICIPATED MILESTONES

beti-cel: Acceptance of the BLA to the US Food and Drug Administration for beti-cel for beta-thalassemia expected this month.
eli-cel: The BLA filing for elivaldogene autotemcel (eli-cel, Lenti-D) for patients with cerebral adrenoleukodystrophy (CALD) is on track for the end of 2021.
eli-cel: The company is in active communication with the FDA to resolve the clinical hold.
bb1111: The company plans to host an investor event on November 18th, 2021, to share further detail on its sickle cell disease program and path to regulatory approval.
American Society of Hematology Annual Meeting: bluebird will present new data on beti-cel and bb1111 at ASH (Free ASH Whitepaper) 2021, including long-term results for beti-cel in adult and pediatric patients with beta-thalassemia, new analyses from Groups A&C of the ongoing Phase 1/2 HGB 206 study of bb1111 for sickle cell disease, and sustained improvements in patient reported quality of life in Group C.
THIRD QUARTER 2021 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2021, and December 31, 2020, were $970.7 million and $1.27 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities.
Revenues: Total revenues were $22.7 million for the three months ended September 30, 2021, compared to $19.3 million for the three months ended September 30, 2020. Total revenues were $42.9 million for the nine months ended September 30, 2021, compared to $240.0 million for the nine months ended September 30, 2020. The increase for the three-month period was primarily driven by our collaborative arrangement revenue recognized under our collaboration arrangement with BMS. The decrease for the nine-month period was primarily driven by a cumulative catch-up adjustment to revenue recorded in connection with the May 2020 BMS contract modification in the second quarter of 2020.
ABECMA Revenue: This quarter Bristol-Myers Squibb (BMS) reported total U.S. revenues of $67 million for ABECMA (idecabtagene vicleucel; ide-cel). bluebird bio reported a net collaboration revenue of $14.8 million for 3Q, which includes the company’s share of revenue and costs associated with the commercialization of ABECMA in the U.S.
R&D Expenses: Research and development expenses were $131.4 million for the three months ended September 30, 2021, compared to $140.4 million for the three months ended September 30, 2020. Research and development expenses were $429.6 million for the nine months ended September 30, 2021, compared to $450.9 million for the nine months ended September 30, 2020. The decrease for the three-month period was primarily driven by decreased collaboration research funding costs resulting from a decrease in expense recognized under our collaboration arrangement with BMS. The decrease for the nine-month period was primarily driven by decreased manufacturing expenses.
SG&A Expenses: Selling, general and administrative expenses were $68.3 million for the three months ended September 30, 2021, compared to $68.0 million for the three months ended September 30, 2020. Selling, general and administrative expenses were $229.7 million for the nine months ended September 30, 2021, compared to $210.0 million for the nine months ended September 30, 2020. The increase for both periods was primarily driven by an increase in fees associated with the spinoff of 2seventy bio as well as increased employee compensation, benefit, and other headcount related expenses.
Restructuring Expenses: Restructuring expenses were $20.2 million and $24.8 million for the three months and nine months ended September 30, 2021, respectively. These costs are related to a reduction in the workforce, primarily driven by the wind down of operations in Europe.
Net Loss: Net loss was $216.8 million for the three months ended September 30, 2021, compared to $194.7 million for the three months ended September 30, 2020. Net loss was $664.3 million for the nine months ended September 30, 2021, compared to $418.8 million for the nine months ended September 30, 2020.