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Bristol Myers Squibb and bluebird bio Provide Regulatory Update on Idecabtagene Vicleucel (ide-cel, bb2121) for the Treatment of Patients with Multiple Myeloma

On May 13, 2020 Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) reported that the companies received a Refusal to File letter from the U.S. Food and Drug Administration (FDA) regarding the Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121) for patients with heavily pre-treated relapsed and refractory multiple myeloma, which was submitted in March 2020 (Press release, Bristol-Myers Squibb, MAY 13, 2020, View Source [SID1234557878]).

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Upon preliminary review, the FDA determined that the Chemistry, Manufacturing and Control (CMC) module of the BLA requires further detail to complete the review. No additional clinical or non-clinical data have been requested or are required. Bristol Myers Squibb is planning to resubmit the BLA no later than the end of July 2020.

Bristol Myers Squibb Investor Call and Webcast

Bristol Myers Squibb will hold an investor conference call to discuss this update today at 8:00 a.m. EDT.

Investors are invited to listen to a live webcast of the call at bms.com/investors or by dialing toll free in the U.S. 877-257-8599 or international 1-631-291-4581, confirmation code: 5938714. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.

bluebird bio Investor Call and Webcast
bluebird bio will hold an investor conference call to discuss the update today at 8:45 a.m. EDT.

Investors are invited to listen to a live webcast of the call on the investors page of www.bluebirdbio.com or by dialing toll free in the U.S. (844) 825-4408 or international (315) 625-3227, confirmation code: 2892826. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.

For Holders of Contingent Value Rights (CVR), Ticker BMY-RT

U.S. FDA approval of ide-cel by March 31, 2021 is one of the required remaining milestones of the Contingent Value Rights issued upon the close of the Celgene acquisition in the fourth quarter of 2019. The other is U.S. FDA approval of liso-cel by December 31, 2020. The company is committed to working with FDA to progress both applications and achieve the remaining regulatory milestones required by the CVR.

About Ide-cel

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

In addition to the pivotal KarMMa trial evaluating ide-cel in patients with relapsed and refractory multiple myeloma, Bristol Myers Squibb and bluebird bio’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the FDA and PRIority Medicines (PRIME) designation, as well as Accelerated Assessment status, by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients’ quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early- to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

ProMIS Neurosciences Announces First Quarter 2020 Results

On May 13, 2020 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF) ("ProMIS or the Company"), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the three months ended March 31, 2020 (Press release, ProMIS Neurosciences, MAY 13, 2020, View Source [SID1234557877]).

"Over the course of the first quarter 2020, the value of our unique discovery and development platform was further evidenced as ProMIS made considerable progress in expanding its portfolio of opportunities across multiple neurodegenerative diseases," stated Eugene Williams, ProMIS’ Executive Chairman.

"PMN310, our lead antibody therapeutic candidate for Alzheimer’s disease (AD), showed further significant positive differentiation in both potential efficacy and safety compared to competitive antibody therapeutics currently in development. In addition, antibody candidates selectively targeting toxic forms of alpha-synuclein for Parkinson’s disease, toxic aggregated forms of TDP- 43 for amyotrophic lateral sclerosis (ALS) and toxic aggregates of tau for AD and other dementias were identified and further characterized to support collaborative partnering discussions."

Corporate Highlights

In January and February 2020, we received proceeds from the exercise of warrants in the amount of $958,000.
In January 2020, José Luis Molinuevo, MD, PhD, was appointed to the Company’s Scientific Advisory Board.
In February, we announced initiation of a natural history study of blood-based biomarkers in Alzheimer’s disease in collaboration with Toronto Memory Program, Canada’s largest and most experienced memory clinic and site for drug treatment trials in AD.
In March 2020, we announced that the Toronto Stock exchange approved the amendment of the exercise price of an aggregate of 44,182,530 outstanding common share purchase warrants. The exercise price of the warrants is being repriced to $0.13 effective April 8, 2020 until May 22, 2020.
Financial Results

Results of Operations – Three months ended March 31, 2020 and 2019

Net loss for the three months ended March 31, 2020 was $1,761,919, compared to a net loss of $2,446,577 for the three months ended March 31, 2019. Included in the net loss amount for the three months ended March 31, 2020 were non-cash expenses of $213,739, representing share-based compensation, warrant modification and amortization of an intangible asset, compared to $263,872 for the three months ended March 31, 2019. The decrease in the net loss for the three months ended March 31, 2020 reflects decreased costs associated with external contract research organizations for internal programs, patent costs, decreased consultant salaries and associated costs and share-based compensation offset by warrant modification.

Research and development expenses for the three months ended March 31, 2020 were $973,586, as compared to $1,770,653 in the three months ended March 31, 2019. The decrease in research and development expense for the three months ended March 31, 2020, compared to the same period ended March 31, 2019 is primarily attributed to decreased spending on external contract research organizations for internal programs, reduced patent expense and share-based compensation offset by increased contracted research salaries and associated costs and external consulting expense.

General and administrative expenses for the three months ended March 31, 2020 were $788,346, as compared to $675,924 in the three months ended March 31, 2019. The increase for the three months ended March 31, 2020, compared to the same period in 2019, is primarily attributable to the warrant modification expense.

Outlook

As a prelude to the first PMN310 clinical trial in AD, ProMIS anticipates using a novel biomarker approach that may show evidence of slowing of neuronal death early in the development program. To accomplish this, we initiated the pilot phase of a natural history evaluation of biomarker changes in untreated, mild AD patients in February 2020.

The Company will also continue to further characterize the potential benefits of its programs selectively targeting toxic aggregates of TDP-43 in ALS, toxic forms of alpha-synuclein in PD and toxic aggregates of tau in AD and other dementias to further support on-going pharmaceutical partnering discussions. ProMIS’ unique platform produces antibodies that meet a key success factor for development of therapeutics for neurodegenerative diseases: the ability to selectively target the neurotoxic form of a protein implicated as a root cause of disease, while sparing the normal forms of the protein.

In the infectious disease setting, we will focus our unique technology platform to identify peptide antigens that can be used as an essential component to create accurate and sensitive serological assays to detect the presence of antibodies that arise in response to a specific infection, such as COVID-19.

FDA Clears AgenTus IND for Allogeneic iNKT Cell Therapy

On May 13, 2020 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of agents designed to activate immune response to cancers and infectious diseases, reported the FDA acceptance of the IND application for an allogeneic iNKT therapy, agenT-797, submitted by its subsidiary, AgenTus Therapeutics. A clinical trial for the treatment of patients with cancer is expected to commence 2H2020 (Press release, Agenus, MAY 13, 2020, View Source [SID1234557874]). AgenTus has also submitted a separate IND for the treatment of COVID-19, which is expected to clear soon.

"We are extremely pleased to rapidly advance our proprietary iNKT cell therapy to the clinic to combat cancer and also address the urgent COVID-19 pandemic," said Dr. Walter Flamenbaum, CEO of AgenTus Therapeutics. "We expect our clinical trials to demonstrate the key features of agent-797 and the benefits of combinations of our allogeneic iNKTs with Agenus’ pipeline of checkpoint antibodies in solid tumors."

"I am heartened by our team’s efforts to rapidly advance our allogeneic iNKT cells to treat patients with cancer and COVID-19," said Dr. Garo Armen, Chairman and CEO of Agenus and Chairman of AgenTus Therapeutics. "Our iNKTs are streamlined for treating large numbers of patients from a single batch. They are manufactured without the need for genetic manipulation, are expected to suppress graft-versus-host disease, can be manufactured affordably, and are designed for quick access by patients."

Separately, Agenus indicated that it is contemplating several options with regard to its AgenTus Therapeutics subsidiary. This is given its previous guidance and the advancement of AgenTus’ first cell therapy candidates towards the clinic. These options include the potential spinning out of AgenTus and issuing a portion of its holdings in AgenTus to Agenus shareholders in the form of a stock dividend. Agenus expects that such a decision will be made by the end of this year.

Alector Reports 2020 First Quarter Financial Results and Business Highlights

On May 13, 2020 Alector, Inc. (Nasdaq: ALEC), a clinical- stage biotechnology company pioneering immuno-neurology, reported business highlights and financial results for the first quarter ended March 31, 2020 (Press release, Alector, MAY 13, 2020, View Source [SID1234557873]).

"At Alector we are committed to developing transformative treatments for neurodegeneration. We believe that our mission could benefit millions of patients and families affected by neurodegenerative diseases, and even with the current COVID-19 health crisis, we remain focused on advancing our portfolio of immuno-neurology programs," said Arnon Rosenthal, Ph.D., Co-founder, and Chief Executive Officer of Alector. "Our COVID-19 task force and the entire Alector team continues to focus on initiating a pivotal Phase 3 study of AL001 in FTD-GRN patients and a Phase 2 study of AL002 in Alzheimer’s disease patients in 2020."

Business Highlights

COVID-19 Response

Alector is actively monitoring the evolving impact of COVID-19 on its operations and clinical trials, with a primary focus on the health and safety of employees, clinical trial participants, and clinical trial site teams. The Company is complying with regulatory, institutional, and governmental guidance for conducting its business worldwide. As the COVID-19 pandemic continues to evolve, it could impact Alector’s programs in the future.

The Company is also continuing with its efforts to complete enrollment across ongoing clinical trials. Currently, certain clinical trial sites have delayed enrollment of new patients and paused clinical trial visits across clinical development programs. Alector is aware that some participants in ongoing trials have not been able to receive scheduled doses on time due to site closures or various state and local shelter-in-place directives. However, the Company is continuing to collect data from all existing clinical trial participants enrolled to date.

The Company remains on track with previously stated guidance to initiate a pivotal Phase 3 study of AL001 in FTD-GRN patients in 2020. Alector also intends to initiate a Phase 2 study of AL002 in Alzheimer’s disease patients in 2020. Ongoing activities for AL003, AL101, and AL014

programs are continuing as planned. The Company believes that its cash and investments as of March 31, 2020 will be sufficient to fund its anticipated operations through 2022.

Progranulin Portfolio: AL001, AL101

The Company remains on track to advance AL001 into a pivotal Phase 3 study in FTD-GRN patients in 2020.

Alector expects to present preliminary Phase 2 data of AL001 in FTD-GRN patients at medical meetings in 2020. The number of patients with available data for presentation may be impacted by the COVID-19 pandemic.

Initial Phase 1a data of AL101 in healthy volunteers are also expected during in 2020.

Alzheimer’s Disease Portfolio: AL002, AL003, AL014

Following the completion of the Phase 1a study with AL002 and based on the safety and biomarker data collected in preclinical studies and in healthy volunteers, and in agreement with its partner AbbVie, Alector has closed enrollment for the Phase 1b study of AL002 and will proceed with initiating a Phase 2 trial in Alzheimer’s disease patients in 2020.

The Company continues to advance the Phase 1b trial of AL003 in Alzheimer’s disease. AL003 is being developed by Alector in collaboration with its partner AbbVie.

Alector plans to initiate Phase 1 development for AL014 within the next 12 months. AL014 is the Company’s latest prioritized candidate that targets MS4A4A, a transmembrane receptor protein that is expressed selectively in microglia in the brain and is associated with control of microglia functionality and potential viability.

Immuno-oncology Portfolio

In March, Alector entered into a regional licensing agreement with Innovent Biologics for the development and commercialization of AL008 in oncology indications in China. AL008 is a potentially best-in-class SIRP-alpha inhibitor with a unique dual mechanism of action that non-competitively antagonizes the CD47-SIRP-alpha pathway by inducing the internalization and degradation of the inhibitory receptor on macrophages to relieve immune suppression (a "don’t eat me signal") while also engaging Fc gamma receptors to promote immuno-stimulatory pathways that drive anti-tumor immunity. Alector retains the global rights for AL008 outside of China.

First Quarter 2020 Financial Results

Revenue. Collaboration revenue for the quarter ended March 31, 2020 was $7.2 million compared to $5.6 million for the same period in 2019. This increase was primarily due to an increase in expenses for the AL002 and AL003 programs compared to the same period last year.

R&D Expenses. Total research and development expenses for the quarter ended March 31, 2020 were $34.6 million compared to $20.6 million for the same period in 2019. This increase was driven by an increase in personnel-related expenses as headcount grew to support the advancement of the clinical and pre-clinical programs. Additionally, expenses increased due to timing of manufacturing runs and continued progression through clinical trials for several

programs. Expenses for AL014 increased as well as for other early stage programs as investment in research and clinical pipeline continues.

G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2020 were $14.6 million compared to $5.8 million for the same period in 2019. This increase was primarily due to an increase in personnel-related expenses due to increased headcount to support the advancement of the clinical and pre-clinical programs and an increase in legal costs associated with our ongoing arbitration proceedings for certain intellectual property matters.

Net Loss. For the quarter ended March 31, 2020, Alector reported a net loss of $40.0 million, compared to a net loss of $18.6 million for the same period in 2019.

Cash Position. Cash, cash equivalents, and marketable securities were $548.5 million as of March 31, 2020.

AbbVie Announces Final Results of Exchange Offers for Allergan Notes

On May 13, 2020 AbbVie Inc. (NYSE:ABBV) ("AbbVie") reported the final results of the offers to exchange (each, an "Exchange Offer" and, collectively, the "Exchange Offers") any and all outstanding notes of certain series issued by Allergan Finance, LLC ("Allergan Finance"), Allergan, Inc. ("Allergan Inc"), Allergan Sales, LLC ("Allergan Sales") and Allergan Funding SCS ("Allergan Funding" and, together with Allergan Finance, Allergan Inc and Allergan Sales, "Allergan") (the "Allergan Notes") for new notes to be issued by AbbVie (the "AbbVie Notes") and the related consent solicitations (each, a "Consent Solicitation" and, collectively, the "Consent Solicitations") being made by AbbVie on behalf of Allergan to adopt certain amendments to each of the indentures (each, an "Allergan Indenture") governing the Allergan Notes (Press release, AbbVie, MAY 13, 2020, View Source [SID1234557872]). The Exchange Offers and Consent Solicitations expired at 5:00 p.m., New York City time, on May 12, 2020 (the "Expiration Date").

On the early participation date of November 7, 2019 (the "Early Participation Date"), requisite consents were received and supplemental indentures were executed eliminating substantially all of the covenants, restrictive provisions, events of default and any guarantees of the related Allergan Notes in each Allergan Indenture. Such supplemental indentures will become operative upon settlement of the Exchange Offers, which is expected to occur on May 14, 2020 (the "Settlement Date").

The Exchange Offers and Consent Solicitations were commenced in connection with AbbVie’s acquisition of Allergan plc (the "Acquisition") and were made pursuant to the terms and subject to the conditions set forth in the confidential offering memorandum and consent solicitation statement, dated October 25, 2019, and the related letter of transmittal, each as amended by the press releases dated November 18, 2019, December 20, 2019, January 27, 2020, February 24, 2020, March 9, 2020, March 23, 2020, April 6, 2020, April 20, 2020, April 27, 2020 and May 5, 2020 (collectively, the "Offering Documents"), and were conditioned upon the closing of the Acquisition, which was completed on May 8, 2020. As of the Expiration Date, all conditions to the Exchange Offers and Consent Solicitations were satisfied.

As of the Expiration Date, an aggregate of $13,994,942,000 principal amount of Allergan USD Notes (as defined below) and an aggregate of €3,064,769,000 principal amount of Allergan Euro Notes (as defined below) had been validly tendered and not validly withdrawn as set forth in the table below:

For each $1,000 principal amount of Allergan USD Notes or €1,000 principal amount of Allergan Euro Notes validly tendered and not validly withdrawn at or prior to the Early Participation Date, eligible holders of such Allergan USD Notes or Allergan Euro Notes are eligible to receive on the Settlement Date an early participation payment of $1.00 or €1.00, as applicable, in cash, even if on such Settlement Date such eligible holder is no longer the holder of record of such Allergan Notes. In addition, for each $1,000 principal amount of Allergan USD Notes or €1,000 principal amount of Allergan Euro Notes validly tendered and not validly withdrawn prior to the Expiration Date, eligible holders are eligible to receive on the Settlement Date $1,000 principal amount of the AbbVie Notes of the applicable series or €1,000 principal amount of the AbbVie Notes of the applicable series, as applicable.

Each AbbVie Note issued in the Exchange Offers for a validly tendered Allergan Note will have an interest rate and maturity date that is identical to the interest rate and maturity date of the tendered Allergan Note, as well as identical interest payment dates and optional redemption prices. No accrued and unpaid interest is payable upon acceptance of any Allergan Notes in the Exchange Offers and Consent Solicitations. However, the first interest payment on the AbbVie Notes will include the accrued and unpaid interest from the applicable Allergan Notes tendered in exchange therefor so that a tendering eligible holder will receive the same interest payment it would have received had its Allergan Notes not been tendered in the Exchange Offers and Consent Solicitations. The AbbVie Notes will be AbbVie’s general, unsecured senior obligations, and will rank equally in right of payment with all of AbbVie’s existing and future unsecured senior indebtedness, liabilities and other obligations.

In this news release, references to the "Allergan Euro Notes" collectively refer to (i) the Floating Rate Notes due 2020 issued by Allergan Funding, (ii) the 0.500% Senior Notes due 2021 issued by Allergan Funding, (iii) the 1.500% Senior Notes due 2023 issued by Allergan Funding, (iv) the 1.250% Senior Notes due 2024 issued by Allergan Funding, (v) the 2.625% Senior Notes due 2028 issued by Allergan Funding and (vi) the 2.125% Senior Notes due 2029 issued by Allergan Funding. References to the "Allergan USD Notes" collectively refer to (i) the 3.375% Senior Notes due 2020 issued by Allergan Inc, (ii) the 4.875% Senior Notes due 2021 issued by Allergan Sales, (iii) the 5.000% Senior Notes due 2021 issued by Allergan Sales, (iv) the 3.450% Senior Notes due 2022 issued by Allergan Funding, (v) the 3.250% Senior Notes due 2022 issued by Allergan Finance, (vi) the 2.800% Senior Notes due 2023 issued by Allergan Inc, (vii) the 3.850% Senior Notes due 2024 issued by Allergan Funding, (viii) the 3.800% Senior Notes due 2025 issued by Allergan Funding, (ix) the 4.550% Senior Notes due 2035 issued by Allergan Funding, (x) the 4.625% Senior Notes due 2042 issued by Allergan Finance, (xi) the 4.850% Senior Notes due 2044 issued by Allergan Funding and (xii) the 4.750% senior notes due 2045 issued by Allergan Funding. The Allergan USD Notes and the Allergan Euro Notes are referred to herein collectively as the "Allergan Notes."

Documents relating to the Exchange Offers and Consent Solicitations were only distributed to eligible holders of Allergan Notes who completed and returned an eligibility form confirming that they were either a "qualified institutional buyer" as defined in Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"), or not a "U.S. person" and outside the United States within the meaning of Regulation S under the Securities Act. The complete terms and conditions of the Exchange Offers and Consent Solicitations are described in the Offering Documents.

This news release does not constitute an offer to sell or purchase, or a solicitation of an offer to sell or purchase, or the solicitation of tenders or consents with respect to, any security. No offer, solicitation, purchase or sale will be made in any jurisdiction in which such an offer, solicitation or sale would be unlawful. The Exchange Offers and Consent Solicitations were made solely pursuant to the Offering Documents and only to such persons and in such jurisdictions as are permitted under applicable law.

The AbbVie Notes offered in the Exchange Offers have not been registered under the Securities Act or any state securities laws. Therefore, the AbbVie Notes may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and any applicable state securities laws.