On August 2, 2018 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2018 (Press release, bluebird bio, AUG 2, 2018, View Source [SID1234528329]).

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"The clinical data presented this spring across our development programs in TDT, SCD and multiple myeloma have further reinforced the strength of our gene and cell therapy platforms, and we are putting tremendous effort towards bringing all four of our clinical programs to patients as soon as possible," said Nick Leschly, chief bluebird. "In the second half of the year, we anticipate reaching a significant milestone for bluebird, by filing for our first potential regulatory approval in Europe with LentiGlobin to treat TDT. As we prepare to make this important transition to a commercial company, with the potential for three initial product approvals by the end of 2020, our readiness and implementation plans are well underway. We are also investing and building for our next phase of growth through a sustainable innovation engine, and a strong development and commercial infrastructure to allow us to bring more transformative therapies to patients."

Recent Highlights

TDT

LENTIGLOBIN ACCELERATED ASSESSMENT – In July 2018, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted an accelerated assessment to LentiGlobin in transfusion-dependent β-thalassemia (TDT). The company is on track to submit a Marketing Authorization Application (MAA) to the EMA for LentiGlobin in 2018.
NEW DATA FROM NORTHSTAR AND NORTHSTAR-2 PRESENTED – At the Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018, bluebird bio presented new data from its studies of LentiGlobin in patients with TDT: Northstar (HGB-204) and Northstar-2 (HGB-207). As of the data cutoff, 7/8 non-β0/β0 patients with ≥ 6 months follow-up were producing normal or near-normal amounts of total hemoglobin (11.1 – 13.3 g/dL) and were transfusion free in Northstar-2. 8/10 of non-β0/β0 patients achieved and maintained transfusion independence for up to 3 years in Northstar. Across both studies, the safety profile was consistent with myeloablative conditioning.
FIRST PEDIATRIC PATIENT TREATED – In April 2018, the first pediatric patient was treated in Northstar-3 (HGB-212), bluebird bio’s Phase 3 study of LentiGlobin in patients with β0/β0 genotypes.
SCD

NEW DATA FROM HGB-206 PRESENTED – At EHA (Free EHA Whitepaper) in June 2018, bluebird bio presented new data from the HGB-206 study of LentiGlobin in patients with severe sickle cell disease (SCD). As of the data cutoff, all patients (n=4) in Group C with ≥ 3 months follow-up were consistently producing ≥ 30% anti-sickling HbAT87Q. The first Group C patient was generating a normal total hemoglobin of 14.2 g/dL with over 60% anti-sickling HbAT87Q at 6 months. Across all patients in the study, the safety profile was consistent with myeloablative conditioning.
MULTIPLE MYELOMA

NEW DATA FROM CRB-401 PRESENTED – At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2018, bluebird bio and Celgene Corporation presented new data from the ongoing CRB-401 Phase 1 clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 43 patients with late-stage relapsed/refractory multiple myeloma. Deep and durable responses were observed at active doses (≥150 × 106 CAR+ T cells). A median progression-free survival (PFS) of approximately one year was achieved in heavily pre-treated patients in the active doses of the dose escalation cohort. Consistent response rates were observed for both low and high BCMA expression levels. Adverse events have been manageable across doses.
CALD

PRIME DESIGNATION FOR LENTI-D – In July 2018, the EMA granted access to its Priority Medicines (PRIME) scheme for Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD). The PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly. To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical data or nonclinical data.
BREAKTHROUGH DESIGNATION FOR LENTI-D – In May 2018, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with CALD. Lenti-D previously was granted Orphan Drug designation by the FDA and EMA, as well as Rare Pediatric Disease designation by the FDA.
COMPANY

STRENGTHENED BALANCE SHEET – In July 2018, bluebird bio raised approximately $600.6 million in net proceeds through a public equity offering. bluebird bio anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations into 2022 based on the company’s current business plan.
Second Half 2018 Anticipated Milestones

TDT

Submission of a MAA to the EMA for LentiGlobin in patients with TDT and non-β0/β0 genotypes
Submission of LentiGlobin clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes to the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting
Submission of LentiGlobin clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype to the ASH (Free ASH Whitepaper) Annual Meeting
SCD

Update on the clinical development plan and registration strategy for LentiGlobin in SCD
Submission of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD to the ASH (Free ASH Whitepaper) Annual Meeting
Multiple Myeloma

Submission of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma to the ASH (Free ASH Whitepaper) Annual Meeting
Initiation by Celgene of a Phase 3 clinical study of bb2121 in third line multiple myeloma
CALD

Presentation of Lenti-D clinical data from the ongoing Starbeam clinical study in patients with CALD in the second half of 2018
Second Quarter 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2018 and December 31, 2017 were $1.46 billion and $1.61 billion, respectively.
Revenues: Total revenues were $7.9 million for the three months ended June 30, 2018 compared to $16.7 million for the three months ended June 30, 2017. The decrease of approximately $8.9 million was primarily attributable to license revenue recognized in the second quarter of 2017 as a result of out-licensing arrangements entered into during that quarter. Total revenues were $23.8 million for six months ended June 30, 2018 compared to $23.5 million for six months ended June 30, 2017. The increase of $0.3 million was primarily attributable to an overall increase in collaboration revenue for the bb2121 license and manufacturing services under the company’s agreement with Celgene, offset by a decrease in license and royalty revenue.
R&D Expenses: Research and development expenses were $115.0 million for the three months ended June 30, 2018 compared to $63.9 million for the three months ended June 30, 2017. Research and development expenses were $212.1 million for six months ended June 30, 2018 compared to $118.9 million for six months ended June 30, 2017. The increase in both periods was driven by costs incurred to advance and expand the company’s pipeline and is attributable to increased clinical trial-related costs and manufacturing costs for development programs, increased laboratory expenses, increased employee-related costs due to headcount growth, and increased license milestones and fees under the company’s strategic collaboration and license agreements.
G&A Expenses: General and administrative expenses were $41.2 million for the three months ended June 30, 2018 compared to $21.2 million for the three months ended June 30, 2017. General and administrative expenses were $76.1 million for six months ended June 30, 2018 compared to $41.5 million for six months ended June 30, 2017. The increase in both periods was attributable to increases in employee-related costs due to increased headcount to support overall growth, commercial-readiness activities, and professional and consulting fees.
Net Loss: Net loss was $146.0 million for the three months ended June 30, 2018 compared to $70.9 million for the three months ended June 30, 2017. Net loss was $261.1 million for six months ended June 30, 2018 compared to $139.6 million for six months ended June 30, 2017.

On August 2, 2018 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 second quarter ended June 30, 2018 (Press release, Agios Pharmaceuticals, AUG 2, 2018, View Source [SID1234528359]). In addition, Agios highlighted select corporate milestones and clinical data from its development programs.

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"The first half of 2018 has been productive across all aspects of our business, culminating in the recent approval and launch of our second internally discovered medicine," said David Schenkein, M.D., chief executive officer at Agios. "This achievement sets us well on the path to becoming a sustainable, multiproduct company with a thriving research engine on track to submit its 7th IND and a broad clinical development program with multiple trials planned or underway to expand our oncology and rare genetic disease portfolios."

SECOND QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS

Received full approval from the U.S. Food and Drug Administration (FDA) on July 20, 2018 for TIBSOVO (ivosidenib) for the treatment of patients with relapsed or refractory AML (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA approved test.
Entered into an exclusive license agreement with CStone Pharmaceuticals to develop and commercialize ivosidenib in Greater China, resulting in a $12 million upfront payment and the potential for $412 million in development and commercial milestones.
Initiated ACTIVATE, a global, placebo-controlled, pivotal trial for mitapivat (AG-348) in approximately 80 adults with PK deficiency who do not receive regular blood transfusions. A second pivotal trial (ACTIVATE-T) in PK deficiency patients who receive regular blood transfusions is ongoing.
Presented new and updated data from the IDH programs at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago. Links to the data presentations, including updated data from the Phase 1 trial combining ivosidenib and azacitidine in the frontline AML setting can be found here.
Secured publication of the ivosidenib Phase 1 data in patients with IDH1m advanced hematological malignancies in theNew England Journal of Medicine.
Supported publication of the results from the Pyruvate Kinase Deficiency Natural History Study in the journal Blood.
Disclosed active research programs in three rare genetic diseases: phenylketonuria, erythroid porphyria and Friedreich’s ataxia, as part of a preclinical pipeline update at the company’s Investor Day in May.
KEY UPCOMING MILESTONES

The company expects to achieve the following remaining milestones in 2018:

Cancer:

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.
Support, in conjunction with Celgene, the initiation of HO150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
Rare Genetic Diseases:

Initiate a Phase 2 proof of concept trial of mitapivat (AG-348) in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
EXPECTED FOURTH QUARTER CLINICAL DATA PRESENTATIONS

Updated data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation has been submitted to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on December 1-4 in San Diego.
Updated data in untreated AML from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
SECOND QUARTER 2018 FINANCIAL RESULTS

Revenue for the quarter ended June 30, 2018 was $40.4 million, which includes $26.4 million of collaboration revenue and $1.6 million of royalty revenue from net U.S. sales of IDHIFA under our collaboration agreements with Celgene, and $12.4 million of collaboration revenue under our agreement with CStone. Revenue for the quarter ended June 30, 2017 was $11.3 million and consisted solely of collaboration revenue under our agreements with Celgene. The year over year increase in collaboration revenue for the second quarter was primarily driven by the $15.0 million milestone related to Celgene’s filing of an MAA to the EMA for IDHIFA and $12.4 million related to the delivery of the license under the CStone Agreement.

Research and development (R&D) expenses were $86.7 million, including $9.7 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $79.8 million, including $8.2 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the mitapivat (AG-348) pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of IND enabling activities for AG-636, our DHODH inhibitor.

General and administrative (G&A) expenses were $26.6 million, including $6.8 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $16.1 million, including $4.0 million of stock-based compensation expense, for the quarter ended June 30, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO.

Net loss for the quarter ended June 30, 2018 was $68.7 million, compared to a net loss of $83.1 million for the quarter ended June 30, 2017.

Cash, cash equivalents and marketable securities as of June 30, 2018 were $936.6 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $8.9 million of cost reimbursements under our collaboration agreements with Celgene and $22.0 million received from employee stock transactions. This was offset by expenditures to fund operations of $178.1 million during the six months ended June 30, 2018.

The company expects that its cash, cash equivalents and marketable securities as of June 30, 2018, 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 second quarter 2018 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 1497883. 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 August 2, 2018, Caladrius Biosciences, Inc., a Delaware corporation (the "Company"),reported that it has entered into a First Amendment (the "Amendment") to that certain Common Stock Sales Agreement, dated February 8, 2018 (the "Original Agreement," and, together with the Amendment, the "Sales Agreement") with H.C. Wainwright & Co., LLC ("Wainwright"), as sales agent, in connection with an "at the market offering" under which the Company from time to time may offer and sell shares of its common stock, par value $0.001 per share (the "Common Stock"), having an aggregate offering price of up to $25,000,000 (the "Shares") (Filing, 8-K, Caladrius Biosciences, AUG 2, 2018, View Source [SID1234528396]). Shares sold under the Sales Agreement will be offered and sold pursuant to the Company’s Registration Statement on Form S-3, which was initially filed on July 24, 2018 and which was declared effective by the Securities and Exchange Commission (the "SEC" on August 2, 2018 (Registration No. 333-226319) (the "Registration Statement") and a prospectus supplement that the Company expects to file with the SEC relating to the Shares shortly after the filing of this Current Report on Form 8-K. The Amendment updates the Original Agreement to reflect that the Shares will be issued pursuant to the Registration Statement, which replaces the Company’s previously effective shelf registration statement.

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This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the Shares, nor shall there be any offer, solicitation or sale of the Shares in any state or country in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or country.
The opinion of the Company’s counsel regarding the validity of the Shares is filed as Exhibit 5.1 to this Current Report on Form 8-K. This opinion is also filed with reference to, and is hereby incorporated by reference into, the Registration Statement.

The Company cautions you that statements included in this Current Report on Form 8-K that are not a description of historical facts are forward-looking statements. These forward-looking statements include statements regarding the Company’s ability to sell Shares pursuant to the Sales Agreement. The inclusion of forward-looking statements should not be regarded as a representation by the Company that any of these statements, results or sales will be achieved or completed due in part to risks and uncertainties inherent in the Company’s business, including those described in the Company’s Form 10-K for the year ended December 31, 2017 filed with the SEC on March 22, 2018, as amended on April 2, 2018. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the Company undertakes no obligation to revise or update this report to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.

On August 2, 2018 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of precisely targeted oncology therapies, reported financial results for the second quarter ended June 30, 2018, and provided a business update (Press release, Corvus Pharmaceuticals, AUG 2, 2018, View Source;p=RssLanding&cat=news&id=2361869 [SID1234528637]).

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"We continue to be a leader in programs addressing the adenosine pathway, with three clinical trials currently enrolling and more than 235 patients treated to-date with our lead product candidate, an A2A receptor antagonist, CPI-444," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "We are enrolling up to 50 patients in a Phase 1/1b study of CPI-444 in combination therapy under an amended protocol focused on patients with earlier stage renal cell cancer. CPI-444 is also being studied in patients with lung cancer. We are also enrolling patients in a Phase 1/1b study of CPI-006, an anti-CD73 antibody, to treat advanced cancers, both as a monotherapy and in combination with CPI-444 and an anti-PD-1 therapy. In the fourth quarter, we expect to report updated data from several of our programs at medical meetings, which will be another important milestone in our development plans."

RECENT ACHIEVEMENTS
CPI-444: A2A Receptor Antagonist of Adenosine

Enrolling patients in a Phase 1/1b clinical trial evaluating CPI-444, the Company’s lead product candidate, administered alone and in combination with Genentech’s Tecentriq (atezolizumab), an anti-PD-L1 antibody, under an amended protocol to enroll up to 50 patients with renal cell cancer (RCC) who have failed no more than two prior treatment regimens, which must have included an anti-PD-(L)1 and a tyrosine kinase inhibitor.
○ Biomarker studies conducted by the company have shown that prior therapy with anti-PD-(L)1 may increase expression of the adenosine pathway.
○ Previous studies evaluated patients who have failed up to five (median three) prior treatment regimens.
Continued enrolling patients in the Phase 1b/2 trial, being conducted by Genentech as part of their MORPHEUS platform, which is evaluating CPI-444 and Tecentriq in up to 60 patients with non-small cell lung cancer (NSCLC) who have failed no more than two prior regimens.
CPI-006: Anti-CD73 Antibody

Began enrolling patients with advanced cancer in a Phase 1/1b clinical trial evaluating CPI-006, the Company’s anti-CD73 antibody, as a single agent and in combination with CPI-444, and in combination with an anti-PD-1. The trial is anticipated to enroll up to 350 patients and is designed to select the dose and evaluate the safety, pharmacokinetics, immune biomarkers and efficacy in patients with NSCLC, RCC, and other cancers who have failed standard therapies.
CPI-818: A small molecule ITK inhibitor

Continued pre-clinical development of the Company’s interleukin-2-inducible kinase (ITK) inhibitor and plan to submit an Investigational New Drug (IND) filing in early 2019. Tumor responses have been observed in a preclinical study in spontaneous canine T-cell lymphoma conducted at Colorado State University, College of Veterinary Medicine Flint Animal Cancer Center.
FINANCIAL RESULTS
At June 30, 2018, Corvus had cash, cash equivalents and marketable securities totaling $133.2 million. This compared to cash, cash equivalents and marketable securities of $90.1 million at December 31, 2017.

Research and development expenses for the three months ended June 30, 2018 totaled $9.7 million compared to $12.4 million for the same period in 2017. The decrease of $2.7 million was primarily due to a $2.2 million decrease in CPI-444 and CPI-006 drug manufacturing costs, a decrease of $2.2 million in CPI-444 clinical trial expense, and a decrease of $0.4 million in contracted research costs. These decreases were partially offset by an increase of $0.9 million in CPI-818 drug manufacturing costs, and increase of $0.6 million in CPI-006 clinical trial expense, and an increase of $0.6 million in personnel related costs.

General and administrative expenses for the three months ended June 30, 2018 totaled $2.5 million compared to $2.8 million for the same period in 2017. The decrease of $0.3 million was primarily due to a decrease of $0.4 million in patent and public company expenses, offset by an increase of $0.1 million in personnel costs.

The net loss for the three months ended June 30, 2018 was $11.6 million compared to $15.0 million for the same period in 2017. Total stock compensation expense for the three months ended June 30, 2018 was $1.7 million compared to $1.5 million for the same period in 2017.

On August 2, 2018, the Company entered into a Third Amendment to Lease Agreement (the "Amendment") with ARE-SD Region No. 20 (the "Landlord") to amend the Lease Agreement, dated June 24, 2014, the First Amendment to Lease dated March 23, 2017, and the Second Amendment to Lease dated April 5, 2018 (the "Amended Lease") between the Company and Landlord (Filing, 8-K, Mirati, AUG 2, 2018, View Source [SID1234528662]). The Amendment expands the size of the existing premises by adding approximately 6,100 square feet of space for an additional base rent of $4,000 per month through January 31, 2020. In addition, our share of operating expenses of the building in which the premises are located, has increased from approximately 43% to 58%. All other material terms and covenants from the Amended Lease remain unchanged.

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