On May 2, 2017 Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation for more patients, reported rapid progress in advancing the company’s strategic vision, including the completion of a $50 million Series B financing; in-licensing a clinical-stage program from Novartis to support the use of stem cell transplantation in a variety of disease settings; and a strategic partnership with Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies (Press release, Magenta Therapeutics, MAY 2, 2017, View Source [SID1234520733]).

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The financing announced today is intended to fuel development of innovative product candidates across multiple aspects of transplantation medicine, including more precise preparation of patients, stem cell harvesting and stem cell expansion. The Series B round, which was oversubscribed, was led by GV (formerly Google Ventures), with participation from all existing investors, including Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. The financing also included Casdin Capital and other crossover investors, as well as Be The Match BioTherapies, a subsidiary of National Marrow Donor Program (NMDP)/Be The Match, the world’s leading organization focused on saving lives through bone marrow and umbilical cord blood transplantation.

"Magenta has quickly established itself as a nexus of innovation in stem cell science, catalyzing interest in this area of medicine with the recognition that improvements will have profound impact on patients," said Jason Gardner, D. Phil., chief executive officer, president and cofounder of Magenta Therapeutics. "We aspire to accelerate products that could unleash the potential of transplantation to more patients, including those with autoimmune diseases, genetic blood disorders and cancer. The resounding interest in Magenta from such a high-quality set of investors is a testament to our solid progress since launch, including building a world-class team and a robust pipeline, and generating promising early data."

MGTA-456: Investigational Product Addressing Significant Unmet Need in Stem Cell Transplant
The clinical-stage program in-licensed by Magenta from Novartis, MGTA-456 (formerly HSC835), aims to expand the number of cord blood stem cells used in transplants to achieve superior clinical outcomes compared to standard transplant procedures, and to enable more patients to benefit from a transplant. Under this agreement, Magenta gains rights to use MGTA-456 in selected applications and will develop MGTA-456 in multiple diseases, including immune and blood diseases.

Early results published in Science[1] demonstrated the ability of MGTA-456 to significantly increase the number of umbilical cord blood stem cells. Clinical results reported in Cell Stem Cell[2] demonstrated that this approach yielded an increased expansion of stem cells.

John E. Wagner, M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota and the study’s lead author, stated: "MGTA-456 markedly shortens time to recovery, addressing one of the most significant challenges in stem cell transplantation today. MGTA-456 achieved a remarkable increase in the number of blood-forming stem cells, greater than that observed by all other methods that have been tested to date. This product has the potential to further improve cord blood transplant outcomes."

Be The Match BioTherapies Strategic Partnership Agreement
Magenta and Be The Match BioTherapies also announced today that in addition to the equity investment, the two organizations have initiated a collaboration to support their shared goals of improving transplant medicine. Magenta and Be The Match BioTherapies will explore opportunities to work together across all of Magenta’s research efforts, from discovery through clinical development. Under this agreement, Magenta may leverage Be The Match BioTherapies’ capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management, and patient outcomes data derived from the NMDP/Be The Match, which operates the largest and most diverse marrow registry in the world. NMDP/Be The Match has a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries.

"We are proud to have made our first equity investment as an organization in Magenta Therapeutics, and we share a vision to improve and advance the use of curative stem cell transplantation for patients with a wide range of diseases," said Amy Ronneberg, president of Be The Match Biotherapies.

On May 2, 2017 Aduro Biotech, Inc. (NASDAQ: ADRO) reported financial results for the first quarter ended March 31, 2017. Net loss for the first quarter of 2017 was $21.8 million, or $0.32 per share, compared to a net loss of $28.8 million, or $0.45 per share for the same period in 2016 (Filing, Q1, Aduro Biotech, 2017, MAY 2, 2017, View Source [SID1234518792]).

Cash, cash equivalents and marketable securities totaled $356.0 million at March 31, 2017, compared to $361.9 million at December 31, 2016.

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"This will be an important year for Aduro, as we generate data in our ongoing ADU-S100/STING monotherapy trial and our planned Phase 2 trial in mesothelioma, as well as look for data from Janssen’s Phase 1 trials in lung and prostate cancers evaluating LADD therapeutic candidates," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We also plan to advance our STING program into additional clinical studies in collaboration with Novartis, and the first antibody from our B-select platform, the novel anti-APRIL antibody, is expected to be cleared for clinical testing this year. With ten product candidates in our diversified portfolio and a healthy balance sheet, we are in a strong position to continue to advance our pipeline and build a leading immunotherapy company."
Key Recent Accomplishments

•
Established a clinical collaboration with Merck to evaluate the combination of Aduro’s LADD agent CRS-207 with Merck’s KEYTRUDA (pembrolizumab) in a Phase 2 study in gastric cancer

•
Entered into an exclusive license agreement with Stanford University for the use of neoantigen identification technology in therapeutics using modified Listeria for our personalized LADD program, pLADD

•
Expanded Aduro’s Scientific Advisory Board with leading immunotherapy and oncology experts

•
Presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on BION-1301, anti-APRIL antibody, and ADU-S100 STING agonist

•
Presented at the Keystone Symposia on Cancer Immunology and Immunotherapy Conference on ADU-S100 and pLADD
Anticipated 2017 Milestones

•
Initiate Phase 2 mesothelioma trial with CRS-207 in combination with anti-PD1 in the first half of 2017 and report early results in the second half of 2017

•
Initiate Phase 2 gastric trial with CRS-207 in combination with anti-PD1 in the first half of 2017

•
Initiate Phase 1 pLADD (personalized LADD) trial in advanced gastro-intestinal cancers in the second half of 2017

•
Janssen expected to initiate Phase 1b/2 trial of ADU-214 in lung cancer and determine next steps for ADU-741 in prostate cancer in the second half of 2017

•
Report top-line findings from Phase 1 monotherapy trial of ADU-S100 in the second half of 2017

•
In collaboration with Novartis, initiate Phase 1b trial of ADU-S100 in combination with anti-PD1 in the second half of 2017

•
File Investigational New Drug Application for BION-1301, anti-APRIL antibody, in the second half of 2017

•
Initiate Phase 1 multiple myeloma trial with anti-APRIL antibody in the second half of 2017
First Quarter 2017 Financial Results
Revenue for the first quarter of 2017 was $3.8 million, compared to $4.0 million for the same period in 2016. The revenue recognized in both quarters primarily relates to deferred upfront payments under the Novartis collaboration agreement. In addition, revenue in the first quarter of 2016 included reimbursed research services of $0.2 million.

Research and development expenses were $20.6 million for the first quarter of 2017, compared to $20.9 million for the same period in 2016. Research and development expenses incurred in the first quarter of 2016 included GVAX Pancreas manufacturing and pancreatic cancer clinical trial expenses, which did not occur in 2017. The decrease in expenses was partially offset by increased costs to manufacture our B-select antibodies and increased research and development expenses for the STING platform, as well as higher personnel and facility related costs in first quarter of 2017.
General and administrative expenses were $8.3 million for the first quarter of 2017, compared to $9.0 million for the same period in 2016. This decrease was primarily due to lower consulting and professional fees.
Income tax benefit was $2.8 million for the first quarter of 2017, compared to a provision for income taxes of $3.2 million for the same period in 2016. The income tax benefit recorded for the first quarter of 2017 was due to the current benefit of federal income taxes paid in 2016.

On May 2, 2017 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) reported unaudited consolidated financial results for the quarter ended March 31, 2017. Total GAAP revenue for the first quarter of 2017 increased to $139.5 million, approximately 28% higher than the same period last year. This increase was primarily driven by growth in net sales of Makena (hydroxyprogesterone caproate injection). The company reported an operating loss of $40.0 million in the first quarter of 2017, compared with operating income of $7.4 million in the same period last year. The operating loss in the first quarter of 2017 was primarily due to a one-time payment of $60 million to Palatin Technologies, Inc. related to the bremelanotide licensing transaction. Non-GAAP adjusted EBITDA increased approximately 21% to $57.6 million in first quarter of 2017, as compared to the corresponding period in 2016.1

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"Today we are reaffirming our 2017 annual revenue guidance on our existing commercial portfolio of Makena, Feraheme and CBR based on the strong underlying fundamentals of those products," stated William Heiden, AMAG’s president and chief executive officer. "While our commercial team drove higher physician-level Makena demand (distributor shipments to end users) in the first quarter of 2017 compared to the fourth quarter of 2016, first quarter 2017 ex-factory sales of Makena (shipments to distributors) decreased compared to the fourth quarter of 2016. The difference between demand and ex-factory sales was primarily due to a decline in channel inventory, as well as one-time items impacting net price in the first quarter of 2017."

The company today also separately announced positive Phase 3 clinical data evaluating the safety of Feraheme (ferumoxytol) compared to Injectafer (ferric carboxymaltose injection) in adults with iron deficiency anemia (IDA). The study achieved all primary and secondary safety and efficacy endpoints and will be the basis for a supplemental new drug application (sNDA) filing planned in mid-2017 to broaden the Feraheme label beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment.

"We made strong progress against key 2017 goals with today’s announcement of a successful Phase 3 study for Feraheme, the April filing of an sNDA for the Makena subcutaneous auto-injector, and the closing of two transactions that expand our product portfolio and position AMAG well for sustainable long-term growth," added Mr. Heiden. "Our license agreements for IntrarosaTM and bremelanotide represent significant opportunities to address unmet medical needs in women’s health, and combined with Makena and our CBR offerings, demonstrate AMAG’s growing commitment to women’s healthcare."

1 See summaries of GAAP to non-GAAP adjustments at the conclusion of this press release.

1

First Quarter 2017 and Recent Business Highlights:
•
Generated strong revenues, including 33% growth in Makena sales and 7% growth in Feraheme sales over the first quarter of 2016
•
Increased Makena market share2 by 2% to 44% over the fourth quarter of 2016
•
Ended the quarter with $558.4 million of cash and investments
•
Filed an sNDA with the U.S. Food and Drug Administration (FDA) in April 2017 for the Makena subcutaneous auto-injector, with an anticipated six month review timeline
•
Announced positive Phase 3 clinical data evaluating the safety of Feraheme compared to Injectafer in adults with IDA (see separate press release dated May 2, 2017)
•
Closed two licensing transactions in the women’s health space for rights to Intrarosa and bremelanotide
•
Initiated the hiring of a new ~150-person women’s health commercial team to support the anticipated mid-2017 launch of Intrarosa
•
Advanced ongoing clinical work with our partner Palatin for the submission of a new drug application for bremelanotide in early 2018

First Quarter Ended March 31, 2017 (unaudited)
Financial Results (GAAP Basis)
Total revenues for the first quarter of 2017 were $139.5 million, compared with $109.3 million in the first quarter of 2016. Net product sales of Makena were $86.5 million in the first quarter of 2017, compared with $65.0 million in the same period last year. Sales of Feraheme and MuGard totaled $26.1 million in the first quarter of 2017, compared with $24.5 million in the first quarter of 2016. Service revenue from Cord Blood Registry (CBR) totaled $26.9 million in the first quarter of 2017, compared with $19.5 million in the same period last year.

Costs and expenses, including costs of product sales and services totaled $179.5 million in the first quarter of 2017, compared with $101.9 million for the same period in 2016. This increase was primarily due to (i) a one-time, upfront payment of $60 million to Palatin pursuant to the terms of the licensing transaction for bremelanotide, (ii) research and development expenses of $4.4 million related to clinical work performed by Palatin, (iii) higher cost of products sold of $9.3 million, of which $7.4 million was an increase in amortization expense related to Makena intangible asset, and (iv) higher selling, general and administrative expenses of $7.2 million related to commercial activities to support our growing product portfolio.

The higher expenses in the first quarter of 2017 resulted in an operating loss of $40.0 million (including the one-time payment of $60 million to Palatin), compared with operating income of $7.4 million for the same period last year. The company reported a net loss of $36.6 million, or $1.06 per basic and diluted share, for the first quarter of 2017, compared with a net loss of $7.5 million, or $0.22 per basic share and diluted share, for the same period in 2016.

Financial Results (Non-GAAP Basis)1
Non-GAAP revenue totaled $140.8 million in the first quarter of 2017, up from $117.9 million in the first quarter of 2016. Non-GAAP CBR service revenue totaled $28.3 million in the first quarter of 2017, compared with $28.1 million in the first quarter of 2016. The difference between GAAP and non-GAAP revenue represents CBR purchase accounting adjustments related to deferred revenue.

Total costs and expenses on a non-GAAP basis totaled $83.2 million in the first quarter of 2017, compared with $70.4 million in the first quarter of 2016.

Non-GAAP adjusted EBITDA for the first quarter of 2017 was $57.6 million, resulting in an adjusted EBITDA margin of 41%. This compares non-GAAP adjusted EBITDA of $47.5 million in the first quarter of 2016, with an adjusted EBITDA margin of 40%. The company maintained a strong adjusted EBITDA margin while also investing significantly in the development of its current and future products to create long-term value.

2 Company estimates Makena market share based on distributor dispensing data and all other market share based on physician market research data conducted by AMAG.

2

Balance Sheet Highlights
As of March 31, 2017, the company’s cash and investments totaled approximately $558.4 million and total debt (principal amount outstanding) was approximately $1.02 billion.

"We have updated our full year 2017 financial guidance to include revenue and expenses related to Intrarosa and are reiterating full year 2017 revenue guidance for our currently marketed products," said Ted Myles, AMAG’s chief financial officer. "During the quarter and throughout 2017, we have and will continue to invest in the launch of Intrarosa, potential expansion of the label for Feraheme, development work to support the bremelanotide NDA, and potential approval and launch of the Makena subcutaneous auto-injector. We believe these investments will deliver long-term value to our shareholders."

Updated 2017 Financial Guidance

2017 GAAP Guidance

2017 Non-GAAP Guidance
$ in millions

Previous
Updated

Previous3
Updated3
Makena sales

$410 – $440
$410 – $440

$410 – $440
$410 – $440
Feraheme and MuGard sales

$100 – $110
$100 – $110

$100 – $110
$100 – $110
CBR revenue

$110 – $120
$110 – $120

$115 – $1254
$115 – $1254
Intrarosa

—
$5 – $15

—
$5 – $15
Total revenue

$620 – $670
$625 – $685

$625 – $675
$630 – $690

Net income (loss)

$19 – $60
($88) – ($44)

N/A
N/A
Operating income (loss)

$103 – $173
($72) – $1

N/A
N/A
Adjusted EBITDA

N/A
N/A

$270 – $340
$210 – $260

On May 1, 2017 Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX:AEZS) (the "Company") reported that the ZoptEC Phase 3 clinical study of Zoptrex (zoptarelin doxorubicin) in women with locally advanced, recurrent or metastatic endometrial cancer did not achieve its primary endpoint of demonstrating a statistically significant increase in the median period of overall survival of patients treated with Zoptrex as compared to patients treated with doxorubicin (Press release, AEterna Zentaris, MAY 1, 2017, View Source [SID1234518774]).

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Dr. Richard Sachse, the Company’s Chief Scientific Officer, stated, "The median overall survival period for patients treated with Zoptrex was 10.9 months compared to 10.8 months for patients treated with doxorubicin. This is not a statistically significant, clinically meaningful increase in overall survival and thus the ZoptEC Phase 3 clinical study did not meet its primary endpoint. In addition, Zoptrex generally performed no better than the comparator drug with respect to the secondary efficacy endpoints. For example, the median period of progression-free survival of the patients in the Zoptrex arm of the study was identical to that for patients in the doxorubicin arm. Finally, there was no meaningful difference between the two arms with respect to safety; the number of patients with cardiac disorders was similar – eight in the Zoptrex arm and nine in the doxorubicin arm. Therefore, the results of the study are not supportive to pursue regulatory approval."

David A. Dodd, the President and Chief Executive Officer of the Company, stated, "We are very disappointed with the outcome of the ZoptEC Phase 3 clinical study. Based on this outcome, we do not anticipate conducting clinical trials of Zoptrex with respect to any other indications. I would like to thank my colleagues within Aeterna Zentaris and our external team of clinical investigators for their dedication to and contributions on this project." Commenting on the Company’s plans, Mr. Dodd continued, "Our focus has now shifted entirely to filing our new drug application for Macrilen and, if the product is approved, to its commercial launch as soon as possible. We will also optimize our resources to be consistent with our focus on Macrilen-related efforts. We continue to believe in the potential that Macrilen provides for us to become a focused specialty pharmaceutical company. Our intention is to submit the Macrilen NDA in the third quarter of 2017 and, if the product receives FDA approval, to commercially launch the product in the first quarter of 2018."

Trieza Therapeutics is a new company developing immunomodulatory oncolytic viruses for the treatment of cancer (Company Web Page, MPM Capital, MAY 1, 2017, View Source [SID1234518761]). Founded as a spin-out from Potenza Therapeutics, Trieza has a portfolio containing multiple pre-clinical candidates.

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