On November 3, 2016 Juno Therapeutics, Inc. (NASDAQ: JUNO), a biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize the treatment of cancer, reported that seven oral and four poster presentations detailing updated clinical and preclinical results generated in partnership with its collaborators will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Juno, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219226 [SID1234516282]). Senior executives will also review results and provide an update on Juno’s clinical development program at an analyst and investor event, which will also be available via webcast.

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"Presentations at ASH (Free ASH Whitepaper) will showcase new data that support our best-in-class CAR T strategy across a range of B-cell malignancies," said Mark J. Gilbert, M.D., Juno’s Chief Medical Officer. "The responses we are seeing with our product candidates, including updated JCAR017 results in adults with relapsed/refractory NHL, demonstrate the potential of our CAR T programs for patients in need of more treatment options. We are also learning more about cell characterization and toxicities, which are critical to improving the patient experience, our product candidates’ benefit-to-risk ratio, and potentially moving CAR T treatment into earlier lines of therapy."
New data with JCAR017 in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), which is a subtype of NHL, and final data from the PLAT-02 trial for pediatric patients with relapsed/refractory acute lymphoblastic leukemia (ALL) will be presented. JCAR017 uses a defined CD4:CD8 cell composition and 4-1BB as the costimulatory domain, which differentiates it from other CD19-directed CAR T product candidates advancing to commercialization. In addition, final data from a Phase I trial with JCAR014 in high-risk, ibrutinib-refractory patients with chronic lymphocytic leukemia (CLL) will also be presented in an oral presentation.
The following will be presented at ASH (Free ASH Whitepaper):
Oral Presentations
CD19 CAR T Cells Are Highly Effective in Ibrutinib-Refractory Chronic Lymphocytic Leukemia (Abstract #56)
Presenter: Cameron J. Turtle, M.B.B.S., Ph.D., Fred Hutchinson Cancer Research Center
Date: Saturday, December 3, 2016, 7:45 a.m. Pacific Time
Location: San Diego Convention Center, Room 6AB
Implications of Concurrent Ibrutinib Therapy on CAR T-Cell Manufacturing and Phenotype and on Clinical Outcomes Following CD19-Targeted CAR T-Cell Administration in Adults with Relapsed/Refractory CLL (Abstract #58)
Presenter: Mark B. Geyer, M.D., Memorial Sloan Kettering Cancer Center
Date: Saturday, December 3, 2016, 8:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6AB
CD19 CAR T Cell Products of Defined CD4:CD8 Composition and Transgene Expression Show Prolonged Persistence and Durable MRD-Negative Remission in Pediatric and Young Adult B-Cell ALL (Abstract #219)
Presenter: Rebecca Gardner, M.D., Seattle Children’s Research Institute
Date: Saturday, December 3, 2016, 4:30 p.m. Pacific Time
Location: Marriott Marquis San Diego Marina, Marriott Grand Ballroom Salons 11-13
Minimal Residual Disease Negative Complete Remissions Following Anti-CD22 Chimeric Antigen Receptor (CAR) in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL) (Abstract #650)
Presenter: Nirali Shah, Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute
Date: Monday, December 5, 2016, 7:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6CF
Creation of The First Non-Human Primate (NHP) Model That Faithfully Recapitulates Chimeric Antigen Receptor (CAR) T cell-mediated Cytokine Release Syndrome (CRS) and Neurologic Toxicity Following B Cell-directed CAR T Cell Therapy (Abstract #651)
Presenter: Agne Taraseviciute, Seattle Children’s Research Institute
Date: Monday, December 5, 2016, 7:15 a.m. Pacific Time
Location: San Diego Convention Center, Room 6CF
Decreased Rates of Severe CRS Seen with Early Intervention Strategies for CD19 CAR T Cell Toxicity Management (Abstract #586)
Presenter: Rebecca Gardner, M.D., Seattle Children’s Research Institute
Date: Monday, December 5, 2016, 7:45 a.m. Pacific Time
Location: Marriott Marquis San Diego Marina, Marriott Grand Ballroom Salons 2-4
EBV-Specific Donor Cells Transduced to Express a High-Affinity WT1 TCR Can Prevent Recurrence in Post-HCT Patients with High-Risk AML (Abstract #1001)
Presenter: Aude G. Chapuis, M.D., Fred Hutchinson Cancer Research Center
Date: Monday, December 5, 2016, 3:45 p.m. Pacific Time
Location: San Diego Convention Center, Room 24
Poster Presentations
Biomarkers of Cytokine Release Syndrome and Neurotoxicity after CD19 CAR-T Cells and Mitigation of Toxicity by Cell Dose (Abstract #1852)
Presenter: Cameron J. Turtle, M.B.B.S., Ph.D., Fred Hutchinson Cancer Research Center
Date: Saturday, December 3, 2016, 5:30 p.m. – 7:30 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Preclinical Analyses Support Clinical Investigation of Combined Anti-CD19 CAR-T Cell, JCAR017 with Ibrutinib for the Treatment of Chronic Lymphocytic Leukemia (Abstract #3231)
Presenter: Jim Qin, Senior Research Associate, Juno Therapeutics
Date: Sunday, December 4, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Transcend NHL 001: Immunotherapy with the CD19-Directed CAR T Cell Product JCAR017 Results in High Complete Response Rates in Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma (Abstract #4192)
Presenter: Jeremy S. Abramson, Massachusetts General Hospital Cancer Center
Date: Monday, December 5, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH
Identification of Small Molecule Modulators to Enhance the Therapeutic Properties of Chimeric Antigen Receptor T Cells (Abstract #4712)
Presenter: Jonathan Rosen, Ph.D., Senior Director, Research & Technology Core, Fate Therapeutics
Date: Monday, December 5, 2016, 6:00 p.m. – 8:00 p.m. Pacific Time
Location: San Diego Convention Center, Hall GH

On November 3, 2016 PDL BioPharma, Inc. (PDL or the Company) (NASDAQ: PDLI) reported financial results for the third quarter ended September 30, 2016 including:

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Total revenues of $53.6 million and $177.8 million for the three and nine months ended September 30, 2016, respectively(Press release, PDL BioPharma, NOV 3, 2016, View Source [SID1234516311]).

GAAP diluted EPS of $0.08 and $0.45 for the three and nine months ended September 30, 2016, respectively.

GAAP net income attributable to PDL’s shareholders of $13.9 million and $73.9 million for the three and nine months ended September 30, 2016, respectively.

Non-GAAP net income of $18.9 million and $118.2 million for the three and nine months ended September 30, 2016, respectively.

The largest component of the difference in non-GAAP net income compared to GAAP net income is the exclusion of (i) the mark-to-market reduction in fair value of our investments in royalty rights and (ii) the amortization of intangible assets. A full reconciliation of all components of the GAAP to non-GAAP quarterly financial results can be found in Table 4 at the end of this release.

Revenue Highlights
Total revenues of $53.6 million for the three months ended September 30, 2016 included:
Royalties from PDL’s licensees to the Queen et al. patents of $15.0 million, which consisted of royalties earned on sales of Tysabri under a license agreement;
Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $16.1 million, which consisted of the change in estimated fair value of our royalty right assets, primarily related to the Depomed, Inc., University of Michigan and AcelRx Pharmaceuticals, Inc. royalty rights acquisitions;
Interest revenue from notes receivable financings to late-stage healthcare companies of $8.6 million; and
Product revenues from sales of Tekturna and Tekturna HCT in the United States and Rasilez and Rasilez HCT in the rest of the world of $14.1 million.
Total revenues decreased by 57 percent for the three months ended September 30, 2016, when compared to the same period in 2015.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc. PDL continues to receive royalties on sales of Tysabri. The duration of this royalty payment is based on the sales of product manufactured prior to patent expiry, the amount of which is uncertain.
The increase in royalty rights – change in fair value was driven by the $9.6 million increase in the fair value of the Depomed royalty rights assets primarily due to a $5.0 million milestone payment based on FDA approval of Invokamet XR, a Type 2 diabetes drug in our Depomed portfolio, an adjustment to the timing of its estimated cashflows and a reduction in discount rate.
PDL received $15.3 million in net cash royalty and milestone payments from its royalty rights in the third quarter of 2016, compared to $6.9 million for the same period of 2015.
The decrease in interest revenues was primarily due to ceasing to recognize interest from Direct Flow Medical, Inc. notes receivable.
Product revenues were derived from sales of Tekturna and Tekturna HCT in the United States and Rasilez and Rasilez HCT in the rest of the world (collectively, the Noden Products). Pursuant to the purchase agreement, when Noden Pharma DAC (Noden) acquired the exclusive worldwide rights to manufacture, market, and sell the Noden Products from Novartis. Novartis continued distributing the Noden Products during the third quarter of 2016 and transferred profits with Noden on a net basis (i.e. net of cost of manufacturing and a fee to Novartis). Noden is commercializing the products in the U.S. as of the fourth quarter of 2016.
Total revenues decreased by 57 percent for the nine months ended September 30, 2016, when compared to the same period in 2015.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc.
The decrease in royalty rights – change in fair value was driven by the $19.2 million decrease in the fair value of the Depomed royalty rights asset, and a $3.4 million decrease in the fair value of the University of Michigan royalty right asset.
PDL received $47.2 million in net cash royalty payments and milestone payments from its acquired royalty rights in the nine months ended September 30, 2016, compared to $9.0 million for the same period of 2015.
Product revenues and interest revenue variances were the same as the three months ended September 30, 2016.
Operating Expense Highlights
Operating expenses were $21.0 million for the three months ended September 30, 2016, compared to $8.5 million for the same period of 2015. The increase in operating expenses for the three months ended September 30, 2016, as compared to the same period in 2015, was primarily a result of the product sales segment acquisition, contributing an additional $6.0 million of acquisition intangible amortization, $2.1 million in a change in fair value in acquisition-related contingent consideration, $1.9 million in research and development costs for the completion of a pediatric trial for the acquired branded prescription medicines Tekturna by Noden and acquisition related costs of $0.5 million. General and administrative expenses increased by $1.9 million, of which $1.1 million relates to an increased headcount and expenses due to the Noden related product acquisitions and $0.3 million relates to additional stock-based compensation expenses and an increase in legal services mostly related to ongoing legal proceedings.
Operating expenses were $40.7 million for the nine months ended September 30, 2016, compared to $23.5 million for the same period of 2015. The increase in operating expenses for the nine months ended September 30, 2016, as compared to the same period in 2015, was the result of the expenses related to the acquisition of the Noden Products.
Other Financial Highlights
PDL had cash, cash equivalents, and investments of $114.6 million at September 30, 2016, compared to $220.4 million at December 31, 2015.
The decrease was primarily attributable to the acquisition of a business, net of cash of $109.9 million, the purchase of a certificate of deposit for $75.0 million, the purchase of additional royalty rights for $59.5 million, repayment of the March 2015 Term Loan for $25.0 million, payment of dividends of $16.4 million, an additional note receivable purchase of $8.0 million, the purchase of short-term investments of $8.0 million, and the payment of debt issuance costs of $0.3 million, partially offset by the repayment of a note receivable balance of $54.7 million, proceeds from royalty right payments of $47.2 million, proceeds from the sale of available-for-sale securities of $1.7 million, cash received from a noncontrolling investor of $0.3 million and cash generated by operating activities of $86.1 million.
Net cash provided by operating activities in the nine months ended September 30, 2016 was $86.1 million, compared with $231.4 million in the same period in 2015.

vTv Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, vTv Therapeutics, 2017, NOV 3, 2016, View Source [SID1234521321]).

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On November 3, 2016 GTx, Inc. (Nasdaq: GTXI) reported financial results for the third quarter ended September 30, 2016 and provided an update on the Company’s clinical development activities (Press release, GTx, NOV 3, 2016, View Source;p=RssLanding&cat=news&id=2219024 [SID1234516223]). The Company has three ongoing Phase 2 clinical trials: two trials evaluating enobosarm as a potential treatment for women with advanced androgen receptor positive breast cancer and another trial assessing enobosarm as a potential treatment for stress urinary incontinence in postmenopausal women.

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"We made excellent progress during the quarter, including demonstrating clinical benefit in Stage 1 of the 9 mg dose group which enabled us to advance this dose group to the second and final stage of the ER+/AR+ breast cancer trial," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "In addition we are pleased with the continued financial support of key existing investors who participated in our equity offering last month which enables us to continue to pursue key milestones in the coming months for our breast cancer and stress urinary incontinence programs, as well as our SARD technology."

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product candidate, a selective androgen receptor modulator (SARM), is being developed as a targeted treatment for two advanced breast cancer indications: (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical trials, the primary efficacy endpoint is a determination of clinical benefit (CB), which is defined as a complete response, partial response or stable disease.

ER+/AR+ breast cancer: The Company has an ongoing Phase 2 clinical trial of enobosarm (GTx-024) in women with advanced, ER+/ AR+ breast cancer. Patients receive orally-administered enobosarm (9 mg or 18 mg) daily for up to 24 months. The first stage of evaluation will be assessed among the first 18 evaluable patients for each cohort. If at least 3 of 18 patients per cohort achieve CB at week 24, the trial will proceed to the second stage of enrollment. The primary efficacy objective of the trial is clinical benefit response following 24 weeks of treatment, which, under the clinical trial protocol, will be demonstrated if at least 9 of 44 evaluable patients achieve CB. Those patients demonstrating CB at week 24 will continue to receive treatment for up to 24 months. The two dose cohorts in the trial will be treated independently for the purpose of assessing efficacy.

The Company announced in September 2016 that the 9 mg dose group demonstrated clinical benefit in Stage 1 of the trial allowing the 9 mg cohort to advance to Stage 2;
The Company expects to announce top-line results from Stage 1 of the 9 mg cohort before the end of 2016.
AR+ TNBC: The Company has an ongoing open-label, multi-center, multinational Phase 2 clinical trial to evaluate the efficacy and safety of orally administered enobosarm in up to 55 women with advanced, AR+ TNBC. Patients will receive 18 mg of enobosarm once daily for up to 12 months. Stage 1 of the trial will be assessed among the first 21 evaluable patients. If at least 2 of 21 patients achieve clinical benefit at week 16, then the trial will proceed to Stage 2 of enrollment of up to a total of 41 evaluable patients. The primary efficacy objective of the trial is clinical benefit response following 16 weeks of treatment in 41 evaluable patients.

The Company expects to have sufficient data from Stage 1 of the trial in the first quarter of 2017 to determine if patient enrollment should continue into Stage 2 of the trial.
SARMs in Non-Oncologic Indications: The Company is exploring SARMs as potential treatments for both stress urinary incontinence (SUI) and Duchenne muscular dystrophy (DMD), a rare disease characterized by progressive muscle degeneration and weakness.

Stress Urinary Incontinence: Enrollment in the Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm in women with SUI is ongoing. This is the first clinical trial to evaluate a SARM for SUI. The Company believes that developing an oral therapy to treat the growing number of women who suffer a diminished quality of life from stress urinary incontinence presents a unique commercial opportunity, especially since current therapies may sometimes involve invasive procedures. The Company expects to have data from the trial during the first half of 2017.

Duchenne muscular dystrophy: Preclinical studies have confirmed the beneficial effects from SARMs in mice genetically altered to simulate DMD, compared to control groups. The Company continues to pursue a potential strategic collaboration with biopharma companies experienced in orphan drug development.

SARDs in Prostate Cancer: Our Selective Androgen Receptor Degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), including those who do not respond or are resistant to currently approved therapies. The Company believes that its SARD compounds will degrade multiple forms of the androgen receptor, including AR splice variants, such as AR-V7, along with mutant versions of the receptor.

Castration-Resistant Prostate Cancer: Lead SARD compounds are undergoing further preclinical development, including formulation and pharmacokinetic studies. The Company plans to initiate its first human clinical trial with a SARD in 2017.

Third Quarter and Nine Months 2016 Financial Results

In October 2016, the Company completed a registered direct offering of its common stock resulting in net proceeds of approximately $13.6 million. Under the terms of the offering, the Company sold 17.3 million shares of its common stock at a purchase price of $0.81 per share, which was the consolidated closing bid price of the Company’s common stock on October 11, 2016. The investors in the offering consisted of certain existing shareholders of the Company, including J.R. Hyde, III, its largest shareholder and Lead Director of the Company’s Board of Directors (the "Board"), Robert J. Wills, the Board’s Executive Chairman, and Marc S. Hanover, the Company’s Chief Executive Officer and a member of the Board.
As of September 30, 2016, cash and short-term investments were $13.4 million, which does not include the proceeds noted above from our recent equity financing, compared to $29.3 million at December 31, 2015.
Research and development expenses for the quarter ended September 30, 2016 were $4.6 million compared to $3.8 million for the same period of 2015.
General and administrative expenses were $2.3 million for the quarter ended September 30, 2016 compared to $2.0 million for the same period of 2015.
The net loss for the quarter ended September 30, 2016 was $6.9 million compared to net income of $34.9 million for the same period in 2015. Net income for the quarter ended September 30, 2015 included a non-cash gain of $40.7 million due to the change in the fair value of the Company’s warrant liability. During the first quarter of 2016, the Company recorded a non-cash reclassification of this warrant liability to stockholders’ equity due to modification of these warrants. No adjustments to the fair value of these warrants are required subsequent to the first quarter of 2016.
The net loss for the nine months ended September 30, 2016 was $10.9 million compared to a net loss of $15.5 million for the same period of 2015. The nine months ended September 30, 2016 included a non-cash gain of $8.2 million due to the change in the fair value of the Company’s warrant liability, recorded during the first quarter of 2016. The nine months ended September 30, 2015 included a non-cash gain of $352,000 due to the change in fair value of the Company’s warrant liability.
GTx had approximately 141.9 million shares of common stock outstanding as of September 30, 2016. Additionally, there remain warrants outstanding to purchase approximately 64.3 million shares of GTx common stock at an exercise price of $0.85 per share.

On November 3, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported that data from five abstracts on the investigational protein therapeutics, luspatercept and sotatercept, will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held in San Diego, California on December 3-6, 2016 (Press release, Acceleron Pharma, NOV 3, 2016, View Source [SID1234516201]).

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The oral and poster presentations will include data from Phase 2 studies with luspatercept, which is being developed under a global partnership with Celgene for the treatment of myelodysplastic syndromes and beta-thalassemia. The clinical presentations at the conference will include updated information beyond that which is contained in the abstracts now available online on the ASH (Free ASH Whitepaper) conference website.

Oral presentations

Title:
Luspatercept Increases Hemoglobin, Decreases Transfusion Burden and Improves Iron Overload in Adults with Beta-Thalassemia (Abstract #851)
Session: 112. Thalassemia and Globin Gene Regulation: Clinical Advances in Thalassemia
Date: Monday, December 5th
Time: 3:45 p.m. PST (San Diego Convention Center, Room 7AB)

Title:
Phase-2 Study of Sotatercept (ACE-011) in Myeloproliferative Neoplasm-Associated Myelofibrosis and Anemia (Abstract #478)
Session: 634. Myeloproliferative Syndromes: Clinical: Clinical Trials with Agents Other Than JAK Inhibitors
Date: Sunday, December 4th
Time: 5:15 p.m. PST (Marriott Marquis San Diego Marina, Marriott Grand Ballroom Salons 8-9)

Poster presentations

Title:
Pharmacokinetics and Exposure-Response of Luspatercept in Patients with Anemia Due to Low- or Intermediate-1-Risk Myelodysplastic Syndromes (MDS): Preliminary Results from Phase 2 Studies (Abstract #1990)
Session: 637. Myelodysplastic Syndromes – Clinical Studies: Poster I
Date: Saturday, December 3rd
Time: 5:30 – 7:30 p.m. PST (San Diego Convention Center, Hall GH)

Title:
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low-Intermediate Risk Myelodysplastic Syndromes (MDS): Long-Term Results from Phase 2 PACE-MDS Study (Abstract #3168)
Session: 637. Myelodysplastic Syndromes – Clinical Studies: Poster II
Date:
Sunday, December 4th
Time: 6:00 – 8:00 p.m. PST (San Diego Convention Center, Hall GH)

Title:
Pharmacokinetics and Exposure-Response of Luspatercept in Patients with Beta-Thalassemia: Preliminary Results from Phase 2 Studies (Abstract #2463)
Session: 112. Thalassemia and Globin Gene Regulation: Poster II
Date: Sunday, December 4th
Time: 6:00 – 8:00 p.m. PST (San Diego Convention Center, Hall GH)

The luspatercept posters and presentation slides will be available in the "Science" section on Acceleron’s website (www.acceleronpharma.com).

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Phase 3 clinical trials are underway to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the "MEDALIST" study) and in patients with beta-thalassemia (the "BELIEVE" study). For more information, please visit www.clinicaltrials.gov.

About Sotatercept

Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in fibrosis, vascular calcification, bone mineral density and late stage erythropoiesis (red blood cell production). Acceleron and Celgene are jointly developing sotatercept as part of a global collaboration. Sotatercept is currently in multiple Phase 2 investigator initiated trials. For more information, please visit www.clinicaltrials.gov.