On December 11, 2017 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a company committed to improving patient lives by manufacturing and delivering high quality biologics, reported financial results for the second quarter of fiscal year (FY) 2018 ended October 31, 2017, and provided an update on its contract manufacturing operations, and other corporate highlights (Press release, Peregrine Pharmaceuticals, DEC 11, 2017, View Source [SID1234522574]).

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Highlights Since July 31, 2017

"Today, we are pleased to report that the company has made great progress in its transition from an R&D focused business to a dedicated contract development and manufacturing organization (CDMO)," stated Roger J. Lias, Ph.D., president of Avid Bioservices. "In late November, the company came to an agreement with an investor group, appointing a highly qualified new board of directors consisting of three new independent members from this investor group and one mutually designated independent member in addition to myself and the two independent members previously appointed. We have now added six highly qualified and independent board members since October. In addition, we are focused on hiring experienced and successful CDMO professionals who are dedicated to revenue growth through the expansion and diversification of Avid’s client base, as evidenced by the recently announced hiring of Tracy Kinjerski as vice president of business operations. We are actively planning to expand Avid’s service offerings and enhance our manufacturing infrastructure to ensure that we are offering the highest quality services, and state-of-the-art facilities to our customers. We are also taking steps to officially change the name of the entire organization to Avid Bioservices, Inc. to formalize this transition. Lastly, we are in continued discussions with third parties regarding the divestiture of the company’s remaining R&D assets and we will keep you apprised on our progress as we advance the process."

Recent Developments at Avid Bioservices

· Established a dedicated CDMO management infrastructure with the hiring of Roger J. Lias, Ph.D., as the President of Avid Bioservices and director.
o Dr. Lias brings more than 20 years of experience in the industry having held senior management positions at several leading CDMOs including Cytovance Biologics, KBI BioPharma, Diosynth RTP (formerly Covance Biotechnology Services) and Lonza Biologics.

· Strengthened Avid’s sales and business development function with the hiring of Tracy Kinjerski as vice president of business operations.
o Ms. Kinjerski brings more than 17 years of experience with a focus in contract development and manufacturing. She is charged with driving Avid’s growth through the strategic expansion and diversification of the company’s commercial and clinical client base.

· Reconstituted the board of directors to include six independent directors, all with significant CDMO experience.
o In October 2017, Mark R. Bamforth was appointed as an independent member of the board of directors. Mr. Bamforth has 30 years of biologics leadership experience including founding two CDMOs, Brammer Bio, where he is currently the president and CEO, and Gallus BioPharmaceuticals, which was acquired by DPx Holdings B.V., the parent company of Patheon. Additionally, he served for more than 20 years in key roles at Genzyme Corporation, including 10 years as a corporate officer responsible for running global manufacturing.
o In October 2017, Patrick Walsh was appointed as an independent member of the board of directors. Mr. Walsh has a record of leading successful, high-growth CDMOs and he has also led complex laboratory and pharmaceutical manufacturing operations including parenteral and active pharmaceutical ingredients (API) on a global scale.
o In November 2017, the company entered into a settlement agreement with its largest shareholder (Ronin/SWIM) regarding the composition of Peregrine’s board of directors. Under the terms of the Agreement, on November 27, 2017, directors Steven W. King, Carlton M. Johnson, Jr., Eric S. Swartz and David H. Pohl each tendered his resignation, effective immediately, from Peregrine’s board of directors, and from the board of directors of Avid Bioservices. The vacancies created by these resignations were immediately filled by three individuals who were nominated by Ronin/SWIM for election at Peregrine’s upcoming 2017 Annual Meeting of Stockholders (Richard B. Hancock, Gregory P. Sargen and Joel McComb), and one director (Joseph Carleone, Ph.D.) who is independent of Ronin/SWIM and new to Peregrine.

• Joseph Carleone, Ph.D. (independent appointee): Dr. Carleone is Chairman of the Board of AMPAC Fine Chemicals LLC, a leading manufacturer of pharmaceutical active ingredients. Prior to this position, Dr. Carleone was President, Chief Executive Officer and director of American Pacific Corporation, a leading custom manufacturer of fine and specialty chemicals and propulsion products.
• Richard B. Hancock (Ronin/SWIM appointee): Richard (Rick) B. Hancock has worked in the biologic CDMO industry for over 30 years in various operational and executive roles, serving most recently as President and CEO of Althea Technologies, Inc., a large molecule CDMO producing a wide range of biologics, vaccines and parenteral products.
• Joel McComb (Ronin/SWIM appointee): Joel McComb is the CEO, Chairman and Co-Founder of BioSpyder Technologies, Inc. Prior to BioSpyder, Mr. McComb served as Senior Vice President and General Manager of Illumina, Inc., President of GE Healthcare’s Life Sciences and Discovery Systems division, and President of GE Healthcare’s Interventional Medicine division.
• Gregory P. Sargen (Ronin/SWIM appointee): Gregory P. Sargen currently serves as Executive Vice President – Corporate Development and Strategy of Cambrex Corporation ("Cambrex"), a global manufacturer and provider of services to life sciences companies. Prior to his current role, Mr. Sargen served as Executive Vice President and Chief Financial Officer of Cambrex.

· Expanded production capacity in the Myford facility to allow organic and significant growth using existing facilities.

o In recent months, the company expanded its capacity in its Myford facility by installing two new 2,000 liter single-use bioreactors.

2

Financial Highlights and Results

· The company maintains its manufacturing revenue guidance for the full FY 2018 of $50 million – $55 million.

· Contract manufacturing revenue from Avid’s clinical and commercial biomanufacturing services was $12.8 million for the second quarter of FY 2018 compared to $23.4 million for the second quarter of FY 2017.

· Avid’s current manufacturing revenue backlog is $33.0 million, representing estimated future manufacturing revenue to be recognized under committed contracts. Most of the backlog is expected to be recognized during the remainder of FY 2018 and into FY 2019.

· Total operating expenses for the second quarter of FY 2018 were $9.2 million, compared to $12.0 million for the second quarter of FY 2017. For the second quarter of FY 2018, total operating expenses included restructuring charges of $1.6 million associated with termination benefits including severance and other employee related costs related to a workforce reduction pursuant to a restructuring plan implemented in August 2017. The company is also actively evaluating its overall operating expenses and cost structure as a dedicated CDMO and plans to align its cost structure to match the future needs of the business.

· Research and development expenses decreased to $3.7 million in the second quarter of FY 2018 compared to $7.0 million for the second quarter of FY 2017. Over the next 60 or fewer days, the Company will continue to rapidly wind down all research and development costs to zero and plans to support only those efforts needed to pursue the license or sale of its research and development assets.

· Cost of contract manufacturing increased to $16.2 million in the second quarter of FY 2018 compared to $15.4 million for the second quarter of FY 2017.

· For the second quarter of FY 2018, selling, general and administrative expenses decreased to $3.9 million compared to $5.0 million for FY 2017.

· Peregrine’s consolidated net loss attributable to common stockholders was $14.1 million or $0.31 per share, for the second quarter of FY 2018, compared to a net loss attributable to common stockholders of $5.5 million, or $0.16 per share, for the same prior year quarter.

· Peregrine reported $27.7 million in cash and cash equivalents as of October 31, 2017, compared to $46.8 million at fiscal year ended April 30, 2017. As further discussed in the Company’s Quarterly Report on Form 10-Q, the Company plans to raise additional capital within the next six months to support its continued operations and other initiatives that will enhance its CDMO operations.

More detailed financial information and analysis may be found in Peregrine’s Quarterly Report on Form 10-Q, which will be filed with the Securities and Exchange Commission today.

Conference Call

Peregrine will host a conference call and webcast this afternoon, December 11, 2017, at 4:30 PM EST (1:30 PM PST).

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Peregrine Pharmaceuticals conference call. To listen to the live webcast, or access the archived webcast, please visit: View Source;

On December 11,2017 Immunocore Limited, the world’s leading TCR company focused on delivering first-in-class biological therapies that transform lives, reported that it has been informed by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) that IMCgp100 has been granted Promising Innovative Medicines (PIM) designation for the treatment of patients with metastatic uveal melanoma(Press release, Immunocore, DEC 11, 2017, View Source [SID1234522505]).

PIM designation is an early indication that IMCgp100 is a promising candidate for the UK’s Early Access to Medicines Scheme (EAMS), intended for the treatment, diagnosis or prevention of metastatic uveal melanoma. This is based on early Phase I clinical trial data published at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November. IMCgp100 will be a suitable candidate for entry into Step II of the EAMS process, for which Immunocore is in a pivotal registrational clinical trial in patients with metastatic uveal melanoma. Following this, IMCgp100 will enter the EAMS scientific opinion assessment step.

For more information on clinical trials involving IMCgp100, please visit clintrials.gov.

James Sandy, Chief Development Officer at Immunocore, commented: "We are delighted by the MHRA’s decision to award PIM designation to IMCgp100, which gives us scope to accelerate the approval process for IMCgp100, and bringing us a step closer toward making IMCgp100 available for patients with uveal melanoma, for which there are currently no effective treatment options available."

IMCgp100 was granted Orphan Drug Designation by the US Food and Drug Administration (FDA) in January 2016 and participated in the EMA’s Adaptive Pathways Pilot Programme.

On December 11, 2017 argenx (Euronext & Nasdaq: ARGX) a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported that it will provide interim data from its Phase 1/2 clinical trial of ARGX-110 in acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) and an update on the Phase 2 part of its clinical trial with ARGX-110 in cutaneous T-cell lymphoma (CTCL) during a workshop being held in conjunction with the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 9-12, 2017 in Atlanta, Georgia (Press release, argenx, DEC 11, 2017, View Source;p=RssLanding&cat=news&id=2321978 [SID1234522502]).

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The workshop is being held on Monday, December 11, 2017 at 12:00pm EST. A live webcast of the presentation will be available on the Company’s website at www.argenx.com. A replay of the webcast will be available for 90 days following the presentation.

argenx is evaluating the safety, tolerability and efficacy of ARGX-110 in an open-label, Phase 1/2 clinical trial in combination with azacitidine in newly diagnosed AML patients unfit for intensive chemotherapy. During the ASH (Free ASH Whitepaper) workshop today, argenx will announce interim results from the dose-escalation part of the Phase 1/2 clinical trial highlighting promising preliminary data from the first set of six AML patients. All six patients showed encouraging signs of clinical activity, including complete remission (3/6), complete remission with incomplete blood count recovery (1/6) and partial response (2/6). One of the patients that achieved a complete remission bridged to allogeneic stem cell transplant after five cycles. The preliminary data from the first set of patients suggest ARGX-110 is active both at the circulating and bone marrow blast levels and at the leukemic stem cell (LSC) level.

In addition, further data will be presented from the currently ongoing Phase 1/2 clinical trial of ARGX-110 in relapsed/refractory cutaneous T-cell lymphoma (CTCL) patients with confirmed overexpression of CD70 who have failed at least one line of prior therapy. The interim data analyses are from 22 patients, including 13 patients from the Phase 1 part of the trial, which has completed recruitment, and nine patients from the Phase 2 part of the trial. Of the 22 patients under analysis, there was one complete response, two partial responses and 10 with stable disease. ARGX-110 continues to show a favorable tolerability profile in CTCL patients.

Poster presentation at ASH (Free ASH Whitepaper)
argenx collaborators from the University of Bern/Inselspital presented a poster at ASH (Free ASH Whitepaper) highlighting the role of hypomethylating agents (HMA) in inducing upregulation of CD70 on LSCs, but not progenitor cells. There were additional data showing the synergistic effect of HMAs in combination with a variant of ARGX-110. More details can be found here. These data further validate the rationale to evaluate ARGX-110 in combination with azacitidine in the ongoing Phase 1/2 clinical trial.

About ARGX-110
ARGX-110 is a SIMPLE Antibody(TM) targeting CD70, an immune checkpoint target involved in hematological malignancies, several solid tumors and severe autoimmune diseases. ARGX-110 is designed to: i) block CD70, ii) kill cancer cells expressing CD70 through complement dependent cytotoxicity, antibody-dependent cell-mediated phagocytosis and enhanced antibody-dependent cell-mediated cytotoxicity and iii) restore immune surveillance against solid tumors (Silence K. et al. mAbs 2014; 6 (2):523-532). ARGX-110 is currently being evaluated in patients with hematological and solid tumors, including a Phase 1/2 trial in combination with azacitidine in patients with newly diagnosed AML and high-risk MDS and the Phase 2 part of a Phase 1/2 trial in patients with relapsed/refractory CTCL. Preclinical work on ARGX-110 in AML was performed in collaboration with the Tumor Immunology Lab of Prof. A. F. Ochsenbein at the University of Bern, who won, together with Prof. Manz from the University Hospital of Zürich, the prestigious 2016 Otto Naegeli Prize for his breakthrough research on CD70/CD27 signaling with therapeutic potential for cancer patients.

On December 10, 2017 Incyte Corporation (Nasdaq:INCY) reported new 208-week (4-year) follow-up data from the ongoing, global, multi-center, open-label Phase 3 RESPONSE study of Jakafi (ruxolitinib) comparing the efficacy and safety of Jakafi with best available therapy (BAT) in patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU) (Press release, Incyte, DEC 10, 2017, View Source;p=RssLanding&cat=news&id=2321950 [SID1234522490]). The pre-planned data analysis showed a durable primary response to Jakafi in patients with PV who are resistant to or intolerant of HU and the overall safety profile for Jakafi remained consistent with previously reported 80-week RESPONSE data.1 The results were shared in an oral presentation today at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2017 in Atlanta, Georgia.

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"With 30 months of additional follow-up, the four-year RESPONSE data analysis presented today at ASH (Free ASH Whitepaper) further reinforces the potential of Jakafi as a long-term option for patients with PV," said Peg Squier, M.D., Ph.D., Head of U.S. Medical Affairs at Incyte. "Given the few treatment options available to treat this chronic and progressive blood cancer, these long-term safety and efficacy data are meaningful to patients with uncontrolled PV."

The 80-week follow-up results from RESPONSE confirmed that among patients who initially responded to Jakafi treatment, the probability of maintaining primary and hematocrit (Hct) responses for ≥ 80 weeks was 92% and 89%, respectively, and hence Jakafi could be an effective long-term treatment option for patients with PV who are HU-resistant or intolerant.

At the week 208 analysis, the overall long-term safety profile remained consistent with the 80-week data analysis and the response was durable. In both the Jakafi arm and the crossover population, around 30% of patients completed the study treatment and 37% of patients were still receiving treatment.

"These are clinically relevant long-term safety and efficacy results, and further support the use of Jakafi in PV patients who have an inadequate response to or are intolerant of hydroxyurea," said Srdan Verstovsek, M.D., Ph.D., medical oncologist and professor, Department of Leukemia at The University of Texas MD Anderson Cancer Center, Houston, Texas.

About the RESPONSE Trial

RESPONSE is an ongoing, global, multi-center, open-label, Phase 3 trial comparing the efficacy and safety of Jakafi (ruxolitinib) with BAT in 222 patients (Jakafi, 110; BAT, 112) with PV who are resistant to or intolerant of hydroxyurea (HU).2

The primary response was a composite endpoint of the proportion of patients who achieved both hematocrit (Hct) control (defined as no phlebotomy eligibility from week 8 through week 32, with no more than 1 post-randomization phlebotomy eligibility up to week 8) and a spleen volume reduction of at least 35% from baseline at week 32. Phlebotomy eligibility was defined as an Hct >45% and at least 3 percentage points greater than baseline or an Hct >48%. Patients randomized to BAT could crossover (CO) to ruxolitinib at week 32 if they did not meet the primary endpoint, or after week 32 in case of disease progression (PBT eligibility, splenomegaly progression, or both).2

The primary endpoint of the RESPONSE study was achieved, demonstrating that Jakafi was superior to BAT at controlling Hct and reducing spleen volume at week 32.2 The 80-week follow-up results from RESPONSE have been published previously and confirmed that ruxolitinib could be an effective long-term therapy option for HU-resistant/intolerant (R/I) patients with PV.3

Durability of the primary response, overall clinicohematologic (CLHM) response (defined as Hct control, platelet count ≤ 400 × 109/L, white blood cell count ≤ 10 × 109/L, and spleen volume reduction ≥ 35% by imaging), as well as long-term safety were updated at week 208.1

At week 208, the Kaplan-Meier (KM) estimate of duration of primary response was 0.73 (95% CI: 0.49, 0.87), and the KM estimate of duration of absence of PBT eligibility was 0.73 (95% CI: 0.60, 0.83). The KM estimate of duration of at least 35% reduction in spleen volume was 0.86 (95% CI: 0.61, 0.95). Median duration of primary and CLHM responses has not been reached.1

Out of the 70 patients (63.6%) in the Jakafi arm who achieved an overall CLHM response at week 32, 21 had progressed by week 208. The KM estimate of duration of complete hematological remission (defined as Hct control, platelet count ≤ 400 × 109/L, and white blood cell count ≤ 10 × 109/L) at 208-weeks was 0.54 (95% CI: 0.31, 0.72). RESPONSE data also demonstrated that the KM estimate for overall survival at 5-years was 90.6% (95% CI: 80.1, 95.7) for patients treated with Jakafi compared to 87.7% (95% CI: 74.8, 94.3) for patients treated with BAT.1

At the week 208 analysis, 41 patients (37%) originally randomized to the Jakafi arm were still receiving therapy (median exposure, 225 weeks) versus no patients on BAT (median exposure, 34 weeks). Among patients in the Jakafi arm, 29% completed the treatment as per protocol. Of the 98 patients who crossed over to Jakafi after week 32, 38% remained on Jakafi (median exposure, 189 weeks) and 31% completed treatment. Other main reasons for the study drug discontinuations (Jakafi + CO patients) were disease progression (11% + 8%), patient decision (6% + 6%), and adverse events (14% + 14%).1

The most common adverse events in the Jakafi randomized arm (week 208 vs week 80) per 100 patient-years of exposure were anemia (9.3 vs 13.2), pruritus (7.3 vs 9.7), diarrhea (7.1 vs 9.7), headache (6.1 vs 10.5), arthralgia (5.9 vs 6.1), increased weight (5.6 vs 7.5) and muscle spasms (5.4 vs 7.9).

The 208-week results (Abstract #322) were presented as a part of an oral session (#634) on Sunday, December 10, 2017, 7:30-9:00 AM Eastern Time (8:15 AM), Building C, Level 2, C208-C210.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) and is typically characterized by elevated hematocrit, the percent volume of red blood cells in the blood, which can lead to a thickening of the blood and an increased risk of blood clots. An elevated white blood cell and/or platelet count may also be present.4 Patients with PV who fail to consistently maintain appropriate hematocrit levels have a four times higher risk of major thrombosis (blood clots) or cardiovascular death.5 Patients with PV can also suffer from an enlarged spleen and a significant symptom burden which may be attributed to thickening of the blood and lack of oxygen to parts of the body.6 Signs and symptoms of PV commonly include fatigue, itching, night sweats, bone pain, fever, and unexplained weight loss.7

Approximately 100,000 patients in the U.S. are living with PV.8 Current standard treatment for PV is phlebotomy (the removal of blood from the body) plus aspirin. When phlebotomy can no longer control PV, chemotherapy such as hydroxyurea, or interferon, is utilized in high-risk patients.9,10 Approximately one in four patients with PV are considered uncontrolled11,12 because they have an inadequate response to or are intolerant of hydroxyurea, the most commonly used chemotherapeutic agent for the treatment of PV.

About Jakafi (ruxolitinib)

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration for treatment of people with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea.

Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

Important Safety Information

Jakafi can cause serious side effects, including:

Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.

Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.

Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.

Increases in Cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.

The most common side effects of Jakafi include: low platelet count, low red blood cell counts, bruising, dizziness, headache.

These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider. Do not drink grapefruit juice while on Jakafi.

Women should not take Jakafi while pregnant or planning to become pregnant, or if breast-feeding.

Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at www.jakafi.com.

On December 10, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, reported preliminary results from the ongoing Phase 2 trials with luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Atlanta, Georgia (Press release, Acceleron Pharma, DEC 10, 2017, View Source [SID1234522480]). Luspatercept is being developed as part of a global collaboration between Acceleron and Celgene.

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"As the Phase 2 results in MDS mature, we are excited to see luspatercept achieving a clinically meaningful erythroid response in over 50% of patients. Luspatercept continues to provide long-term benefit to multiple patients now nearing three years on treatment. These results further reinforce luspatercept’s potential to be a transformative treatment option for patients living with lower-risk MDS," said Habib Dable, President and Chief Executive Officer of Acceleron. "We look forward to the upcoming MEDALIST and BELIEVE Phase 3 trial top-line data readouts in mid-2018, and we and Celgene continue to make considerable progress toward initiating the COMMANDS Phase 3 trial during the first half of 2018."

Phase 2 Results

A total of 99 lower-risk MDS patients have been treated with therapeutic dose levels of luspatercept (≥ 0.75 mg/kg) in the ongoing Phase 2 trials.

53% (52 of 99 patients) achieved a clinically meaningful erythroid response of an increase in hemoglobin or reduction in red blood cell (RBC) transfusion burden as per the International Working Group’s Hematologic Improvement Erythroid (IWG HI-E) response criteria.
43% (29 of 67 patients) with an RBC transfusion burden at baseline of ≥ 2 units per 8 weeks achieved RBC transfusion independence (RBC-TI) for ≥ 8 weeks.
In an updated analysis of the 23 RBC-TI responders previously reported at EHA (Free EHA Whitepaper) 2017, the median duration of treatment increased to 19.0 months from 14.7 months. The current duration of treatment for RBC-TI responders ranges from 2.8 months to 37.3 months.
Patients with a low transfusion burden at baseline ( < 4 RBC units per 8 weeks and hemoglobin < 10 g/dL) demonstrated a clinically meaningful increase in hemoglobin for up to 34 months, with multiple ongoing.
The results presented at ASH (Free ASH Whitepaper) 2017 confirm and extend previously reported results across the lower-risk MDS patient subpopulations, showing erythroid responses regardless of prior use of erythropoiesis-stimulating agents (ESA), baseline erythropoietin (EPO) levels, and ring sideroblast (RS) status.

Phase 2 Safety Summary

A total of 106 lower-risk MDS patients have been treated with luspatercept in the ongoing Phase 2 trials (all dose levels).

The majority of adverse events (AEs) were Grade 1 or 2. AEs possibly or probably related to study drug that occurred in at least three patients during the studies were headache, hypertension, fatigue, bone pain, diarrhea, arthralgia, injection site erythema, myalgia, and edema peripheral.
Grade 3 non-serious AEs possibly related to study drug were ascites, blast cell count increase, blood bilirubin increase, bone pain, hypertension, mucosal inflammation, platelet count increase, and pleural effusion. These Grade 3 non-serious AEs occurred in seven individual patients with one patient reporting both the ascites and pleural effusion.
Serious AEs (SAEs) possibly related to study drug were general physical health deterioration, muscular weakness, musculoskeletal pain, and myalgia. These SAEs occurred in three individual patients with one patient reporting both the muscular weakness and musculoskeletal pain.
The MEDALIST trial, a global Phase 3 trial of luspatercept in lower-risk MDS patients, is fully enrolled with top-line results expected in mid-2018. The MEDALIST trial enrolled patients who are RS-positive, RBC transfusion dependent, and are ESA-refractory or ESA-treatment ineligible, based on EPO levels greater than 200 units per liter at baseline. Acceleron and Celgene plan to initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS patients during the first half of 2018.

The MDS clinical poster presentation is available under the Science page of the Company’s website at www.acceleronpharma.com.

Luspatercept is an investigational product that is not approved for use in any country.

Acceleron ASH (Free ASH Whitepaper) Conference Call Information

Acceleron will host a conference call and live webcast to discuss data presented at the ASH (Free ASH Whitepaper) meeting on December 11, 2017, at 7:00 a.m. EST.

Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron ASH (Free ASH Whitepaper) 2017 Update."

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com.

A replay of the webcast will be available approximately two hours after the event.

About the Ongoing MDS Phase 2 Studies

Data from two Phase 2 trials were presented at the conference: the base study in which patients received treatment with luspatercept for three months and the long-term extension study in which patients who completed the base study may receive treatment with luspatercept for up to an additional five years. In both the three-month base study and the long-term extension study, lower-risk MDS patients were enrolled and treated with open-label luspatercept, dosed subcutaneously once every three weeks.

The outcome measures for the trials included the proportion of patients who had an erythroid response (IWG HI-E) or achieved RBC transfusion independence (RBC-TI). IWG HI-E was defined as hemoglobin increase ≥ 1.5 g/dL sustained for ≥ 8 weeks in patients with < 4 units RBC / 8 weeks transfusion burden at baseline and hemoglobin levels below 10 g/dL. For patients with a ≥ 4 units RBC / 8 weeks transfusion burden at baseline, erythroid response was defined as a reduction of ≥ 4 units RBC sustained for ≥ 8 weeks. RBC-TI was defined as receiving no RBC transfusions for ≥ 8 weeks in patients with a ≥ 2 units RBC / 8 weeks baseline transfusion burden.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the TGF-beta superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept is a first-in-class erythroid maturation agent (EMA) that regulates late-stage erythrocyte (red blood cell) precursor cell differentiation. This mechanism of action is distinct from that of erythropoiesis stimulating agents (ESAs), which stimulate 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 trial) and in patients with beta-thalassemia (the BELIEVE trial). A Phase 3 trial is being planned in first-line, lower-risk, myelodysplastic syndromes patients (the COMMANDS trial). For more information, please visit www.clinicaltrials.gov.