With the Federal Reserve hinting that it could lower rates before the end of the year, the stock market has caught a bid. Biotech stocks, while extremely risky gambits in the current market environment, could rally and so we are continuing our weekly Sunday biotech stocks to watch report.
Here are 7 biotechnology stocks to watch for the coming week. Each of these biotech stocks have fast approaching catalysts that could cause these stocks to explode higher or crash into a heap of worthless junk.
EDSIVO for Vascular Ehlers-Danlos Syndrome. PDUFA date under priority review June 25, 2019.
Acer continues progress toward goal of commercializing EDSIVO™
NEWTON, Mass., Dec. 26, 2018 (GLOBE NEWSWIRE) — Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and ultra-rare diseases with critical unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. Priority review is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.
“The acceptance of our NDA for EDSIVO™ is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for EDSIVO™. We are excited about the possibility of making EDSIVO™ available in the U.S. for patients in the near future.”
“We continue to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vEDS if it is approved by the FDA,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious rare diseases over the next several years.”
About EDSIVO™ and vEDS
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The average mortality is 51 years of age. An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO™ (celiprolol), a new chemical entity (NCE), for the treatment of vEDS based on a randomized controlled clinical study of celiprolol(1). FDA granted a priority review of the EDSIVO™ NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. EDSIVO™ received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.
About Acer Therapeutics
Acer, headquartered in Newton, MA, is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for patients with serious rare and ultra-rare diseases with critical unmet medical need. Acer’s late-stage clinical pipeline includes two candidates for severe genetic disorders: EDSIVO™ (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS), and ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD and limited options for UCD, which collectively impact approximately 7,000 patients in the U.S. Acer’s product candidates have clinical proof-of-concept and mechanistic differentiation, and Acer intends to seek approval for them in the U.S. by using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission, and approval of a marketing application.
Vyleesi (Bremelanotide) for female sexual dysfunction (FSD). PDUFA date extended to June 23, 2019.
WALTHAM, Mass., January 7, 2019 – AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) today announced preliminary unaudited fourth quarter and full year 2018 financial results and provided 2019 financial guidance.
The company will provide a portfolio update at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 9, 2019 at 7:30 a.m. PT (10:30 a.m. ET). A live audio webcast of the presentation and following breakout session will be accessible through the Investors section of AMAG’s website at www.amagpharma.com.
“In 2018, we achieved several key milestones including receiving two approvals from the U.S. Food and Drug Administration (FDA), launching both the Makena subcutaneous auto-injector and Feraheme broad label, and receiving FDA acceptance of our new drug application for VyleesiTM (bremelanotide). In addition, we bolstered our pipeline by adding two promising late-stage development assets, AMAG-423 and ciraparantag, targeting orphan patient populations,” said William Heiden, AMAG’s president and chief executive officer.
“As we enter 2019, AMAG is in a unique position with a number of growing commercial products that together generate significant cash flows, allowing us to invest in the development and launch of our new pharmaceutical products. We have built an innovative and diversified portfolio that I believe positions us well for long-term growth and will deliver significant shareholder value,” added Mr. Heiden.
ARQ 531 (EHA 2019) for B-cell malignancies. Phase 1 presentation at EHA June 15, 2019.
BURLINGTON, Mass.–(BUSINESS WIRE)–Jun. 7, 2019– ArQule, Inc. (Nasdaq: ARQL), today announced that it will present clinical data from the company-sponsored phase 1 dose escalation study on its BTK inhibitor, ARQ 531, for the treatment of relapsed or refractory B-cell lymphoid malignancies in a poster presentation at the 24thCongress of European Hematology Association (EHA), held from June 13-16, 2019 in Amsterdam.
Title:A Phase 1 Dose Escalation Study Of ARQ 531 In Patients with Relapsed or Refractory B-Cell Lymphoid Malignancies
Abstract #: PS1150
Session: 6. Chronic lymphocytic leukemia and related disorders – Clinical
Date:Saturday, June 15, 2019
Time:5:30-7:00 p.m. CEST
Location: RAI Amsterdam; Poster area
ArQule will host a conference call and webcast for investors on Friday, June 14, 2019 at 8:00 a.m. EDT to discuss the ARQ 531 clinical data. The live webcast can be accessed in the “Investors and Media” section of our website, www.arqule.com, under “Events & Presentations” or by visiting http://public.viavid.com/index.php?id=134824. You may also listen to the call by dialing 1-800-239-9838 within the U.S. or 1-323-794-2551 outside the U.S. and providing conference ID 3110780. A replay will be available two hours after the completion of the call and can be accessed in the “Investors & Media” section of our website, www.arqule.com, under “Events and Presentations.”
ArQule Management will also be hosting an Investor event to answer questions and discuss these data on Friday, June 14th from 5:30 – 8:00 p.m. CEST. Investors, sell side analysts and industry representatives are welcome to attend. To register to attend, please click on the link here.
About BTK and ARQ 531
Bruton’s tyrosine kinase, BTK, is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in blood cancers. ARQ 531 is an orally bioavailable, potent and reversible dual inhibitor of both wild type and C481S-mutant BTK. The C481S-mutation is a known resistance mechanism for first generation irreversible BTK inhibitors. ARQ 531 has demonstrated a good safety profile, predictable PK, profound pharmacodynamic effects and emerging signs of dose-proportional clinical activity in phase 1 clinical testing.
ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases. ArQule’s mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients. Our clinical-stage pipeline consists of four drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader among companies our size in precision medicine. ArQule’s pipeline includes: ARQ 531, an orally bioavailable, potent and reversible dual inhibitor of both wild type and C481S-mutant BTK, in phase 1 for patients with B-cell malignancies refractory to other therapeutic options; miransertib (ARQ 092), a potent and selective inhibitor of the AKT serine/threonine kinase, planned to initiate registrational trial cohorts in Proteus syndrome and PROS in 2019, and in phase 1b in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer; ARQ 751, a next generation highly potent and selective AKT inhibitor, in phase 1 for patients with AKT1 and PI3K mutations; and derazantinib, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, in a registrational trial for iCCA in collaboration with Basilea and Sinovant. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds.
Avatrombopag for Immune Thrombocytopenic Purpura (ITP). PDUFA date for sNDA filing June 30, 2019.
DURHAM, N.C., Nov. 05, 2018 (GLOBE NEWSWIRE) — Dova Pharmaceuticals, Inc. (NASDAQ: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, today announced the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for DOPTELET (avatrombopag) for a new indication, the treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment. ITP is an autoimmune bleeding disorder characterized by thrombocytopenia, i.e., an abnormally low level of platelets. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision on the sNDA is June 30, 2019.
DOPTELET, a second generation, orally administered thrombopoietin receptor agonist (TPO-RA), was approved by FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.
“Chronic ITP affects approximately 60,000 adults in the United States and despite the currently available therapies, which include two other TPO-RAs, there remains an important unmet need,” said Lee F. Allen, MD, PhD and Chief Medical Officer of Dova. “Acceptance of this sNDA is another significant milestone for Dova, and an important step towards addressing this underserved patient population and expanding the applications for DOPTELET as a treatment for thrombocytopenia. We look forward to working closely with the FDA as they review this sNDA.”
The ITP sNDA is supported by safety and efficacy data from one pivotal randomized, placebo-controlled Phase 3 clinical trial in the target indication that met its primary (number of weeks with a platelet count ≥50×109/L in the absence of rescue therapy) and first secondary (proportion of subjects with platelet counts ≥50×109/L on Day 8) efficacy endpoints with high statistical significance (P<0.0001). Data from the Phase 3 clinical trial has been recently published online (Br J Haematol. 2018 Sep 7. doi: 10.1111/bjh.15573. [Epub ahead of print]), and will be included in an upcoming volume of the British Journal of Haematology. Additional supportive efficacy data for the ITP sNDA are provided by two Phase 2 ITP clinical trials, as well as the two Phase 3 trials for the treatment of thrombocytopenia in patients with CLD. Data from all 24 studies in the avatrombopag clinical development program support the safety and tolerability of avatrombopag across multiple indications.
Tazemetostat (ICML 2019) for Follicular lymphoma. Phase 2 updated data due at ICML on June 21, 2019.
CAMBRIDGE, Mass.–(BUSINESS WIRE)–May 16, 2019– Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, today announced that new data from the epithelioid sarcoma and follicular lymphoma cohorts of the company’s ongoing Phase 2 clinical trials of tazemetostat will be reported during oral presentations at medical meetings in June.
Updated data from the fully enrolled cohort of epithelioid sarcoma patients in the company’s molecularly defined solid tumor program will be presented at the American Society of Clinical Oncology (ASCO) 2019 Annual Meeting in Chicago. Updated data from the fully enrolled cohorts of patients with follicular lymphoma, both with and without EZH2 activating mutations, will be reported at the International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland. The company will also report findings from its follicular lymphoma natural history study during a poster session at the 24thCongress of the European Hematology Association (EHA) in Amsterdam.
“The updated data to be presented at these upcoming meetings showcase tazemetostat’s potential to positively impact patients in need of new treatment options and, importantly, support the two planned NDA submissions this year,” said Robert Bazemore, president and chief executive officer of Epizyme. “We remain on track to submit our epithelioid sarcoma NDA to the U.S. FDA in the second quarter, followed by our planned NDA submission for follicular lymphoma patients, both with and without EZH2 mutations, in the fourth quarter of this year. We look forward to these data presentations and continuing our work to execute these pivotal milestones.”
Details of the presentations are listed below:
ASCO Oral Presentation
Title: Safety and efficacy of tazemetostat, a first-in-class EZH2 inhibitor, in patients (pts) with epithelioid sarcoma (ES) (NCT02601950)
Presenter: Silvia Stacchiotti, M.D., Fondazione IRCCS Istituto Nazionale Tumori, Milan
Abstract No.: 11003
Date: Monday, June 3, 2019; 9:00 – 9:12 a.m. CDT
EHA Poster Discussion
Title: EZH2 gain-of-function mutations are not associated with more favorable prognosis in relapsed/refractory follicular lymphoma: a preliminary analysis on 590 patients
Session: Indolent and mantle-cell non-Hodgkin lymphoma – Clinical
Presenter: Dr. Jessica Okuson, Barts Cancer Institute, Queen Mary University of London
Abstract No.: PS1247
Date: Saturday, June 15, 2019; 5:30 – 7:00 p.m. CEST
Location: Poster Area
ICML Oral Presentation
Title: Interim update from a Phase 2 multicenter study of tazemetostat, an EZH2 inhibitor, in patients with relapsed or refractory follicular lymphoma
Presenter: Franck Morschhauser, M.D., Ph.D., Centre Hospitalier Régional Universitaire de Lille, France
Abstract No.: 105
Date: Friday, June 21, 2019; 2:45 – 3:00 p.m. CEST
Location: Room A
About Epizyme, Inc.
Epizyme, Inc. is a late-stage biopharmaceutical company committed to rewriting treatment for cancer and other serious diseases through novel epigenetic medicines. Epizyme is broadly developing its lead product candidate, tazemetostat, a first-in-class EZH2 inhibitor, with studies underway in both solid tumors and hematological malignancies, as a monotherapy and combination therapy in relapsed and front-line disease. The company also is developing a novel G9a program with its next development candidate, EZM8266, which is targeting sickle cell disease. By focusing on the genetic drivers of disease, Epizyme’s science seeks to match targeted medicines with the patients who need them. For more information, visit www.epizyme.com.
GBT440 (EHA 2019) for Sickle cell disease. Phase 3 data to be presented at EHA on June 14, 2019. NDA to be filed under accelerated approval.
Phase 3 HOPE Study 24-Week Efficacy Results from Full Patient Cohort to be Presented During Presidential Symposium
New Drug Application (NDA) Submission Planned for Voxelotor to Include HOPE Study Data Being Presented at EHA 2019
Company to Host Investor Webcast on Friday, June 14
SOUTH SAN FRANCISCO, Calif., May 16, 2019 (GLOBE NEWSWIRE) — Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that data supporting the company’s voxelotor program in sickle cell disease (SCD) will be presented during the 24thEuropean Hematology Association (EHA) Congress, which is taking place June 13-16 in Amsterdam.
Three abstracts have been accepted for presentation, including new 24-week efficacy and safety data from approximately 270 patients in the Phase 3 HOPE Study, which will be presented during the Presidential Symposium on Friday, June 14.
“We are pleased that the new long-term efficacy and safety data from our HOPE Study was selected for presentation during the Presidential Symposium, which showcases the top six abstracts selected by the EHA Scientific Program Committee each year. The HOPE Study results we will present at EHA represent the data we will include in our New Drug Application for voxelotor, which we are preparing to submit in the second half of this year,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Together with our two poster presentations, the HOPE Study data supports the potential of voxelotor to safely sustain improvements in hemoglobin levels, reduce hemolysis and be a disease-modifying treatment that could reduce the morbidity and mortality of patients with SCD.”
The HOPE Study data that will be presented at the Congress includes additional efficacy and safety data not available at the time the abstracts were submitted and, as such, remains under embargo until Friday, June 14.
The EHA abstracts are now available at www.ehaweb.org. Details of GBT’s presentations are as follows:
Friday, June 14
Oral Session: Presidential Symposium
Abstract #S147: Results from the Randomized Placebo-Controlled Phase 3 Hope Trial of Voxelotor in Adults and Adolescents with Sickle Cell Disease
Presenter: Jo Howard, MB BChir, MRCP, FRCPath, Guy’s and St. Thomas’ NHS Foundation Trust and King’s College, London, UK
Time: 4:15-4:30 p.m. CEST / 10:15-10:30 a.m. ET
Location: Hall 5
Poster Session: Sickle Cell Disease
Abstract #PF740: Cerebral Blood Flow in Adolescents with Sickle Cell Anemia Receiving Voxelotor
Presenter: Jeremie H. Estepp, M.D., Assistant Member, Faculty, Hematology Department, St. Jude Children’s Research Hospital, Memphis, Tenn.
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area
Saturday, June 15
Poster Session: Sickle Cell Disease
Abstract #PS1522: Central Physiologic Mechanisms Which Augment Oxygen Release (Bohr Effect and 2,3-DPG Binding) Are Preserved in the Presence of Voxelotor at the Therapeutic Target of 30% Hb Modification
Presenter: Mira Pochron, Ph.D., Scientist II, GBT, South San Francisco, Calif.
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area
Investor Webcast Details
GBT will host an investor event webcast on Friday, June 14, 2019, at 7:00 p.m. CEST / 1:00 p.m. ET to review the HOPE Study data being presented at EHA 2019. The event will be webcast live and available for replay from GBT’s website at www.gbt.com in the Investors section.
About Sickle Cell Disease
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). In its deoxygenated state, HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which causes hemolytic anemia (low hemoglobin due to RBC destruction) that can lead to multi-organ damage and early death. This sickling process also causes blockage in capillaries and small blood vessels. Beginning in childhood, SCD patients typically suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities.
About Voxelotor in Sickle Cell Disease
Voxelotor (previously called GBT440) is being developed as an oral, once-daily therapy for patients with SCD. Voxelotor works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes voxelotor blocks polymerization and the resultant sickling and destruction of red blood cells. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that voxelotor may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted voxelotor Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency(EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.
GBT is evaluating voxelotor in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical study in patients age 12 and older with SCD. Additionally, voxelotor is being studied in the ongoing Phase 2a HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in pediatric patients (age 4 to 17) with SCD. The HOPE-KIDS 1 Study is assessing the safety, tolerability, pharmacokinetics and exploratory treatment effect of voxelotor.
GBT is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. GBT is developing its lead product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
Molgradex – IMPALA for autoimmune pulmonary alveolar proteinosis (PAP). Phase 3 data due sometime in June 2019.
Savara Announces Molgradex Received Fast Track Designation by FDA for Treatment of Autoimmune Pulmonary Alveolar Proteinosis
AUSTIN, Texas–(BUSINESS WIRE)–May 6, 2019– Savara Inc. (NASDAQ: SVRA), an orphan lung disease company, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). Molgradex, the Company’s lead product candidate, is being investigated in a pivotal Phase 3 study, called IMPALA, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). Topline results from the study are expected in June 2019. Positive results would facilitate the submission of a Biologics License Application (BLA) in the first half of 2020, with an anticipated commercial launch later in 2020 or early 2021.
The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. A drug granted Fast Track designation may be eligible for Priority Review or Rolling Review of the BLA, if relevant criteria are met.
“We are excited by this designation as it reinforces that a better treatment option is needed for people living with aPAP,” said Rob Neville, Chief Executive Officer, Savara. “With the potential for Priority or Rolling Review, we are optimistic about the opportunity to accelerate the submission of Molgradex on behalf of patients with this devastating disease.”
Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP), in Phase 2a development for nontuberculous mycobacterial (NTM) lung infection in both non-cystic fibrosis (CF) and CF-affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3-stage inhaled vancomycin for treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2-ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization. More information can be found at www.savarapharma.com. (Twitter: @SavaraPharma, LinkedIn: www.linkedin.com/company/savara-pharmaceuticals/)
Disclosure: I do not hold any position in any stock mentioned in this article.