Onconova Announces Four Presentations from Rigosertib Clinical Trials in Myelodysplastic Syndromes (MDS) at the 2018 ASH Annual Meeting & Exposition

On November 5, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported that four abstracts relating to the Company’s lead product candidate, rigosertib, were accepted for presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in San Diego, California, which takes place December 1-4, 2018 (Press release, Onconova, NOV 5, 2018, View Source [SID1234530731]).

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ORAL PRESENTATION:

Phase 2 Expansion Study of Oral Rigosertib Combined with Azacitidine (AZA) in Patients (Pts) with Higher-Risk (HR) Myelodysplastic Syndromes (MDS): Efficacy and Safety Results in HMA Treatment Naïve & Relapsed (Rel)/Refractory (Ref) Patients

Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: Novel Therapeutics I

Date: Saturday, December 1, 2018

Session Time: 4:00 – 5:30 PM

Presentation Time: ORAL SESSION 4:15 PM PST

Room: Manchester Grand Hyatt San Diego, Grand Hall A

Presenter: Shyamala C. Navada, MD, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

POSTER PRESENTATIONS:

1) Truncation Products of Stromal Cell Derived Factor-1 (CXCL12) Quantified By Mass Spectrometry in Patients with Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) Treated with Rigosertib in a Phase I-II Study

Session Name: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Poster I

Date: Saturday, December 1, 2018

Presentation Time: 6:15 – 8:15 PM

Location: San Diego Convention Center, Hall GH

Presenter: John Roboz, PhD, Icahn School of Medicine at Mount Sinai, New York, NY

2) Evaluation of Underlying Cause of Genitourinary (GU) Adverse Events (AEs) in Patients with Myelodysplastic Syndromes upon Oral Administration of Rigosertib: Safety and Pharmacokinetic Analysis of Rigosertib across Three Clinical Trials

Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: Poster II

Date: Sunday, December 2, 2018

Presentation Time: 6:00 – 8:00 PM

Location: San Diego Convention Center, Hall GH

Presenter: Manoj Maniar, PhD, Onconova Therapeutics, Inc., Newtown, PA

3) Amelioration of Rigosertib Treatment Related Genitourinary (GU) Adverse Events (AEs) in Patients with Myelodysplastic Syndromes: Implementation of Novel Dosing Regimen Derived through Pharmacokinetic Modeling in Phase 2 Study of Oral Rigosertib in Combination with Azacitidine

Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: Poster III

Date: Monday, December 3, 2018

Presentation Time: 6:00 – 8:00 PM

Location: San Diego Convention Center, Hall GH

Presenter: David Taft, PhD, Arnold & Marie Schwartz College of Pharmacy and Health Sciences, Long Island University, Brooklyn, NY

BioCryst to Present Abstracts at Annual Scientific Meeting of American College of Allergy, Asthma & Immunology

On November 5, 2018 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present three abstracts at the upcoming Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) November 15-19 in Seattle (Press release, BioCryst Pharmaceuticals, NOV 5, 2018, View Source [SID1234530749]).

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Oral Administration of a Liquid Formulation of BCX7353 Rapidly Provides Sustained Concentrations and Kallikrein Inhibition; Poster P153, Friday, November 16, 3:30 p.m. PT

Relationship of Target Concentrations with Efficacy: Results from the APeX-1 Study of BCX7353; Poster P157, Friday, November 16, 4:10 p.m. PT

Pharmacokinetics and Pharmacodynamics of BCX7353, an Oral Plasma Kallikrein Inhibitor, in Healthy Japanese Subjects, Poster P158, Friday, November 16, 4:20 p.m. PT
"The Annual Scientific Meeting of the ACAAI provides a focused opportunity for us to meet with many of the clinical and academic leaders driving continued innovation to improve HAE therapy, and we are excited to, once again, have an important scientific presence at the meeting," said Dr. William Sheridan, chief medical officer of BioCryst.

SpringWorks Therapeutics Receives FDA Orphan Drug Designation for MEK Inhibitor
(PD-0325901) for the Treatment of Neurofibromatosis Type 1

On November 5, 2018 SpringWorks Therapeutics, a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for PD-0325901, an investigational, oral, small molecule selective inhibitor of MEK1 and MEK2, for the treatment neurofibromatosis type 1 (Press release, SpringWorks Therapeutics, NOV 5, 2018, View Source [SID1234538852]).

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Neurofibromatosis type 1, or NF1, is a rare genetic disorder that is caused by mutations in the NF1 gene, and that affects both children and adults. NF1 mutations can result in a loss of activity in the protein neurofibromin, which can lead to MAPK pathway overactivation, uncontrolled cellular growth, and tumor formation.1 NF1 is a heterogeneous condition that frequently causes plexiform neurofibromas, which are painful, disfiguring tumors of the peripheral nervous system. It is estimated that there are approximately 100,000 patients in the United States living with NF1. 2

"There is an urgent need for an effective treatment for patients with NF1. We are pleased that the FDA has granted Orphan Drug designation for our MEK inhibitor, recognizing its potential to improve the outlook for patients and families facing NF1," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We look forward to working closely with the FDA as we prepare to initiate a Phase 2b study of PD-0325901 in patients with NF1-associated plexiform neurofibromas, a severe form of NF1 that causes tumors to grow on nerves throughout the body, which can cause severe pain, disfigurement, loss of range of motion, and shortened lifespan."

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan drug designation to drugs and biologic products that are intended for treatment of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug status is intended to facilitate drug development for rare diseases and may provide certain benefits and incentives to drug developers, including seven years of market exclusivity if the drug is approved, FDA assistance in clinical trial design, and tax credits for qualified clinical trial costs. 3

SpringWorks expects to initiate a Phase 2b single-arm, open-label study of PD-0325901 in patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-associated PN) in the first half of 2019.

About Neurofibromatosis Type 1
Neurofibromatosis type 1, or NF1, is a rare genetic disorder that is caused by mutations in the NF1 gene, and that affects both children and adults. Throughout their lifetime, about 30 to 50 percent of NF1 patients progress to a more severe form of the disease that results in the development of plexiform neurofibromas (PN), which are progressive peripheral nerve sheath tumors that cause severe pain, morbidity, and can significantly shorten lifespan.4-6 The clinical course of NF1-associated PN is heterogeneous with varying manifestations and severity across patients.

It is estimated that NF1 affects 1 in 3,000 individuals worldwide, and that there are approximately 100,000 patients in the United States living with this disease.2 Most patients with NF1-associated PN are treated with surgical removal of the tumors, sometimes requiring amputation; however, surgery has variable success rates and a high rate of recurrence has been observed because of the aggressive nature of these tumors.7 There are no therapies currently approved for the treatment of NF1-associated PN.

About PD-0325901
PD-0325901 is an investigational, oral, small molecule, selective inhibitor of MEK1 and MEK2, proteins that play key roles in the MAPK pathway. The MAPK pathway is critical for cell survival and proliferation, and overactivation of this pathway has been shown to help enable tumor growth. By blocking activity of the MAPK pathway, PD-0325901 may help arrest uncontrolled cellular growth associated with many types of tumors.

PD-0325901 has been tested in several Phase 1 and Phase 2 clinical trials, and approximately 260 subjects have been exposed to treatment. SpringWorks is evaluating PD-0325901 as a monotherapy for the treatment of patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-associated PN). In addition, given the critical role that the MAPK pathway plays in the growth and proliferation of a large number of tumor types, SpringWorks is also pursuing PD-0325901 in combination with other rational anticancer agents across a range of solid tumors.

Dynavax Reports Third Quarter 2018 Financial Results, Progress on HEPLISAV-B Launch, and Updated SD-101 Data in Three Patient Populations

On November 5, 2018 Dynavax Technologies Corporation (NASDAQ: DVAX) reported financial results for the third quarter ended September 30, 2018 along with an update on the launch progress of HEPLISAV-B and an overview of recently presented data for SD-101 in combination with KEYTRUDA (Press release, Dynavax Technologies, NOV 5, 2018, View Source [SID1234530715]).

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Recent Highlights

HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]

P&T approval in six of the top ten integrated delivery networks
402 of the largest targeted customers have received P&T committee approval, of whom 200 have progressed to purchase and 68 have implemented HEPLISAV-B throughout their system
Another 291 target customers have sub-committee or P&T committee reviews scheduled
Q3 sales of $1.5 million compared to $1.2 million in Q2
Immuno-oncology

Encouraging results for SD-101 Phase 1b/2 data in combination with KEYTRUDA presented at the ESMO (Free ESMO Whitepaper) 2018 Congress:

In 47 advanced melanoma patients naïve to anti PD-1 therapy who received 2mg dose:
— Overall response rate (ORR) of 70%, identical ORR to previous report at ASCO (Free ASCO Whitepaper) with a greater than 50 percent increase in number of patients
— 85% 6-month progression-free survival (PFS) rate
— Observed responses in injected lesion(s) and non-injected distant lesions, including visceral metastases in the liver and lung
— Responses were independent of baseline PD-L1 expression
— Adverse events related to SD-101 treatment were transient, mild to moderate flu-like symptoms
21.4 percent ORR in 29 melanoma patients refractory or resistant to anti-PD-1 therapy who received the 8 mg dose
27.3% ORR in 22 patients with head and neck squamous cell carcinoma who were naïve to anti-PD-1 and received the 8 mg dose
End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) completed
"We achieved multiple objectives over the past few months in both the HEPLISAV-B commercial program and the SD-101 clinical program. Our HEPLISAV-B results in September and particularly October, together with our projections for November and December give us confidence we are now experiencing the start of the inflection in HEPLISAV-B sales we consistently have anticipated for year end. We remain firm in our expectation that HEPLISAV-B will become cash generative before the end of 2019," said Eddie Gray, chief executive officer of Dynavax. "In addition, our studies of SD-101 in combination with pembrolizumab continue to generate consistent, encouraging results beyond those reported with monotherapy. We are evaluating multiple opportunities, including partnerships, expansion of tumor types under study and selection of best options for progression into registrational studies."

Financial Results
Cash, cash equivalents and marketable securities totaled $180.2 million at September 30, 2018, compared to $216 million at June 30, 2018, with $75 million available from our term loan agreement.

Dynavax’s first commercial product, HEPLISAV-B, was launched in the first quarter of 2018 and net product revenue for the three and nine months ended September 30, 2018 were $1.5 million and $2.9 million, respectively. Product revenue from sales is recorded at the net sales price which includes estimates of product returns, chargebacks, discounts and other fees.

Cost of sales, product was $3.9 million and $9.3 million for the three and nine months ended September 30, 2018 and consists of inventory reserves and fill, finish and overhead costs incurred after FDA approval for the vial presentation of HEPLISAV-B. Also included are costs associated with resuming operations at our manufacturing facility in Dusseldorf after receiving regulatory approval for the pre-filled syringe presentation, which costs previously were included in research and development expense.

Research and development expenses for the three months ended September 30, 2018 and 2017, were $16.8 million and $16.4 million, respectively. Research and development expenses for the nine months ended September 30, 2018 and 2017, were $52.1 million and $47.6 million, respectively. The increase in 2018 reflects increased compensation and related personnel costs related to the ongoing development of SD-101, DV281 and earlier stage oncology programs. Upon approval of pre-filled syringes in Q1, 2018, costs associated with resuming activities at our manufacturing facility in Dusseldorf were charged to cost of sales-product while costs incurred to manufacture HEPLISAV-B for commercial sale were accounted for as inventory.

Selling, general and administrative expenses for the three months ended September 30, 2018 and 2017, were $15.8 million and $6.0 million, respectively. Selling, general and administrative expenses for the nine months ended September 30, 2018 and 2017 were $48.3 million and $18.1 million, respectively. The increase is primarily due to an overall increase in HEPLISAV-B sales, marketing and commercial activities, including full-deployment of a contract sales force, post-marketing studies and consultants for commercial development services.

The net loss for the third quarter of 2018 was $40.5 million, or $0.65 per share, compared to $22.1 million, or $0.38 per share, for the third quarter of 2017. The net loss for the nine-month period ended September 30, 2018 was $118.9 million, or $1.91 per share, compared to $67.7 million, or $1.36 per share, for the same period in 2017.

Conference Call and Webcast Information
Dynavax will hold a conference call today at 4:30pm ET/1:30pm PT. To access the call, participants must dial (866) 420-4066 in the U.S. or (409) 217-8237 internationally, and use the conference ID 5179228. The live call will be webcast and can be accessed in the "Investors and Media" section of the company’s website at www.dynavax.com. A replay of the webcast will be available for 30 days following the live event.

About Hepatitis B
Hepatitis B is a viral disease of the liver that can become chronic and lead to cirrhosis, liver cancer and death. The hepatitis B virus is 50 to 100 times more infectious than HIV,i and transmission is on the rise. In 2015, new cases of acute hepatitis B increased by more than 20 percent nationally.ii There is no cure for hepatitis B, but effective vaccination can prevent the disease.

In adults, hepatitis B is spread through contact with infected blood and through unprotected sex with an infected person. The CDC recommends vaccination for those at high risk for infection due to their jobs, lifestyle, living situations and travel to certain areas.iii Because people with diabetes are particularly vulnerable to infection, the CDC recommends vaccination for adults age 19 to 59 with diabetes as soon as possible after their diagnosis, and for people age 60 and older with diabetes at their physician’s discretion.iv Approximately 20 million U.S. adults have diabetes, and 1.5 million new cases of diabetes are diagnosed each year.v

About HEPLISAV-B
HEPLISAV-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax’s proprietary Toll-like Receptor (TLR) 9 agonist to enhance the immune response. Dynavax has worldwide commercial rights to HEPLISAV-B.

For more information about HEPLISAV-B, visit View Source

About SD-101
SD-101, the Company’s lead clinical candidate, is a proprietary, second-generation, Toll-like receptor 9 (TLR9) agonist CpG-C class oligodeoxynucleotide. Dynavax is evaluating this intratumoral TLR9 agonist in several clinical studies to assess its safety and activity, including a Phase 1b/2 study in combination with KEYTRUDA (pembrolizumab), an anti-PD-1 therapy, in patients with advanced melanoma and in patients with head and neck squamous cell cancer, in a clinical collaboration with Merck. Dynavax maintains all commercial rights to SD-101.

About Dynavax
Dynavax is a fully-integrated biopharmaceutical company focused on leveraging the power of the body’s innate and adaptive immune responses through toll-like receptor (TLR) stimulation. Dynavax discovers and develops novel vaccines and immuno-oncology therapeutics. The Company’s first commercial product, HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted], was approved by the United States Food and Drug Administration in November 2017 for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. Dynavax’s lead immunotherapy product, SD-101, is an investigational cancer immunotherapeutic currently being evaluated in Phase 1/2 studies and its second cancer immunotherapeutic, DV281, is in Phase 1 development. For more information, visit www.dynavax.com.

Unum Therapeutics to Host Third Quarter 2018 Financial Results Conference Call and Webcast on November 12, 2018 at 4:30 P.M. ET

On November 5, 2018 Unum Therapeutics Inc. (Nasdaq: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T-cell Receptor (ACTR) technology platform, reported that the company will host a conference call and live audio webcast on Monday, November 12, 2018 at 4:30 p.m. ET to discuss financial results for the third quarter of 2018 (Press release, Unum Therapeutics, NOV 5, 2018, View Sourcenews-releases/news-release-details/unum-therapeutics-host-third-quarter-2018-financial-results" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-host-third-quarter-2018-financial-results" rel="nofollow">View Source [SID1234530733]). Unum management will also provide an update on the Company’s recent progress and upcoming milestones.

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Participants may access the conference call by dialing 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 2287146. To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at View Source at least 10 minutes before the event begins.

A webcast replay will be available at the same location on the Unum Therapeutics website beginning approximately two hours after the event, and will be archived for 90 days.