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Investor Presentation dated March 2020

On March 13, 2020 Moleculin Biotech Presented the Corporate Presentation (Presentation, Moleculin, MAR 13, 2020, View Source [SID1234555607]).

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ENETS 2020 : Safety Profile and AESI for Surufatinib in Chinese Patients with Advanced Extra-Pancreatic Neuroendocrine Tumors: Analysis of the Phase 3 SANET-ep Trial

On March 13, 2020 Hutchison China MediTech reported SANET-ep trial (clinicaltrials.gov identifier NCT02588170) demonstrated that surufatinib significantly improves progression-free survival ("PFS") in patients with advanced extrapancreatic (non-pancreatic) neuroendocrine tumors compared to placebo, with a median PFS of 9.2 months versus 3.8 months, respectively (hazard ratio = 0.334, 95% CI 0.223 to 0.499, p<0.0001) (Press release, Hutchison China MediTech, MAR 13, 2020, https://www.chi-med.com/enets-2020/ [SID1234555570]). This presented analysis evaluated the safety profile and adverse events of special interest ("AESI") of surufatinib from SANET-ep data. 93.8% of patients in the surufatinib group and 73.5% of patients in the placebo group had at least one treatment-emergent AESI.

The analysis concluded that AESIs of Grade 3 or higher hypertension and proteinuria occurred more frequently with surufatinib than with placebo, with the most common (at least 3% of patients) grade 3 or greater AESIs being hypertension (40.3% with surufatinib vs. 16.2% with placebo), proteinuria (23.3% vs. 0) and hemorrhage (3.1% vs. 2.9%). It also concluded that AESIs leading to treatment discontinuation were uncommon, with AESIs leading to dose discontinuation in at least 1% of patients being proteinuria (3.9% with surufatinib vs. 0 with placebo), hemorrhage (1.6% vs 1.5%), and hepatic failure (0.8% vs 1.5%). Surufatinib has a manageable safety profile in patients with advanced extra-pancreatic neuroendocrine tumors.

Title: Safety Profile and Adverse Events of Special Interest for Surufatinib in Chinese Patients with Advanced Extra-Pancreatic Neuroendocrine Tumors: Analysis of the Phase 3 SANET-ep Trial

Authors: Jie Li, Jianming Xu, Zhiwei Zhou, Chunmei Bai, Yihebali Chi, Zhiping Li, Nong Xu, Enxiao Li, Tianshu Liu, Yuxian Bai, Sha Guan, Lin Shen

Abstract: #2914

Introduction The previously reported SANET-ep trial (NCT02588170) demonstrated surufatinib significantly improves progression-free survival (PFS) in patients (pts) with advanced extrapancreatic neuroendocrine tumors (epNETs) compared to placebo; median PFS (9.2 vs. 3.8 months; HR = 0.334, 95% CI 0.223 to 0.499, p<0.0001).

Aims The present analysis evaluates the safety profile and adverse events of special interest (AESIs) of surufatinib from SANET-ep data.

Patients and Methods Pts were randomized (2:1) to receive surufatinib (300 mg once daily continuously) or placebo. Treatment-related AESIs and time-to-first occurrence of AESIs were summarized. Predefined AESIs included hepatic failure (HF), proteinuria (P), hypertension (HTN), haemorrhage (H), and acute renal failure (ARF), which were searched with narrow MedDRA Standardized MedDRA Query (SMQ).

Results A total of 121/129 (93.8%) pts in the surufatinib group and 50/68 (73.5%) in the placebo group had ≥ 1 treatment-emergent AESI; the mean relative dose intensity was 86.42% and 96.78%, respectively. The most commonly reported (>10% of pts) AESIs were P (84.5% vs 57.4%), HTN (68.2% vs 30.9%), and H (55.8% vs 27.9%). The most common (≥3% of pts) grade ≥3 AESIs were HTN (40.3% vs 16.2%), P (23.3% vs 0) and H (3.1% vs 2.9%); the median time-to-onset of these events in the surufatinib group was 13.5, 28, and 32 days, respectively. AESIs (≥1% of pts) leading to dose discontinuation were P (3.9% vs 0), H (1.6% vs 1.5%), and HF (0.8% vs 1.5%).

Conclusions The AESIs of Grade ≥ 3 HTN and P occurred more frequently with surufatinib; however, AESIs leading to treatment discontinuation were uncommon. Surufatinib has a manageable safety profile in pts with advanced epNETs.

Keywords extra-pancreatic, neuroendocrine tumors, safety

FDA Clearance Brings RefleXion Closer to Expanding Cancer Treatment Market

On March 13, 2020 RefleXion Medical, a therapeutic oncology company pioneering the use of biology-guided radiotherapy (BgRT)* to treat all stages of cancer reported it has received marketing clearance from the U.S. Food & Drug Administration (FDA) for stereotactic body radiotherapy (SBRT), stereotactic radiosurgery (SRS) and intensity modulated radiotherapy (IMRT) for its RefleXion X1 machine (Press release, RefleXion Medical, MAR 13, 2020, View Source [SID1234555559]).

"We are at the forefront of an enormous change in expanding the use of radiotherapy from a treatment solely for early-stage cancer patients to an entirely new group of patients who need it most, those with advanced stage cancer," said Todd Powell, CEO and president of RefleXion. "This initial marketing clearance of our RefleXion X1 machine is a steppingstone on the path to our goal of providing BgRT as a novel treatment modality that will expand the overall radiotherapy market significantly.

"We have been preparing for commercial operations in the brand new factory we opened last year and expect to begin machine shipments within the next quarter," continued Powell.

The RefleXion X1 is the only machine that combines high quality computed tomography (CT) imaging, known as fan-beam CT, with a linear accelerator to reduce motion artifacts that can inhibit accurate targeting of the radiation dose to a patient’s tumor. Its groundbreaking design rotates up to 60 times faster than other linear accelerators and modulates dose delivery from 100 points per beam station. These combined improvements may reduce the side effects of radiotherapy by allowing radiation oncologists to better localize the tumor, reduce patient setup errors and precisely deliver dose to complex tumor targets while avoiding nearby normal structures.

"Transitioning this company from a research effort to what is, as of today, a commercial entity has been a thrilling 10-year journey," said Sam Mazin, Ph.D., founder and CTO of RefleXion. "Along the way, RefleXion has become an organization supported by dedicated and brilliant people in every corner, including our clinical partners. We will celebrate this milestone and then quickly turn our sights to bringing our biology-guided radiotherapy to market."

BgRT, a novel treatment modality under development, uses the biological signature of a tumor to characterize its movement and to deliver a precisely tracked therapeutic radiation dose to the tumor. The RefleXion X1 machine with BgRT is designed to overcome the technical limitations that currently restrict radiotherapy to one or two tumors. Once developed, RefleXion will scale BgRT to treat all visible tumors, even those that move rapidly due to involuntary processes such as breathing or digestion, in the same treatment session.

Eiger BioPharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update

On March 13, 2020 Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, reported financial results for the fourth quarter and full year 2019 and provided a business update (Press release, Eiger Biopharmaceuticals, MAR 13, 2020, View Source [SID1234555557]).

"We are pleased to announce the submission of the MAA for lonafarnib in Progeria and Progeroid Laminopathies and look forward to completing the NDA submission by end of the month as planned," said David Cory, President and CEO of Eiger. "Site activations, enrollment, and dosing are ongoing in our global Phase 3 HDV study, D-LIVR, and we expect to complete enrollment in 2020. We are closely monitoring the potential impact of COVID-19 on the timing and conduct of D-LIVR. We will remain responsive to any developments and take necessary steps to protect our patients and the integrity of the study."

Recent Highlights and Upcoming Milestones

Lonafarnib in Hepatitis Delta Virus (HDV)

Enrollment of Phase 3 D-LIVR study (N=400) planned to complete in 2020
Lonafarnib in Progeria and Progeroid Laminopathies

Marketing Authorization Application (MAA) to EMA completed
Accelerated Assessment for MAA granted by EMA
New Drug Application (NDA) to FDA on-track for completion by end of March 2020
Peginterferon Lambda (lambda) in HDV

Positive End of Phase 2 meeting with FDA with agreement on single Phase 3 study
Plan to finalize Scientific Advice with EMA
End-of-Treatment data from LIFT (lambda combo with lonafarnib) at EASL 2020
Corporate

Appointed Eldon Mayer as Executive Vice President and Chief Commercial Officer
Fourth Quarter and Full Year 2019 Financial Results

Cash, cash equivalents, and short-term investments as of December 31, 2019 totaled $95.0 million compared to $100.4 million at December 31, 2018, a decrease of $5.4 million.

The Company reported net losses of $16.9 million, or $0.69 per share, and $70.3 million, or $3.08 per share, for the fourth quarter and full year 2019, respectively, as compared to $16.5 million, or $0.93 per share, and $52.4 million, or $3.84 per share, for the same periods in 2018.

Research and Development expenses were $11.9 million and $51.8 million for the fourth quarter and full year 2019, respectively, as compared to $12.0 million and $37.1 million for the same periods in 2018. The increase in full year 2019 expenses was primarily due to costs associated with the Phase 3 D-LIVR HDV study, including drug supply costs, which ramped-up in 2019, employee-related costs from increased headcount and an increase in regulatory related expenses.

General and Administrative expenses were $4.6 million and $17.1 million for the fourth quarter and full year 2019, respectively, as compared to $4.1 million and $14.0 million for the same periods in 2018. The increases in fourth quarter and full year 2018 were primarily due to increases in employee-related costs, including stock-based compensation, from increased headcount and outside services for legal, consulting, advisory and accounting services.

Total operating expenses include total non-cash expenses of $1.6 million and $6.6 million for the fourth quarter and full year 2019, respectively, as compared to $1.6 million and $5.7 million for the same periods in 2018.

As of December 31, 2019, the company had 24.5 million of common shares outstanding

Five Prime Therapeutics Announces Publication of the Phase 1 Bemarituzumab Study in the Journal of Clinical Oncology

On March 13, 2020 Five Prime Therapeutics, Inc. (NASDAQ: FPRX), a clinical-stage biotechnology company focused on developing immune modulators and precision therapies for solid tumor cancers, reported the publication of results from the phase 1 escalation and expansion study of bemarituzumab in patients with advanced solid tumors and FGFR2b-selected gastroesophageal adenocarcinoma in the digital edition of the Journal of Clinical Oncology (Press release, Five Prime Therapeutics, MAR 13, 2020, View Source [SID1234555555]).

The purpose of the phase 1 trial was to evaluate the safety, pharmacokinetics, and preliminary activity of single-agent bemarituzumab in patients with FGFR2b-overexpressing GEA. Seventy-nine patients were enrolled in the trial and no dose-limiting toxicities were reported. Bemarituzumab was well tolerated and the most frequent treatment-related adverse events (TRAEs) were fatigue, nausea, and dry eye. The overall response rate observed in this study of advanced-stage patients with high FGFR2b-overexpressing GEA was 17.9% (95% CI 6.1% to 36.9%) with five of 28 patients achieving a confirmed partial response.

"Gastroesophageal adenocarcinoma is the third most common cause of cancer death worldwide and the median overall survival of patients who present with advanced disease remains dismal at only 11 months," said Helen Collins, M.D., Executive Vice President and Chief Medical Officer of Five Prime Therapeutics. "The results of this study underscore the potential of bemarituzumab evaluation as a novel treatment option for patients with advanced gastric and gastroesophageal junction cancer."

"Monotherapy activity of bemarituzumab and its lack of significant overlapping toxicities with standard chemotherapeutic agents suggest that combining bemarituzumab with chemotherapy may potentially benefit patients in the front-line setting whose GEA tumors overexpress FGFR2b," said Daniel Catenacci, M.D. and Associate Professor, the University of Chicago Medical Center and Biological Sciences.

Bemarituzumab is being evaluated in combination with mFOLFOX6 in the Phase 3 FIGHT (FGFR2b Inhibition in Gastric and Gastroesophageal Junction Cancer Treatment) trial in the front-line treatment setting.