On March 18, 2019 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the full year ended December 31, 2018 and highlighted progress in 2018 and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 18, 2019, View Source [SID1234534501]).

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Key 2018 Accomplishments

Completed initial public offering and raised approximately $86 million in gross proceeds.
Entered into a strategic fibrosis-focused collaboration with Gilead Sciences, Inc. to discover and develop highly specific inhibitors of TGFβ activation ($80 million received upfront and eligible for up to an additional $1,450 million in potential milestone payments).
Transitioned to a clinical-stage company with the initiation of a Phase 1 clinical trial of SRK-015 in healthy volunteers.
Published preclinical data on the therapeutic benefit of inhibiting myostatin activation in mouse models of Spinal Muscular Atrophy (SMA) in peer-reviewed journal Human Molecular Genetics.
Received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product Designation from the European Commission (EC) for SRK-015 for the treatment of SMA.
Presented compelling preclinical data that demonstrate SRK-181-mIgG1 (murine version of SRK-181), a highly specific TGFβ1 inhibitor, can render resistant solid tumors vulnerable to PD1 blockade.
Progressed antibody platform for neuromuscular disorders, cancer immunotherapy, fibrosis, and anemias.
"2018 was a year of remarkable progress with our platform and for establishing a strong foundation as we transitioned to a public company," said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. "We are focused on advancing our growing portfolio of product candidates, including SRK-015 for the treatment of SMA and SRK-181 to overcome primary resistance to checkpoint blockade therapy, as we continue to pursue our mission of developing innovative therapies to address significant unmet medical needs of patients."

R&D Highlights and Upcoming Milestones

SRK-015 Program:

Presented Interim Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers. In February 2019, Scholar Rock presented positive interim results from the Phase 1 clinical trial of SRK-015, a highly specific inhibitor of myostatin activation, which was observed to be well tolerated with no dose limiting toxicities identified up to the highest evaluated dose of 30 mg/kg. Pharmacodynamic data, measuring serum concentrations of latent myostatin, demonstrated robust and sustained target engagement, providing initial proof-of-mechanism for this unique therapeutic approach. Collectively, the favorable interim safety and tolerability, pharmacodynamic, and pharmacokinetic data supported the advancement of SRK-015 to a Phase 2 proof-of-concept clinical trial in patients with SMA.
Initiating Phase 2 Proof-of-Concept Trial for SRK-015 in SMA in First Quarter 2019. Scholar Rock is initiating a Phase 2 proof-of-concept trial to assess the safety and efficacy of SRK-015. The trial will consist of three non-overlapping cohorts, each evaluating a distinct subpopulation of patients with Type 2 and Type 3 SMA and all patients will receive SRK-015 dosed once every four weeks either as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy. The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA, over a 12-month treatment period. Scholar Rock plans to provide additional details on the Phase 2 trial design at the time of initiating patient dosing in the second quarter of 2019. Interim safety and efficacy results for a subset of patients in each cohort with at least six months of treatment exposure are expected in the first half of 2020.
Plan to Identify Second Indication for SRK-015 in 2020. Scholar Rock continues to see multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 Program:

Nominated SRK-181 as Cancer Immunotherapy Product Candidate. In March 2019, Scholar Rock selected SRK-181, a highly specific inhibitor of TGFβ1 activation, as the first product candidate in its TGFβ1 cancer immunotherapy program based on the strength of preclinical data and human translational insights. SRK-181 is being developed for the treatment of tumors resistant to checkpoint blockade therapies (CBTs), such as anti-PD(L)1 antibodies. Scholar Rock plans to initiate a Phase 1 trial in patients with solid tumors in mid-2020.
Additional SRK-181-mIgG1 Preclinical Data to be Presented at Upcoming AACR (Free AACR Whitepaper) Annual Meeting. Scholar Rock will present additional preclinical data from multiple syngeneic mouse models of primary checkpoint resistance at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held March 29-April 3, 2019. These studies demonstrate that co-administration of SRK-181-mIgG1 (murine version of SRK-181) with an anti-PD1 antibody permits effector T cell infiltration and expansion into the tumor microenvironment, resulting in tumor regression or control as well as significant survival benefit, while minimizing toxicities traditionally associated with pan-TGFβ inhibitors.
Full Year 2018 Financial Results

For the year ended December 31, 2018, net loss was $49.3 million or $3.15 per share compared to a net loss of $25.0 million or $15.30 per share for the year ended December 31, 2017.

Research and development expense was $36.3 million for the year ended December 31, 2018 compared to $19.9 million for the year ended December 31, 2017. The increase year-over-year in both periods reflect development and manufacturing costs associated with lead product candidate, SRK-015, research costs associated with preclinical studies, as well as increased personnel-related costs to support continued progress with the pipeline.
General and administrative expense was $14.4 million for the year ended December 31, 2018 compared to $5.1 million for the year ended December 31, 2017. The increase year-over-year was primarily attributable to increased headcount and higher professional and consulting fees associated with the IPO and collaboration with Gilead, as well as ongoing business activities and operations as a public company.
As of December 31, 2018, Scholar Rock had cash, cash equivalents, and marketable securities of $175.6 million, compared to $58.0 million as of December 31, 2017.

"We established a strong financial foundation in 2018 with contributions from our successful IPO and our strategic fibrosis collaboration with Gilead," said Rhonda Chicko, Chief Financial Officer of Scholar Rock. "As illustrated by the recent announcement of positive interim Phase 1 results for SRK-015 and the nomination of SRK-181 as the first product candidate in our cancer immunotherapy program, we continue to focus on our robust pipeline of highly specific growth factor modulators across a diverse range of therapeutic areas."

On March 18, 2019 Alpine Immune Sciences, Inc. (Nasdaq:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer, autoimmune/inflammatory, and other diseases, reported financial results for the year ended December 31, 2018 (Press release, Alpine Immune Sciences, MAR 18, 2019, View Source [SID1234534423]).

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"This was a year of continued execution for Alpine as we transitioned from a preclinical company to a development stage company, dosing earlier this quarter the first subjects in our Phase I healthy volunteer study of ALPN-101, our lead therapeutic for the potential treatment of autoimmune and inflammatory diseases. In addition, we continue to advance our lead oncology program, ALPN-202, preparing it for the clinic with the goal of filing an IND or IND-equivalent late this year," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "With our recently completed private placement of $25.3 million led by Decheng Capital, we are well positioned to achieve key catalysts later this year in both of our programs."

Recent Corporate and Clinical Highlights

First Subjects Dosed in Phase I Healthy Volunteer Trial for Lead Autoimmune/Inflammatory Disease Program, ALPN-101: On February 11, 2019, we announced the successful initial dosing of our first-in-human Phase I study of ALPN-101, a first-in-class dual ICOS/CD28 antagonist. The study is designed to evaluate the safety and tolerability of single- and multiple-ascending intravenous and/or subcutaneous doses of ALPN-101 in healthy volunteers. In addition, pharmacokinetics, pharmacodynamics, and exploratory biomarkers are being evaluated to help determine ALPN-101’s potential for the treatment of autoimmune and inflammatory diseases.

Completion of $25.3 Million Private Placement: On January 15, 2019, we entered into a securities purchase agreement for the sale of units consisting of shares of common stock and warrants to purchase common stock in a private placement providing gross proceeds to us of approximately $25.3 million. The private placement was led by Decheng Capital with participation from existing investors OrbiMed Advisors, Frazier Healthcare Partners, Alpine BioVentures, and BVF Partners, L.P.

Strengthened Board of Directors and Scientific Advisory Board with Recent Key Appointments:

Upon closing of our $25.3 million private placement in January 2019, Min Cui, Ph.D., Founder and Managing Director of Decheng Capital, was appointed to our Board of Directors. Dr. Cui’s focus at Decheng is on partnerships with entrepreneurs and building early stage biotechnology companies, and he currently holds Board positions at several companies. He has co-founded two companies, Pacific Pharmaceuticals and Hucon Biopharmaceuticals, where he led the research and development of key inflammatory and oncology therapies.
In addition, we appointed Vijay Kuchroo, DVM, Ph.D., Rafi Ahmed, Ph.D., James Welsh, M.D., Anne Davidson, M.B.B.S., and John Thompson, M.D. to our Scientific Advisory Board. They join a team of distinguished translational and clinical scientists in inflammatory and autoimmune diseases and cancers, including Scientific Advisory Board Chair Andrew Scharenberg, M.D., Manish Butte, M.D., Ph.D., and Paul Tumeh, M.D.
Key Preclinical Data Presentations at Medical Meetings: We showcased preclinical data for our lead programs, ALPN-101 and ALPN-202, at the following recent medical meetings:

ALPN-202 preclinical data presented at SITC (Free SITC Whitepaper): In November 2018, we presented preclinical data of our lead oncology program, ALPN-202, a PD-L1/CTLA-4 dual antagonist with PD-L1 dependent CD28 costimulation, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting in Washington, D.C. Data presented demonstrated the superiority of ALPN-202 over PD-L1 inhibition in a preclinical tumor model.
ALPN-101 GvHD preclinical data highlighted in oral presentation and posters at ASH (Free ASH Whitepaper) 2018: In December 2018, we presented positive results from multiple preclinical studies of our lead autoimmune/inflammatory program, ALPN-101, via poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s (ASH) (Free ASH Whitepaper) 60th Annual Meeting & Exposition in San Diego, CA. In addition, in an oral presentation, researchers from the lab of Indiana University School of Medicine’s Sophie Paczesny, M.D., Ph.D., one of our collaborators, found ALPN-101 significantly improved survival in preclinical models of acute graft versus host disease (GvHD) and highlighted the novel role of ICOS ligand in GvHD.
ALPN-101 GvHD preclinical data presented at 2019 TCT Meetings: In February 2019, we presented preclinical data for ALPN-101 GvHD at the 2019 Transplantation & Cellular Therapy Meetings of ASBMT and CIBMTR (TCT Meetings) in Houston, TX. Data presented demonstrated potent and dose-dependent suppression of GvHD in a human/NSG xenograft model, with activity superior to CD28 or ICOS single pathway antagonists.
Full Year 2018 Financial Results

As of December 31, 2018, we had cash, cash equivalents, and short-term investments totaling $52.3 million. Net cash used in operating activities for the year ended December 31, 2018 was $28.4 million compared to $16.6 million for the year ended December 31, 2017. We recorded a net loss of $36.5 million and $7.8 million for the years ended December 31, 2018 and 2017, respectively.

Collaboration revenue for the year ended December 31, 2018 was $705,000 compared to $1.7 million for the year ended December 31, 2017. The increase was primarily attributable to the timing of revenue recognized under our collaboration agreement with Kite Pharma, Inc., a Gilead (Nasdaq: GILD) company. As previously announced, under the terms of this research collaboration and license agreement, we received upfront payments of $5.5 million, which were initially recorded as deferred revenue and expensed over the period of the research term. The research term of the agreement with Kite was extended in October 2018.

Research and development expenses for the year ended December 31, 2018 were $29.0 million compared to $10.6 million for the year ended December 31, 2017. The increase was primarily attributable to an increase in direct research, contract manufacturing, and process development activities to support ALPN-101 and ALPN-202, plus increases in research personnel related to expanding research and discovery programs and associated overhead and facility costs.

General and administrative expenses for the year ended December 31, 2018 were $8.4 million compared to $6.1 million for the year ended December 31, 2017. The increase was primarily attributable to professional and legal fees and operating as a public company, in addition to personnel-related expenses and the costs associated with expanding the company’s operations as we accelerate preclinical activity.

Cash Guidance

We expect to have sufficient cash to fund operations into 2021, including the clinical advancement of our lead autoimmune/inflammatory program, ALPN-101, and our lead oncology program, ALPN-202.

About ALPN-101

ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), and a first-in-class therapeutic simultaneously inhibiting the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely related costimulatory molecules with partially overlapping roles in T cell activation likely connected to multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, and multiple sclerosis, ALPN-101 demonstrates efficacy superior to blockade of the CD28 or ICOS pathways alone.

ALPN-101 was engineered using our vIgD platform, which uses directed evolution to transform native IgSF proteins into multifunctional protein therapeutics. ALPN-101 is currently enrolling a Phase I healthy volunteer trial.

On March 18, 2019 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the full year ended December 31, 2018 (Press release, Mustang Bio, MAR 18, 2019, View Source [SID1234534424]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "2018 was a transformational year for Mustang that positioned the company for further exciting advances in 2019. We enhanced our pipeline of therapies in August by adding a clinical-stage lentiviral gene therapy product candidate with curative potential for X-linked severe combined immunodeficiency ("XSCID"), for which we’re expecting compelling data to be published in a major medical journal this year. In June, we opened our cell processing facility in Worcester, Mass., which is now fully operational. We anticipate processing patients’ cells in the coming months under Mustang’s first IND—a significant milestone for the company. In December, the U.S. Food and Drug Administration ("FDA") granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of blastic plasmacytoid dendritic cell neoplasm ("BPDCN"), a rare and incurable blood cancer. Most recently, in February we licensed an oncolytic virus (C134) that we plan to combine with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme. With these achievements, Mustang has built a strong foundation for success in the coming year."

Financial Results:

·As of December 31, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $34.6 million, compared to $41.3 million as of September 30, 2018, and $61.5 million as of December 31, 2017, a decrease of $6.7 million for the fourth quarter and a decrease of $26.9 million year-to-date.
·Research and development expenses were $21.1 million for the year ended December 31, 2018. This compares to $7.9 million for 2017. Non-cash, stock-based compensation expenses included in research and development were $3.4 million for the year ended December 31, 2018, compared to $0.7 million for 2017.
·Research and development expenses from license acquisitions totaled $3.4 million for the year ended December 31, 2018, compared to $12.4 million for 2017. Non-cash, stock-based compensation expenses included in research and development – licenses acquired were $2.1 million for the year ended December 31, 2018, compared to $9.6 million for 2017.
·General and administrative expenses were $6.8 million for the year ended December 31, 2018. This compares to $11.4 million for 2017. Non-cash, stock-based compensation expenses included in general and administrative expenses were $1.5 million for the year ended December 31, 2018, compared to $2.6 million for 2017.
·Net loss attributable to common stockholders was $30.7 million, or $1.14 per share, for the year ended December 31, 2018, compared to a net loss attributable to common stockholders of $31.3 million, or $1.24 per share, for 2017.

2018 and Recent Corporate Highlights:

·In May 2018, Mustang announced the publication of preclinical data in JCI Insight demonstrating that glioblastoma-targeted CD4+ CAR T cells mediate superior antitumor activity over CD8+ CAR T cells. The data, published by research partner City of Hope, will be applied in the ongoing Phase 1 trial of Mustang’s IL13Rα2-specific CAR T MB-101 in glioblastoma.

·In June 2018, Mustang opened a proprietary CAR T cell therapy manufacturing facility at UMass Medicine Science Park in Worcester, Mass. The facility will support the clinical development and commercialization of Mustang’s CAR T and gene therapy product candidates and enable proprietary cell therapy research.
·Also in June 2018, Mustang was added to the Russell 2000, 3000 and Microcap Indexes.
·In July 2018, Mustang completed a pre-Investigational New Drug ("pre-IND") meeting with the FDA for MB-102 (CD123 CAR T). Based on the meeting, Mustang expects to initiate a multicenter Phase 1/2 trial of MB-102 in acute myeloid leukemia ("AML"), BPDCN and high-risk myelodysplastic syndrome in the second half of 2019.
·In August 2018, Mustang announced that it entered into an exclusive worldwide license agreement with St. Jude Children’s Research Hospital for the development of a potentially first-in-class ex vivo lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease. The therapy is currently being evaluated in a Phase 1/2 multicenter trial in infants under the age of two. This study is the world’s first lentiviral gene therapy trial for infants with XSCID. The therapy is also being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health ("NIH"). The company believes these may be registration trials.
·In October 2018, Mustang announced that City of Hope initiated a first-of-its-kind Phase 1 clinical trial evaluating the safety and effectiveness of intraventricular delivery of CAR T cells to the brains of patients with HER2-positive breast cancer with brain metastases; the first patient was dosed in December 2018. In addition, Mustang announced that City of Hope dosed the first patient in a Phase 1 clinical trial of HER2-specific CAR T cells in treating recurrent or refractory grade III-IV glioma. The trial is evaluating the side effects and best dose of HER2-specific CAR T cells in treating patients with grade III-IV glioma that has come back or does not respond to treatment.
·In November 2018, Mustang announced that additional safety and efficacy Phase 1 data evaluating MB-102 (CD123 CAR) in relapsed or refractory AML and BPDCN were presented in an oral session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Special Conference on Tumor Immunology and Immunotherapy.
·In December 2018, the FDA granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of BPDCN.
·In February 2019, Mustang announced that it partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop an oncolytic virus (C134) for the treatment of glioblastoma multiforme. Mustang intends to combine the oncolytic virus with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme.

On March 18, 2019 Salarius Pharmaceuticals, LLC, a clinical-stage oncology company targeting the epigenetic causes of cancers, reported that its Chief Executive Officer David Arthur will present at the 29th Annual Oppenheimer Healthcare Conference held March 19-20, 2019, at the Westin New York Grand Central Hotel (Press release, Salarius Pharmaceuticals, MAR 18, 2019, View Source [SID1234534475]).

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Mr. Arthur will present an overview of Salarius’ business, including its development strategy for its lead compound, Seclidemstat, which targets the epigenetic dysregulation underlying Ewing sarcoma, a devastating pediatric, adolescent and young adult bone cancer for which no targeted therapies currently exist. Salarius is currently enrolling patients in an open-label Phase 1 dose escalation/dose expansion study, which is expected to conclude in 2020.

Details of Salarius’ presentation are as follows:

Event:

29th Annual Oppenheimer Healthcare Conference

Date:

Wednesday, March 20, 2019

Time:

11:30 a.m. (Eastern Time)

Location:

Westin New York Grand Central; Track 2

An audio webcast will be accessible via the News and Events section of the Salarius Pharmaceuticals website: View Source An archive of the audio will remain available for 90 days following the presentation.

On March 18, 2019 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical-stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center, reported that Walter Klemp, Chairman and CEO and Jonathan Foster, Chief Financial Officer, will present at the following investor conferences (Press release, Moleculin, MAR 18, 2019, View Source [SID1234534425]):

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Oppenheimer 29th Annual Healthcare Conference – Management will present on Wednesday, March 20, 2019 at 3:20 p.m. ET. The Conference will be held at the Westin New York Grand Central Hotel in New York City.
31st Annual ROTH Conference – Management will be available on Monday, March 18, 2019 for one-on-one meetings with investors. The Conference will be held at the Ritz-Carlton Hotel in Dana Point, CA.