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Moleculin Files with FDA for Expedited Approval Pathway for Annamycin

On March 13, 2019 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported that it has submitted a request for Fast Track Designation with the US Food and Drug Administration (FDA) for its drug, Annamycin, for the treatment of relapsed or refractory acute myeloid leukemia (AML) (Press release, Moleculin, MAR 13, 2019, View Source [SID1234534272]).

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"Now that we have some traction in our clinical trials of Annamycin for the treatment of relapsed or refractory AML," commented Walter Klemp, Moleculin’s Chairman and CEO, "we believe it is appropriate to request Fast Track designation for Annamycin. Importantly, this is a valuable first step in ultimately qualifying for Accelerated Approval and Priority Review."

A drug that receives Fast Track designation is eligible for some or all of the following:

More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

LEXICON PHARMACEUTICALS REPORTS FOURTH QUARTER AND FULL-YEAR 2018 FINANCIAL RESULTS AND PROVIDES A BUSINESS UPDATE

On March 13, 2019 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months and full-year ended December 31, 2018 and provided a business update (Press release, Lexicon Pharmaceuticals, MAR 13, 2019, View Source [SID1234534256]).

"In 2018, we achieved continued growth in XERMELO net sales and executed well against our strategic priorities," said Lonnel Coats, Lexicon’s president and chief executive officer. "We made significant progress on our pipeline, which included Sanofi’s submission of marketing applications for sotagliflozin in type 1 diabetes in the U.S. and Europe as well as advancement of our earlier-stage product candidates, LX2761 in diabetes and LX9211 in neuropathic pain. In 2019, our focus remains on creating long-term value for the company by executing on our strategic and financial objectives."

Fourth Quarter and Full-Year 2018 Product and Pipeline Highlights

XERMELO (telotristat ethyl) 250 mg

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XERMELO U.S. net sales reached $25.0 million in 2018.
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XERMELO received national reimbursement approval in Scotland, Denmark, Sweden, Greece, Luxemburg, Northern Ireland, Wales, Germany, Belgium and the Netherlands for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy.
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Positive analyses on time to sustained improvement in bowel movement frequency with XERMELO were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Gastrointestinal Cancers Symposium (ASCO GI) and the European Neuroendocrine Tumor Society Conference (ENETS).
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Favorable changes in weight in patients on XERMELO with neuroendocrine tumors (NETs) and carcinoid syndrome who participated in the TELESTAR study along with biochemical and metabolic improvements in diarrhea severity and nutritional status were published in Clinical Therapeutics.

Sotagliflozin
Type 1 Diabetes

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Additional positive 52-week data from the pivotal inTandem1 and inTandem2 studies for sotagliflozin in type 1 diabetes were presented at the 78th annual American Diabetes Association Scientific Sessions (ADA) and the European Association for the Study of Diabetes (EASD) 54th annual meeting and published in Diabetes Care.
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Lexicon’s collaborator, Sanofi, submitted a New Drug Application (NDA) and a Marketing Authorization Application (MAA) for sotagliflozin in type 1 diabetes and the regulatory filings were accepted by the Food and Drug Administration (FDA) and European Medicines Agency (EMA), respectively.

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On January 17, 2019, the FDA Endocrinologic and Metabolic Drugs Advisory Committee voted eight to eight on the question of whether the overall benefits of sotagliflozin outweighed the risks to support approval in type 1 diabetes.
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On February 28, 2019, the EMA Committee for Medicinal Products for Human Use adopted a positive opinion recommending regulatory approval of sotagliflozin for use as an adjunct to insulin therapy to improve glycemic control in adults with type 1 diabetes with a body mass index of 27 kg/m2 or greater, who have failed to achieve adequate glycemic control despite optimal insulin therapy.
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A target FDA action date under the Prescription Drug User Fee Act (PDUFA) is set for March 22, 2019 and a regulatory decision by the European Commission is expected in Q2 2019.

Type 2 Diabetes

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Patient enrollment continued for eleven Phase 3 sotagliflozin clinical trials in type 2 diabetes being conducted by Sanofi.
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Patient enrollment was completed in the nine Phase 3 clinical trials that support the planned filings for regulatory approval of sotagliflozin in type 2 diabetes.
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Sanofi initiated two additional Phase 3 studies for sotagliflozin in Chinese patients with type 2 diabetes (NCT03760965, NCT03761134).

LX2761

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Lexicon announced topline results from Phase 1 clinical studies of LX2761, an orally-administered, selective sodium-glucose cotransporter type 1 (SGLT1) inhibitor, in healthy subjects and patients with type 2 diabetes that confirmed the drug’s unique preclinical profile as a potent gastrointestinal tract-selective SGLT1 inhibitor.

LX9211

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Lexicon announced positive topline results from a Phase 1a clinical study of LX9211, an orally-administered, selective adapter-associated kinase 1 (AAK1) inhibitor that is being developed for neuropathic pain. The Phase 1a study met its primary objectives, identifying a maximum tolerated dose and demonstrating a safety and tolerability profile in healthy human subjects supporting progression of the clinical program.

Fourth Quarter and Full-Year 2018 Financial Highlights

Unless otherwise stated, all comparisons are for the fourth quarter and full year of 2018 compared to the fourth quarter and full year of 2017.

Revenues: Revenues for the fourth quarter decreased to $17.1 million from $34.0 million for the corresponding period in 2017, primarily due to lower revenues recognized under collaboration and license agreements. Full-year 2018 revenues decreased to $63.2 million from $91.7 million, primarily due to timing of revenues recognized from clinical trial activities under the collaboration and license agreements with Sanofi and decreases in milestone payments from Ipsen, partially offset by an increase in net product revenue. Net product revenues for full-year 2018 included $25.0 million and $1.6 million, respectively, from net sales of XERMELO in the U.S. and the sale of bulk tablets to Lexicon’s collaborator, Ipsen.

Cost of Sales: Cost of sales related to sales of XERMELO was $0.6 million and $0.5 million, respectively, for the fourth quarter of 2018 and 2017. Full-year 2018 and 2017 cost of sales was $2.5 million and $1.9 million, respectively.

Research and Development (R&D) Expenses: Research and development expenses for the fourth quarter decreased to $12.3 million from $46.3 million for the corresponding period in 2017, primarily due to decreases in our external clinical development costs relating to sotagliflozin. Full-year 2018 R&D expenses decreased to $100.2 million from $152.2 million, primarily due to lower external clinical development costs relating to sotagliflozin and professional and consulting fees.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the fourth quarter were $16.6 million compared to $16.1 million for the same period in 2017. Full-year 2018 SG&A expenses decreased to $63.8 million from $66.1 million, primarily due to lower salaries and benefits, and decreased marketing costs.

Income Tax Benefit: During 2018, there was no income tax benefit. During 2017, Lexicon recognized an $8.7 million income tax benefit when the intangible assets relating to XERMELO were reclassified from indefinite-lived to finite-lived assets. The income tax benefit was remeasured to $12.7 million for full year 2017.

Net Loss: Net loss for the fourth quarter was $16.8 million, or $0.16 per share, compared to a net loss of $26.6 million, or $0.25 per share, in the corresponding period in 2017. For the fourth quarter 2018, net loss included non-cash, stock-based compensation expense of $2.8 million. For the fourth quarter 2017, net loss included non-cash, stock-based compensation expense of $2.3 million. Net loss for the full-year 2018 was $120.5 million, or $1.14 per share, compared to a net loss of $123.0 million, or $1.17 per share, in 2017. For the full-year 2018, net loss included non-cash, stock-based compensation expense of $11.7 million. For the full-year 2017, net loss included non-cash, stock-based compensation expense of $9.5 million.

Cash and Investments: As of December 31, 2018, Lexicon had $160.1 million in cash and investments, as compared to $310.8 million as of December 31, 2017.

Anticipated Upcoming Milestones

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Q1 2019 – Manuscript publications for XERMELO in carcinoid syndrome diarrhea
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Q1 2019 – Initiation of a Phase 1b study for LX9211
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March 22, 2019 – PDUFA date for sotagliflozin in type 1 diabetes in the U.S.
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Q2 2019 – European Commission decision on marketing application for sotagliflozin in type 1 diabetes in the EU
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June, September 2019 – Presentation of new analyses from pivotal studies of sotagliflozin in type 1 diabetes at the annual ADA and EASD meetings
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2H 2019 – Topline Phase 1b data for LX9211
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2019 – Topline data from core Phase 3 studies for sotagliflozin in type 2 diabetes
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2019 – Patient enrollment in a Phase 2 study for telotristat ethyl in biliary tract cancer

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am EDT / 7:00 am CDT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 6598765. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/investors. An archived version of the webcast will be available on the website for 14 days.

About XERMELO (telotristat ethyl)

Discovered using Lexicon’s unique approach to gene science, XERMELO (telotristat ethyl) is the first and only approved oral therapy for carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSAs. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells. Lexicon has built the in-house capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights. Lexicon also retains rights to market XERMELO in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize XERMELO in Europe and other countries outside of U.S. and Japan.

XERMELO was approved by the U.S. Food and Drug Administration on February 28, 2017 and by the European Commission on September 19, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with metastatic NETs (mNETs) and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing an additional treatment option for patients suffering from carcinoid syndrome diarrhea.

XERMELO (telotristat ethyl) Important Safety Information

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Warnings and Precautions: XERMELO may cause constipation, which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe, persistent, or worsening abdominal pain develops.
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Adverse Reactions: The most common adverse reactions (≥5%) include nausea, headache, increased gamma-glutamyl-transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia.
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Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. If combination treatment with XERMELO and short-acting octreotide is needed, administer short-acting octreotide at least 30 minutes after administering XERMELO.

For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.

About Sotagliflozin

Sotagliflozin is an investigational oral dual inhibitor of two proteins responsible for glucose regulation known as sodium-glucose co-transporter types 1 and 2 (SGLT1 and SGLT2). SGLT1 is responsible for glucose absorption in the gastrointestinal tract, and SGLT2 is responsible for glucose reabsorption by the kidney.

Lexicon entered into a collaboration and license agreement with Sanofi in November 2015 under which Lexicon granted Sanofi an exclusive, worldwide (excluding Japan), royalty-bearing right and license to develop, manufacture and commercialize sotagliflozin. Lexicon is responsible for all clinical development activities relating to type 1 diabetes and has exercised an exclusive option to co-promote and have a significant role, in collaboration with Sanofi, in the commercialization of sotagliflozin for the treatment of type 1 diabetes in the U.S. Sanofi is responsible for all clinical development and commercialization of sotagliflozin for the treatment of type 2 diabetes worldwide (excluding Japan) and is solely responsible for the commercialization of sotagliflozin for the treatment of type 1 diabetes outside the U.S. (excluding Japan). A New Drug Application and Marketing Authorization Application for sotagliflozin in type 1 diabetes are currently under review at the U.S. Food and Drug Administration and European Medicines Agency, respectively. The product has not yet been approved for use in the U.S., European Union or any other jurisdiction.

Flow Pharma, Inc. and OncoImmunity AS partner for the Development of FlowVaxTM Personalized Cancer Vaccines

On March 12, 2019 Flow Pharma Inc., a San Francisco Bay Area biotechnology company focused on developing personalized cancer vaccines, reported a collaboration with Norwegian bioinformatics company OncoImmunity AS (Press release, Flow Pharma, MAR 12, 2019, View Source [SID1234553907]).

Under the agreement OncoImmunity will license the Immuneoprofiler software to Flow Pharma to support the clinical development and commercialisation of products based on the FlowVax platform. The Immuneoprofiler has recently received the CE-IVD mark certification as a medical device, and fills some of the key gaps in neoantigen prediction. The FlowVax vaccine platform developed by Flow Pharma, is optimized to simultaneously deliver multiple neoantigen peptide targets to enhance tumor-specific killer T-cell attack. OncoImmunity will develop a customised machine-learning layer, using clinical data generated by Flow Pharma, that will work-in-sync with the Immuneoprofiler software to make predictions tailored to the FlowVax platform.

"This exciting partnership and licensing deal represents a fantastic opportunity for OncoImmunity to apply its Immuneoprofiler prediction software to the FlowVax technology in a clinical setting. It also affords OncoImmunity the opportunity to leverage its in-depth knowledge of immune-informatics and expertise in machine learning to develop a customised solution for the FlowVax platform that will support Flow Pharma’s needs now and in the years ahead" said Richard Stratford., Chief Executive Officer at OncoImmunity.

"We believe that OncoImmunity’s Immuneoprofiler software represents the best-in-class solution for predicting clinically relevant bona fide neoantigens. Flow Pharma’s bioinformatics team will be working closely with OncoImmunity AS to customize the way we utilize Immuneoprofiler in order to optimize the use of the data we will be generating" said Reid Rubsamen, M.D., CEO of Flow Pharma.

Secura Bio Acquires Global Rights To Farydak®

On March 12, 2019 Secura Bio, Inc. (www.securabio.com), an integrated, commercial-stage biopharmaceutical company dedicated to the worldwide commercialization of significant oncology therapies, reported that it has acquired the global rights to Farydak (panobinostat) from Novartis (Press release, Secura Bio, MAR 12, 2019, View Source [SID1234553835]).

Farydak (panobinostat) is a prescription oral medication used in combination with proteasome inhibitors and corticosteroids to treat patients with relapsed or refractory multiple myeloma who had received prior treatments. Farydak is a member of the histone deacetylase (HDAC) inhibitor family with a very potent and unique mode of action slowing the growth of multiple myeloma cells.

Multiple myeloma is a cancer that forms malignant plasma cells in the bone marrow, and is the second most common blood cancer with over 160,000 cases diagnosed globally per year, with over 30,000 in the United States. Despite the increased availability of treatments, the disease is characterized by recurrent relapses and remains incurable for most patients leading to approximately 106,000 deaths per year worldwide, over 12,000 of which are in the United States.

On February 23, 2015 Farydak received FDA accelerated approval for use in patients with multiple myeloma, and on August 28, 2015 it was approved by the European Medicines Agency for the same use. Farydak has received marketing authorization in 53 countries to date for the treatment of patients with multiple myeloma.

Secura Bio is dedicated to working with health care professionals to ensure Farydak’s benefit to patients is well understood by cancer care teams and by ensuring that the medicine is available to those who need it. Secura Bio is also investigating the opportunity to develop new potential dosages and product combinations for Farydak.

"We are very pleased to acquire worldwide rights to Farydak as we execute our plan to aggressively build a meaningful, worldwide oncology portfolio," said Joseph M. Limber, President and CEO of Secura Bio. "Farydak will be a critical cornerstone of our strategy, as it offers a potentially valuable and high-growth treatment option for physicians in their therapeutic regimen for multiple myeloma patients when combined with a variety of other multiple myeloma therapies."

"Farydak represents an exciting agent with a unique mechanism of action that is part of a promising class of drugs in this setting," said study investigator Paul Richardson, MD, Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at Dana–Farber Cancer Institute. "Importantly, Farydak has been shown to improve progression–free survival in relapsed multiple myeloma patients who have received at least two prior regimens, including bortezomib and an IMiD, which is an area of particular unmet medical need."

About Farydak (panobinostat)

Farydak (panobinostat) is a histone deacetylase (HDAC) inhibitor that inhibits the enzymatic activity of HDACs at nanomolar concentrations. HDACs catalyze the removal of acetyl groups from the lysine residues of histones and some non-histone proteins. Inhibition of HDAC activity results in increased acetylation of histone proteins, an epigenetic alteration that results in a relaxing of chromatin, leading to transcriptional activation. In vitro, panobinostat caused the accumulation of acetylated histones and other proteins, inducing cell cycle arrest and/or apoptosis of some transformed cells. Increased levels of acetylated histones were observed in xenografts from mice that were treated with panobinostat. Panobinostat shows more cytotoxicity towards tumor cells compared to normal cells.

About multiple myeloma

Epigenetics is the cell programming that governs gene expression and cell development. In multiple myeloma, the normal epigenetic process is disrupted (also called epigenetic dysregulation) resulting in the growth of cancerous plasma cells, potential resistance to current treatment, and ultimately disease progression.

Multiple myeloma impacts approximately 81,000 people in the United States. Multiple myeloma is a cancer of the plasma cells, a kind of white blood cell present in bone marrow—the soft, blood–producing tissue that fills the center of most bones. The cancer is caused by the production and growth of abnormal cells within the plasma, which multiply and build up in the bone marrow, pushing out healthy cells and preventing them from functioning normally. Multiple myeloma is an incurable disease with a high rate of relapse (when the cancer returns) and resistance (when the therapy stops working), despite currently available treatments. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40.

Farydak Important Safety Information

Farydak can cause serious side effects, including diarrhea and heart problems.

Diarrhea is common with Farydak and can be severe. Patients should tell their healthcare provider (HCP) right away if they have abdominal (stomach) cramps, loose stool, diarrhea, or feel like they are becoming dehydrated. HCPs may prescribe medicines to help prevent or treat these side effects. Taking or using stool softeners or laxative medicines may worsen diarrhea, patients should talk to their HCP before taking or using these medicines.

Farydak can cause severe heart problems which can lead to death. Risk of heart problems may be increased with a condition called "long QT syndrome" or other heart problems. Patients should call their HCP and get emergency medical help right away if they have any of the following symptoms of heart problems: chest pain, faster or slower heartbeat, palpitations (feel like heart is racing), feel lightheaded or faint, dizziness, blue colored lips, shortness of breath, or swelling in legs.

Farydak can cause severe bleeding which can lead to death. It may take patients longer than usual to stop bleeding while taking Farydak. Patients should tell their HCP right away if they get any of the following signs of bleeding: blood in stools or black stools (look like tar), pink or brown urine, unexpected bleeding or bleeding that is severe or that cannot be controlled, vomit blood or vomit looks like coffee grounds, cough up blood or blood clots, increased bruising, feeling dizzy or weak, confusion, change in speech, or headache that lasts a long time.

Farydak is a prescription medicine used, in combination with bortezomib and dexamethasone, to treat people with a type of cancer called multiple myeloma after at least two other types of treatment have been tried. It is not known if Farydak is safe and effective in children.

Patients should tell their HCP about all of the medicines they take, including prescription and over–the–counter medicines, vitamins and herbal supplements.

Patients should take Farydak exactly as the HCP tells them to take it. The HCP will tell patients how much Farydak to take and when to take it. The HCP may change the dose or stop treatment temporarily if patients experience side effects. Patients should not change the dose or stop taking Farydak without first talking with their HCP.

Patients should avoid eating star fruit, pomegranate or pomegranate juice, and grapefruit or grapefruit juice while taking Farydak. These foods may affect the amount of Farydak in the blood.

Low blood cell counts are common with Farydak and can be severe. Low platelet count (thrombocytopenia) can cause unusual bleeding or bruising under the skin. Low white blood cell count (neutropenia) can cause infections. Low red blood cell count (anemia) may make a patient feel weak, tired, or they may get tired easily, look pale, or feel short of breath.

There is an increased risk of infection while taking Farydak. Patients should contact their HCP right away if they have a fever or have any signs of an infection including sweats or chills, cough, flu–like symptoms, shortness of breath, blood in phlegm, sores on body, warm or painful areas on body, or feeling very tired.

Patients should call their HCP right away with any of the following symptoms of liver problems: feel tired or weak, loss of appetite, dark amber colored urine, upper abdominal pain, yellowing of skin or the white of eyes.

The most common side effects of Farydak include tiredness, nausea, swelling in arms or legs, decreased appetite, fever and vomiting. Patients should tell their HCP if they have any side effect that is bothersome or that does not go away.

Scholar Rock to Develop Cancer Immunotherapy Product Candidate, SRK-181, a Selective Inhibitor of TGFβ1 Activation, to Overcome Checkpoint Inhibitor Resistance

On March 12, 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 that it has 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 its preclinical data and human translational insights (Press release, Scholar Rock, MAR 12, 2019, View Source [SID1234537354]). Scholar Rock has initiated manufacturing and is progressing preclinical development efforts with plans to initiate a Phase 1 trial in patients with solid tumors in mid- 2020.

"Given that a majority of cancer patients fail to respond to checkpoint blockade therapies, we are eager to advance the next product candidate from our pipeline of growth factor modulators to potentially address a key mechanism of pre-existing resistance," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "A growing body of evidence strongly implicates elevated TGFβ1 activity as a cause of immunotherapy failure, and we see tremendous potential for SRK-181 to expand the number of patients who could benefit from checkpoint blockade therapies by potently and selectively inhibiting the activation of TGFβ1."

SRK-181 is a fully human antibody designed to bind to, and prevent the activation of, latent TGFβ1 with high affinity and high selectivity, as evidenced by minimal or no binding to latent TGFβ2 and latent TGFβ3 isoforms. Several important factors led to the decision to advance SRK-181 as a clinical development product candidate for the treatment of tumors resistant to checkpoint blockade therapies (CBTs), such as anti-PD1 antibodies. These factors include:

TGFβ signaling has been implicated as a culprit in primary resistance to CBTs in multiple peer-reviewed studies.
Translational data analyses by Scholar Rock highlight the prominent expression of TGFβ1 in many human tumor types, such as bladder cancer, non-small cell lung cancer and melanoma, for which CBTs have either been approved or demonstrated clinical activity in trials.
Clinical correlation and preclinical model data suggest that TGFβ1 excludes effector cell entry into the tumor, thereby limiting immune system access to tumor cells.
Preclinical studies in syngeneic mouse tumor models resistant to CBT show SRK-181-mIgG1 (the murine version of SRK-181), when combined with anti-PD1 antibodies, permitted effector T cell infiltration and expansion into the tumor microenvironment and led to tumor regression or control as well as significant survival benefit.
A 28-day pilot toxicology study of SRK-181 in adult rats showed no observed drug-related toxicity up to a weekly dose of 100 mg/kg for 4 weeks.
Detailed preclinical results for SRK-181-mIgG1 (formerly referred to as SRTβ1-Ab3) were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting in November 2018. The poster presented at SITC (Free SITC Whitepaper) can be accessed by visiting the Scholar Rock website at View Source Additional preclinical data for SRK-181-mIgG1 will be presented at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting scheduled to take place March 29 to April 3, 2019 in Atlanta, GA.

About SRK-181
SRK-181 is a highly specific inhibitor of TGFβ1 activation being developed to overcome primary resistance to checkpoint blockade therapies (CBTs). TGFβ1 is the predominant TGFβ isoform expressed in many human tumors, particularly for those tumors where checkpoint therapies are currently approved. Based on analyses of human tumors that are resistant to CBT, TGFβ1 is implicated as a key contributor to exclude immune cell entry into the tumor microenvironment, thereby preventing normal immune function. By overcoming this immune cell exclusion, SRK-181 has the potential to induce tumor regression when administered in conjunction with CBT.