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]).

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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.

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"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.

Dong-A ST and Beactica expand their research and license agreement to develop new cancer treatments

On March 12, 2019 Dong-A ST Co., Ltd. (170900: Korea SE), the Korean pharmaceutical company, and Beactica AB, the Swedish drug discovery company, reported an expansion of their research and licensing agreement (Press release, Dong-A ST, MAR 12, 2019, View Source [SID1234535724]). The collaboration – which was initiated in October 2016 – is now expanded with an aim to jointly identify and develop novel small molecules targeting a protein–protein interaction of therapeutic relevance for immuno-oncology. The partnership further builds on Beactica’s unique early-stage lead generation capabilities and Dong-A ST’s strengths in the pharmacological proof of novel target concepts as well as downstream pre-clinical and clinical development of new therapeutic agents.

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Under the terms of the additional agreement, Dong-A ST will gain exclusive global rights for the further development and commercialization of compounds discovered. In return, Beactica is eligible to receive research funding as well as milestone payments for certain research, preclinical, clinical and regulatory achievements as well as royalties on commercial sales of the products resulting from the partnership. Beactica is also entitled to a revenue share from any related future licensing activities by Dong-A ST. Full financial details remain undisclosed.

"The collaboration between Dong-A ST and Beactica brings together our complementary strengths and establishes a powerful platform for the discovery and development of next generation anti-cancer therapeutics" said Mr Daesik Eom, Chairman and CEO of Dong-A ST. "Beactica’s capabilities and expertise accelerates the advancement of Dong-A ST’s oncology pipeline and will enhance Dong-A ST’s global competitiveness in the pharmaceutical industry."

"Following recent success in our collaboration we are pleased with Dong-A’s confidence and trust to expand this partnership into new target areas." said Dr Per Källblad, CEO of Beactica. "As a long-term strategic partner we are proud to have delivered valuable contributions to Dong-A’s research and look forward to a continued successful collaboration."

Slide Presentation – 2019 Analyst Day

On March 19, 2019 Ligand presented Analyst Day presentation (Presentation, Ligand, MAR 12, 2019, View Source [SID1234534297]).

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Onconova Therapeutics, Inc. to Participate at the 31st Annual ROTH Conference March 17-19, 2019 in Orange County, CA

On March 12, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes, reported that the Company will participate in the 31st Annual ROTH Conference to be held March 17-19, 2019 at the Ritz Carlton Laguna Niguel in Orange County, CA (Press release, Onconova, MAR 12, 2019, View Source [SID1234534298]).

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Steven M. Fruchtman, MD, President & CEO, and Mark Guerin, CFO, will attend the conference and be available to meet with investors. Dr. Fruchtman will also participate on a panel to discuss myeloid diseases on Monday, March 18th, from 3:00-4:00p.