On April 1, 2021 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform oncology drug discovery and development, reported a collaboration with Johns Hopkins Pediatric Oncology Division of The Sidney Kimmel Comprehensive Cancer Center and Dr. Eric Raabe, M.D., Ph.D. focused on Lantern’s drug candidate LP-184 in the area of brain tumors, and specifically in Atypical Teratoid Rhabdoid Tumors ("ATRT"), an ultra-rare and fast-growing cancerous tumor of the brain that presents primarily in children (Press release, Lantern Pharma, APR 1, 2021, View Source [SID1234577504]).

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"As we enriched our RADR A.I. platform for additional cancer indications, we began to discover common molecular pathways that drive response to our drug candidate, LP-184, across multiple additional CNS cancers," stated Panna Sharma, President and CEO of Lantern Pharma. "Chief among these newly identified CNS cancers was ATRT, an ultra-rare and fast-growing cancerous tumor of the brain that presents primarily in children with no effective therapies. The urgency of directing LP-184 towards helping children battle this particularly aggressive cancer was self-evident, as was the opportunity to collaborate with the Johns Hopkins’ pediatric oncologist, Dr. Eric Raabe, who has devoted his career to studying pediatric brain cancers, including ATRT."

Rhabdoid tumors (RTs) can emerge in the brain, kidneys, liver and all compartments of the central nervous system ("CNS"). Approximately 66% of RTs occur in the CNS and are called ATRTs. ATRTs predominantly affect infants and young children, with up to 15% of ATRTs arising in the brain. Incidence of ATRT is between 1.4 and 3.0 per million, and the survival rate is between 10% and 15% depending on the age at diagnosis. Pediatric brain cancer is the second-leading cause of pediatric cancer death with the incidence rate growing at ~2.7% per year in the United States.

Dr. Eric Raabe, M.D., Ph.D., is assistant professor of oncology in the Division of Pediatric Oncology at Johns Hopkins and a co-principal investigator at the Pacific Pediatric Neuro-Oncology Consortium. A physician-scientist, Dr. Raabe has devoted his career to the pursuit of treatment options for the most high-risk pediatric brain cancers, including ATRT where Dr. Raabe uses a unique and highly curated panel of cell lines and xenografts in preclinical studies for drug development and research. These models have had extensive molecular and genomic profiling including biomarker studies to help better understand the ATRT and other related CNS cancers.

Over 90% of cases of ATRT are caused by a mutation which drives a partial or whole loss of chromosome 22, resulting in the inactivation of the SMARCB1 gene (Switch/sucrose nonfermentable [SWI/SNF] related, Matrix-associated, Actin-dependent Regulator of Chromatin, subfamily B1). SMARCB1 is a protein encoding and tumor suppressor gene which drives downstream production of the SMARCB1 protein and other SWI/SNF protein subunits which are thought to act as tumor suppressors. While ATRT is diagnosed with standard immunochemistry staining to detect loss of the respective protein(s), no standard of care currently exists for ATRT and ATRT in the brain is typically unresectable. Treatment options are typically limited to only chemotherapy agents since radiotherapy is not advised in children.

"To support the discovery and development of innovative medicines that may help children diagnosed with rare diseases, the U.S. FDA has created a Rare Pediatric Disease Designation. We believe that the rarity of incidence of ATRT in the U.S and its prevalence in children supports the potential for LP-184 to qualify in the future for a possible grant by the US. FDA for a Rare Pediatric Disease Designation for use of LP-184 for ATRT," continued Mr. Sharma. "Moreover, if we are successful in receiving a Rare Pediatric Disease Designation, we believe LP-184, if it receives ultimate approval, may possibly qualify for the granting by the U.S. FDA of a Rare Pediatric Disease Priority Review Voucher ("PRV"). We believe the award of a PRV would represent a significant value enhancing milestone for Lantern Pharma."

Lantern Pharma plans on continuing to use RADR to potentially uncover and develop other indications in brain and CNS cancers where LP-184 has the potential to show efficacy.

On April 1, 2021 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported the completion of enrollment for the first-line patient cohort in the DisTinGuish study, a clinical trial evaluating Leap’s anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with tislelizumab, BeiGene Ltd.’s anti-PD-1 antibody, with or without chemotherapy, in patients with gastric or gastroesophageal junction cancer (G/GEJ) (Press release, Leap Therapeutics, APR 1, 2021, View Source [SID1234577503]).

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"The completion of enrollment for the first-line patients in the DisTinGuish study is an important milestone for the DKN-01 and tislelizumab combination development program," said Cynthia Sirard, M.D., Chief Medical Officer of Leap Therapeutics. "In collaboration with our partner, BeiGene, we are committed to realizing the potential of DKN-01 as part of a new combination therapy with tislelizumab aimed at treating gastric and gastroesophageal junction cancer patients, where a high global unmet medical need remains."

The DisTinGuish trial (NCT04363801) is a Phase 2a, nonrandomized, open-label, multicenter study of DKN-01 in combination with tislelizumab with or without chemotherapy as first-line or second-line therapy in adult patients with inoperable, locally advanced G/GEJ adenocarcinoma. The study, which will be conducted in two parts in the United States and the Republic of Korea, includes up to 24 patients with first-line G/GEJ cancer and up to 48 patients with second-line G/GEJ cancer whose tumors express high levels of DKK1. Initial data is expected in the second half of 2021. Leap is conducting this combination study as part of an exclusive option and license agreement with BeiGene for the development of DKN-01 in Asia (excluding Japan), Australia, and New Zealand. Leap retains exclusive rights for the development, manufacturing, and commercialization of DKN-01 for the rest of the world.

About gastric / gastroesophageal junction cancer
Gastric adenocarcinoma (gastric cancer) remains one of the most common and deadly cancers worldwide, especially among older malesi. Based on GLOBOCAN 2018 data, stomach cancer is the 5th most common neoplasm and the 3rd most deadly cancer, with an estimated 783,000 deaths globally in 2018i. Ninety-five percent of cancers of the stomach are adenocarcinomasi. Gastric cancer incidence and mortality are highly variable by region and highly dependent on diet and Helicobacter pylori infectioni. The gastroesophageal junction (GEJ) is the area where the esophagus and stomach join together. Given its anatomic location, GEJ adenocarcinomas have often been grouped together with either esophageal or gastric cancers in clinical trials.

About DKN-01
DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein. DKK1 modulates the Wnt/Beta-catenin and PI3kinase/AKT signaling pathways, which have an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK cell ligands on tumor cells. The U.S. Food and Drug Administration has granted DKN-01 Orphan Drug Designation for the treatment of gastric and gastroesophageal junction cancer and Fast Track Designation in combination with tislelizumab for the treatment of patients with gastric and gastroesophageal junction adenocarcinoma whose tumors express high DKK1 protein, following disease progression on or after prior fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, human epidermal receptor growth factor (HER2)/neu-targeted therapy.

On April 1, 2021 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported the successful completion of the cash tender offer, through a subsidiary, for all of the outstanding shares of common stock of Pandion Therapeutics, Inc. (Nasdaq: PAND) at a purchase price of $60 per share (Press release, Merck & Co, APR 1, 2021, View Source [SID1234577502]). As of the tender offer expiration, 27,770,123 shares of common stock of Pandion were validly tendered and not withdrawn from the tender offer, representing approximately 88.6% percent of the outstanding common stock of Pandion on a fully diluted basis. All such shares have been accepted for payment in accordance with the terms of the tender offer, and Merck expects to promptly pay for such shares.

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Following the finalization of the tender offer, Merck completed the acquisition of Pandion today through a merger of Merck’s wholly-owned subsidiary with and into Pandion in which all shares not tendered into the offer were cancelled and converted into the right to receive cash equal to the $60 offer price per share, without interest, less any applicable tax withholding. At the completion of the merger, Pandion became a wholly-owned subsidiary of Merck. The common stock of Pandion will no longer be listed or traded on the Nasdaq Global Select Market.

On April 1, 2021 Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) reported it has acquired Zikani Therapeutics, Inc. in an all-stock transaction, with the potential to create a leader in ribosomal RNA-targeted therapies for treatment of rare diseases and oncology (Press release, Zikani Therapeutics, APR 1, 2021, View Source [SID1234577500]). Sumit Aggarwal, previously the President and Chief Executive Officer of Zikani, has been named President and Chief Executive Officer of Eloxx, and Vijay Modur, M.D., Ph.D., who was Zikani’s Chief Scientific and Medical Officer, has been named Eloxx’s Head of Research and Development.

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"With the strength of our ELX-02 program for cystic fibrosis, this acquisition provides us with the opportunity to amplify the potential of our innovative science by developing a new class of therapies to treat diseases with limited to no treatment options under the stewardship of leaders with a proven ability to translate technology into treatments for patients," said Tomer Kariv, Eloxx Chairman.

"We are excited about the potential of ELX-02 and combining the companies opens the door to build a leadership position in genetic therapy by rapidly developing treatments that can restore functional proteins in patients with nonsense mutations in their RNA," said Aggarwal. "The combined capabilities of Eloxx and Zikani in chemistry, biology, regulatory and drug development, including Zikani’s TURBO-ZMTM synthetic chemistry platform for designing macrolide-based Ribosome Modulating Agents (RMAs), along with a committed leadership team and talented employees, will further accelerate our ability to impact the lives of those who have rare diseases with the type of urgency and novel thinking that they deserve," added Aggarwal.

ELX-02 is currently in Phase 2 clinical trials in Cystic Fibrosis (CF) patients affected by nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The investigational therapy has shown strong activity across a full range of mutations in CF preclinical models. In Phase 1 testing, ELX-02 was generally well- tolerated and demonstrated high bioavailability with consistent pharamacokinetics across both single and multiple-dose studies.

"The Phase 2 trials are designed to validate the safety of ELX-02 and assess its biological activity. We look forward to completing enrollment in the first four treatment arms by mid-year and reporting data from these treatment arms in the second half of this year," said Dr. Modur.

In addition to CF, the company plans to file an IND in 2022 for what could potentially become the first oral therapy for protein restoration for patients with nonsense mutations in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB). RDEB is an incurable, extremely painful and often fatal skin blistering condition caused by a lack of collagen type VII that is estimated to affect more than 3,000 people worldwide. JEB is the most severe form of EB, with most patients dying in infancy.

By extending the application of ribosomal RNA modulation to the readthrough of nonsense mutations in tumor suppressor genes, the company is also rapidly advancing preclinical research for familial adenomatous polyposis (FAP), an inherited pre-cancerous colorectal disease frequently caused by nonsense mutations in the adenomatous polyposis coli (APC) gene.

Nonsense mutations cause approximately 10-12 percent of rare inherited diseases. ELX-02 along with the TURBO-ZM library of compounds are anticipated to significantly expand to include the treatment of many other rare diseases and certain cancers.

Acquisition Terms

Under the terms of the merger agreement, stockholders of Zikani received approximately 7.6 million Eloxx common shares and own approximately 16 percent of the combined company.

Board and Management Changes

In connection with the acquisition, Silvia Noiman, Ph.D., and Martijn Kleijwegt have stepped down from the Eloxx Board. Alan Walts, Ph.D., and Raj Parekh, Ph.D., who have both served as Zikani directors, were appointed to fulfill the vacancies and serve out the remaining terms of office.

"We’re pleased to welcome Sumit and Vijay to the Eloxx leadership team. They demonstrated their ability to transform Zikani by following the science and pursuing the creation of a new class of therapies on behalf of patients with unmet medical need. We want to extend our thanks and appreciation to Dr. Greg Williams for his stewardship of Eloxx and his commitment to advancing the critical work of the company. We are pleased that Greg will continue to advise Eloxx to facilitate a smooth transition," said Kariv.

Live Webcast: accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or by clicking here. A replay of this conference call will be available on the Eloxx and Zikani websites.

Leadership Profiles

Sumit Aggarwal

Sumit Aggarwal served as Zikani’s President and CEO. He has led the transformation of Zikani from an early-stage technology company to a development-stage rare disease and oncology-focused organization. Under Aggarwal’s leadership, Zikani has concentrated its focus on demonstrating pre-clinical proof of efficacy across several disease states using its TURBO-ZM technology platform.

In his more than 20 years in pharmaceutical and biotechnology commercial operations, investment management and management consulting, Aggarwal has been successful in transforming companies by re-invigorating innovation, growth and profitability, and raising capital for promising technology companies.

Prior to joining Zikani, he reinvigorated growth and profitability at Progenity, raised $125 million in capital and built a novel drug delivery-based GI pipeline. He also held leadership roles in healthcare and biotechnology at Adage Capital and as an Associate Partner at McKinsey & Company in its healthcare practice.

Aggarwal has an MBA with distinction from the Johnson School, Cornell University, and a Bachelor of Technology with Honors in Chemical Engineering from the Indian Institute of Technology, Kharagpur.

Vijay Modur, M.D., Ph.D.

Vijay Modur, M.D., Ph.D., served as Zikani’s Chief Scientific and Medical Officer and has led the scientific efforts to transform medicines based on ribosomal modulation using Zikani’s proprietary TURBO-ZM technology platform.

In his more than 20 years in pharmaceutical and diagnostic roles in R&D, he has successfully translated research discovery efforts into products that have impacted medical practice.

Prior to joining Zikani, Dr. Modur led the venglustat rare disease program at Sanofi across multiple rare disease indications into Phase 2 and Phase 3 clinical development along with leading other early development programs. Prior to Sanofi, he held leadership roles in HTG Molecular, Novartis Oncology and Merck Research Labs.

Dr. Modur obtained his MBBS from Karnatak University and his Ph.D. from the University of Utah. He was a resident in Clinical Pathology at Washington University School of Medicine where he also completed his post-doctoral fellowship.

On April 1, 2021 The Mt. Sinai Health Care Foundation—continuing Mt. Sinai Medical Center’s century-old tradition of caring for Northeast Ohio—reported that has expanded its commitment to the region through a $1 million challenge grant to the Case Western Reserve University School of Medicine (Press release, Case Western Reserve University, APR 1, 2021, View Source [SID1234577499]). With this grant, the Mt. Sinai Health Care Foundation has provided over $31 million in lifetime support for Case Western Reserve.

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The grant will benefit the Dean’s Catalytic Fund, designed to provide resources for the school’s leader to invest in new opportunities to advance medicine, such as emergency student scholarship support, bridge funding to advance promising early-stage research, and recruitment and retention of exceptional faculty.

In this instance, Interim Dean Stan Gerson will use Mt. Sinai’s support to inspire additional philanthropic commitments for recruitment of the next chair of the school’s department of pharmacology.

Within the school, the Department of Pharmacology is a core basic science department which historically and currently houses some of the schools most accomplished scientists, investigators, and educators of both graduate students and medical students. Expertise within the department ranges from basic pathway discovery of physiologic, organelle and disease processes to the discovery of interventions in diseases, from drugs to peptides to gene correction strategies, and across a wide spectrum of diseases. Technology expertise from structural biology, CRYOEM, imaging technologies, DNA and energy metabolism, cell biology and drug metabolism all reflect the wide range of capabilities that will be expanded with the recruitment of the new department chair.

"Funds generated through this new challenge will help us expand on existing strengths in pharmacology," Gerson said, "and help ensure our continued success as a leader in research, medical and graduate education. Expansion of the department will enhance coordination with our hospital affiliates, to improving the health of our community."

That dedication to improving care for the people of Cleveland and beyond is a core principle of the Mt. Sinai Health Care Foundation. It’s the same commitment to people and partnerships that earned the former medical center a national reputation.

"For 25 years, the Case Western Reserve School of Medicine has been among Mt. Sinai Health Care Foundation’s highest-performing grantee partners," said Mitchell Balk, president of The Mt. Sinai Health Care Foundation, the philanthropic legacy of the former nonprofit health care center. "We know that when we invest in the School of Medicine, our investments will increase many fold and that the dividends paid will benefit not only Case Western Reserve, not only Cleveland’s bioscience sector, but indeed, all of humankind."

Gerson is grateful to the foundation and its shared vision for bolstering Northeast Ohio’s reputation as a global biomedical leader. "On behalf of the School of Medicine," he said, "I cannot thank Mitch and the Mt. Sinai Health Care Foundation enough for their incredible support."