On September 22, 2020 Lantern Pharma (Nasdaq: LTRN), a clinical-stage biopharma company using its proprietary RADR artificial intelligence ("A.I.") platform to improve drug discovery & development and identify patients who will benefit from its targeted oncology therapeutics, reported a collaboration and research agreement with Fox Chase Cancer Center for the further development of Lantern’s LP-184 in pancreatic cancer (Press release, Lantern Pharma, SEP 22, 2020, View Source [SID1234565493]). Based in Philadelphia, Fox Chase is a leading research center for pancreatic cancers and one of the four original cancer centers to receive comprehensive cancer center designation from the National Cancer Institute (NCI).

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The Fox Chase collaboration will focus on advancing the targeted use of LP-184 in molecularly-defined sub-types of pancreatic cancer. The goal of the collaboration is to create a more biologically relevant and robust gene signature in preparation for future clinical trials, enabling pancreatic cancer patients to potentially benefit from a more effective and personalized cancer therapy.

"Collaborations with world-leading cancer centers are an essential part of our strategy to rapidly advance the insights driving our therapeutic programs and grow our RADR A.I. platform by adding millions of new, unique, and proprietary data points," said Panna Sharma, CEO of Lantern Pharma. "This relationship with Fox Chase will allow us to use state-of-the-art models and biological methods to add more physiologically relevant data and insights into the mechanisms of LP-184, and will further shape our algorithms for how certain compounds interact with specific tumor types. The unique insights we gain will equip Lantern with critical advantages in our aim of accelerating LP-184’s path to clinical trials and ultimately commercialization, while saving millions of dollars in development costs. This data-enabled, and biomarker-based approach has the potential to meaningfully bend the cost curve of cancer drug development and help bring personalized cancer therapies to patients with reduced economic burden, and greater efficacy."

The research will be led by Igor Astsaturov, MD, Ph.D., an internationally-recognized researcher in gastrointestinal cancers at the Molecular Therapeutics Program at Fox Chase where he specializes in investigating signaling pathways that inform the choice of biomarkers and innovative therapy combinations in clinical trials. Dr. Astsaturov is known for his research in a number of cancer indications spanning pancreatic, stomach, liver, and several others, as well as his belief that each individual cancer patient will soon be defined by the molecular makeup of their cancer cells.

LP-184 is a DNA-damaging small molecule drug candidate currently in preclinical development for certain genomically defined solid tumors, including pancreatic cancer. As a next-generation alkylating agent that preferentially damages DNA in cancer cells that overexpress certain biomarkers, LP-184 has the potential to be used as both monotherapy as well as a synergistic agent in combination with other drugs.

"We are very pleased to partner with Lantern Pharma in establishing a collaboration that will play an important role in our research," said Igor Astsaturov, MD, PhD, and Associate Professor, Department of Hematology/Oncology at Fox Chase. "Our advanced research approach using patient-derived cancer models will provide us with critical insights into the efficacy of LP-184 in pancreatic cancers. We look forward to sharing these results with the broader scientific community and hopefully bringing this drug to cancer patients that can best benefit from this compound."

The research program is at the forefront of translational cancer medicine and will use patient-derived cancers that are grown in the lab and transformed into physiologically relevant 3D organoids and PDx models. This innovative approach allows researchers to more precisely understand the biology of what actually happens inside the cancer tumor, which will more accurately establish the precise biomarker signatures and help provide data-driven insight into additional mechanisms that can be leveraged in the fight against pancreatic cancer.

Among several objectives, the research will determine whether the overexpression of the gene PTGR1, a biomarker that has been linked to cancer cell proliferation, will indicate heightened sensitivity to LP-184 and a more favorable response rate and efficacy as compared to standard of care agents. LP-184 has been advanced using Lantern’s proprietary RADR A.I. platform that leverages over 500 million data points, machine learning, genomics, and computational biology to accelerate the discovery of potential mechanisms of action, and genomic and biomarker signatures that correlate to drug response in cancer patients.

Although significant recent advances have been made in the use of targeted and biomarker-based therapies in cancer, pancreatic cancer remains an area that has not experienced significant improvement in patient outcomes. The overall five-year survival rate for pancreatic cancer across all stages remains at only 10.0% in the US and 8.2% globally, and pancreatic cancer is expected to become the 2nd leading cause of cancer death in the USA in 2020 behind lung cancer according to the National Cancer Institute’s SEER Stat Database.

On September 22, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported positive top-line results from the Phase 1 EXPLORER and Phase 2 PATHFINDER clinical trials of AYVAKIT (avapritinib) in patients with advanced systemic mastocytosis (SM) (Press release, Blueprint Medicines, SEP 22, 2020, View Source [SID1234565492]). Consistent with previously reported EXPLORER trial results, the registrational data for AYVAKIT showed profound reductions in mast cell burden, high overall response and complete remission rates, and durable clinical benefit, including prolonged median overall survival (OS). AYVAKIT was generally well-tolerated, with an improved safety profile at the 200 mg once daily (QD) dose. Based on these data, Blueprint Medicines plans to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for AYVAKIT for the treatment of advanced SM in the fourth quarter of 2020.

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SM is a rare, debilitating disease driven by the KIT D816V mutation in nearly all patients. Uncontrolled mast cell proliferation and activation may lead to life-threatening complications across the SM patient population. In advanced SM subtypes, the median OS is approximately 3.5 years in aggressive SM (ASM), approximately two years in SM with an associated hematologic neoplasm (SM-AHN) and less than six months in mast cell leukemia (MCL). AYVAKIT, an investigational precision therapy for the treatment of SM, is the only highly potent KIT D816V inhibitor that has been clinically validated in SM.

"New treatment options are urgently needed to address mast cell infiltration associated with advanced systemic mastocytosis, which often leads to extensive organ damage and poor survival despite existing therapeutic interventions," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "These top-line data underscore the transformative impact shown by AYVAKIT, with patients achieving profound reductions in mast cell burden, durable responses that deepen over time and prolonged overall survival relative to historical outcomes. Based on these positive results, we aim to rapidly bring this promising treatment to patients, with the goal of improving and extending their lives beyond what is possible with currently available therapy."

Top-line EXPLORER and PATHFINDER Trial Data

Across both trials, 85 patients were evaluable for response per the modified IWG-MRT-ECNM criteria (IWG criteria), including 44 patients treated with a starting dose of 200 mg QD. Top-line results are being reported as of a data cutoff date of May 27, 2020 in the EXPLORER trial and a data cutoff date of June 23, 2020 in the PATHFINDER trial, with response assessments per central review completed in September 2020. Registrational endpoints are overall response rate (ORR) and duration of response (DOR), based on central review. ORR was defined as complete remission with full or partial recovery of peripheral blood counts (CR/CRh), partial remission or clinical improvement. All reported clinical responses were confirmed.

In the EXPLORER trial, 53 patients were response evaluable, with a median follow-up of 27.3 months. In EXPLORER, the ORR was 76 percent (95% CI: 62%, 86%), and 36 percent of patients had a CR/CRh. The median DOR was 38.3 months (95% CI: 21.7 months, not estimable). The median OS was not estimable (95% CI: 46.9 months, not estimable).

In a pre-specified interim analysis from the PATHFINDER trial, 32 patients were response evaluable, with a median follow-up of 10.4 months. The ORR was 75 percent (95% CI: 57%, 89%), and 19 percent of patients had a CR/CRh. In addition, the data showed that responses are continuing to deepen over time, at a rate consistent with the EXPLORER trial. The median DOR was not estimable (95% CI: not estimable, not estimable), and OS was not assessed due to the length of time patients have been enrolled in PATHFINDER. The top-line PATHFINDER results were based on a pre-planned analysis designed to assess the superiority of AYVAKIT versus the ORR per IWG criteria previously reported for the multi-kinase inhibitor midostaurin. The interim analysis achieved its primary endpoint with a p-value of p=0.0000000016.

In a pooled efficacy analysis from the 200 mg QD dose group, 44 patients were response evaluable, with a median follow-up of 10.4 months. In this group, the ORR was 68 percent, and 18 percent of patients had a CR/CRh.

Safety data were consistent with previously reported results, and no new signals were observed. AYVAKIT was generally well-tolerated with most adverse events (AEs) reported as Grade 1 or 2. In the EXPLORER and PATHFINDER trials, AYVAKIT demonstrated improved tolerability at a starting dose of 200 mg QD, compared to all doses. Across both trials, 8.1 percent of patients discontinued AYVAKIT due to treatment-related AEs.

Previously reported results from the EXPLORER trial showed that pre-existing severe thrombocytopenia, which occurs in approximately 10 to 15 percent of advanced SM patients based on Blueprint Medicines estimates, and starting doses of 300 mg QD or higher were risk factors for intracranial bleeding (ICB). Based on these data, Blueprint Medicines implemented treatment management guidelines in the EXPLORER and PATHFINDER trials, including exclusion criteria for pre-existing severe thrombocytopenia, routine platelet monitoring and dose interruption guidelines for emergent severe thrombocytopenia. In 76 EXPLORER and PATHFINDER trial patients without pre-existing severe thrombocytopenia treated at the 200 mg QD dose, two patients (2.6 percent) had ICB events. Both AEs were Grade 1 and asymptomatic. These safety data validate the clinical impact of the treatment management guidelines.

Blueprint Medicines plans to present detailed results from the EXPLORER and PATHFINDER trials at a future medical meeting.

Conference Call Information

Blueprint Medicines will host a live webcast today beginning at 8:30 a.m. ET to discuss the top-line results of AYVAKIT in advanced SM. To access the live call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international) and refer to conference ID 5338896. A webcast of the conference call will be available under "Events and Presentations" in the Investors & Media section of Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

About SM

SM is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor OS.

Debilitating symptoms associated with SM, including anaphylaxis, maculopapular rash, pruritis, brain fog, fatigue and bone pain, often persist despite treatment with a number of symptomatic therapies. Patients often live in fear of attacks, have limited ability to work or perform daily activities, or isolate themselves to protect against unpredictable triggers.

Currently, there are no approved therapies that selectively inhibit D816V mutant KIT. A multi-kinase inhibitor, midostaurin, is approved for the treatment of advanced SM and has shown an ORR of 28 percent per IWG criteria, with ORR defined as complete remission, partial remission or clinical improvement.

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit www.AYVAKIT.com.

AYVAKIT is not approved for the treatment of any other indication, including SM, in the U.S. by the FDA or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and indolent SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of ASM, SM-AHN and MCL.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for AYVAKIT in the rest of the world.

About the Clinical Development Program for AYVAKIT in Advanced SM

AYVAKIT is currently being evaluated in two ongoing, registrational clinical trials for advanced SM: the EXPLORER trial (ClinicalTrials.gov Identifier: NCT02561988) and the PATHFINDER trial (ClinicalTrials.gov Identifier: NCT03580655).

The EXPLORER trial is an open-label, single-arm trial designed to identify the recommended Phase 2 dose and demonstrate proof-of-concept in patients with advanced SM. Key trial endpoints include ORR, DOR, quantitative measures of mast cell burden, patient-reported outcomes and safety. The EXPLORER trial has completed patient enrollment.

The PATHFINDER trial is an open-label, single-arm registration-enabling trial designed to confirm the clinical activity of AYVAKIT in patients with advanced SM. Key trial endpoints include ORR, DOR, quantitative measures of mast cell burden, patient-reported outcomes and safety. Patient enrollment is ongoing in the PATHFINDER trial.

On September 22, 2020 AIM ImmunoTech Inc. (NYSE American:AIM) reported receipt of statistically significant positive pancreatic cancer survival results from a multi-year Early Access Program (EAP) conducted at Erasmus University Medical Center in the Netherlands (Press release, AIM ImmunoTech, SEP 22, 2020, View Source [SID1234565491]).

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Prof. Casper van Eijck, MD Ph.D., and his team at Erasmus MC found a statistically significantly positive survival benefit when using Ampligen in patients with locally advanced/metastatic pancreatic cancer after systemic chemotherapy. Prof. van Eijck states, "The overall survival of the experimental group was compared to a large historical control cohort matched for age, gender, stage of disease, and number of cycles of Folfirinox chemotherapy. Median survival was approximately two-fold higher, that is 200%, in the Ampligen arm as compared to the historical controls. These results were obtained with a very high degree of statistical significance. Based on these data, I see the potential for Ampligen as a meaningful extension of the standard of care for advanced pancreatic cancer, which we are planning to investigate further." A detailed clinical report and an article for publication are being prepared by Prof. van Eijck and his team at Erasmus MC.

AIM CEO Thomas K. Equels states: "We started this program in January 2017. These exceptional results from Erasmus exceed even our most optimistic expectations. I am deeply grateful to Prof. Casper van Eijck, his team at Erasmus, and Ronald Brus, MD, the guiding hand at myTomorrows, for their vision and careful diligence in implementing this important analysis of Ampligen as a single-agent therapy for late-stage pancreatic cancer. I extend deep gratitude to the government of the Netherlands for its pioneering support and commitment to advancing a critical medical innovation in a deadly cancer with very limited medical interventions. Medical advances in lethal unmet medical needs depend on this sort of outstanding clinical and governmental cooperation and support. We could not have accomplished this but for the support of the Netherlands."

AIM will work with its Contract Research Organization, Amarex Clinical Research LLC, to seek FDA "fast-track" and possibly even FDA "breakthrough" designations and to obtain IND authorizations to conduct a follow-up pancreatic cancer Phase 2/3 clinical trial with sites in the Netherlands at Erasmus MC under Prof. van Eijck, and also at major cancer research centers in the United States. AIM also plans to file dual orphan drug status applications for use of Ampligen in the treatment of late-stage pancreatic carcinoma with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). If granted, this serves to extend a company’s exclusivity rights once a drug reaches market.

On September 22, 2020 The Upstate Foundation reported that it has awarded grants to two Upstate Medical University researchers to support their work on pediatric cancer (Press release, SUNY Upstate, SEP 22, 2020, View Source [SID1234565486]).

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Following a competitive grant application and evaluation process overseen by Upstate’s Pediatric Research Advisory Committee, chaired by Leonard Weiner, MD, vice chair of academic affairs, the Foundation is providing funding of $25,000 each for projects proposed by Jeffrey Amack, PhD, associate professor of Cell and Developmental Biology, and Jason Horton, PhD,assistant professor of Orthopedic Surgery.

The grants, made possible through an anonymous estate gift to the Upstate Foundation, honor September as Pediatric Cancer Awarness Month.

"We are pleased to help fund these projects," said Eileen Pezzi, vice president for development at Upstate. "This generous and kind donor had an abiding interest in supporting both pediatric and adult oncology research. We’re proud to fulfill her wish and honor her memory in this way."

Amack’s research project, titled Regulation of cell proliferation by V-ATPase activity, seeks to better understand the role that the proton (H+) pump V-ATPase plays in controlling cell proliferation in multiple tumor types, including in several pediatric cancers.

Horton’s research initiative, entitled Treatment of tumor-associated osteolysis in Ewing sarcoma, will seek to determine if the antibiotic Mithramycin A (MithA) can mitigate tumor-associated osteolysis typical of the highly metastatic pediatric bone cancer, Ewing sarcoma.

"The funded projects were selected because they were exceedingly well-considered, well-structured, and quite feasible in terms of the results they seek to achieve," said Weiner. "Additionally, the advisory committee has had the pleasure of seeing Dr. Amack devise, manage, and execute a previous research project. These two new efforts fall squarely into each researcher’s respective areas of expertise and experience."

Those interested in learning more about these research projects are encouraged to contact Amack at [email protected] and Horton at [email protected]

Caption: From left: Jeffrey Amack, PhD, associate professor of cell and developmental biology, and Jason Horton, PhD, assistant professor of orthopedic surgery, have been awarded grants by the Upstate Foundation to further their work in pediatric cancer research.

On September 22, 2020 Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") reported the public release of the results of its’ multicenter, open-label, Phase 1b Study to Evaluate Safety and MTD of Epidural Resiniferatoxin Injection for the Treatment of Intractable Cancer Pain, at the 14th Annual Pain Therapeutics Summit held virtually from September 21 to 22, 2020 (Press release, Sorrento Therapeutics, SEP 22, 2020, View Source [SID1234565485]). Data was presented by Srdjan Nedeljkovic, MD, Associate Professor of Anesthesia, Harvard Medical School/Brigham & Women’s Hospital.

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"We are extremely encouraged by the results of this initial study. Even in patients with high levels of pain, RTX given via an epidural injection has been found to reduce pain intensity without having any long-term adverse safety consequences," said Associate Professor of Anesthesia, Srdjan S. Nedeljkovic, M.D. from the Department of Anesthesiology, Perioperative and Pain Medicine, Brigham and Women’s Hospital at Harvard Medical School. "The patient population had intractable pain that did not respond to other standard therapeutic approaches, including opioids. The addition of RTX to the management of patients with intractable advanced-stage cancer pain offers the prospect of reducing suffering and improving quality of life for this underserved patient population".

This multicenter, open-label study enrolled 17 adults with intractable moderate to severe cancer pain. Subjects were treated with a one-time epidural administration of RTX at escalating dose level cohorts, ranging from 0.4 µg to 25 µg in 3 ml saline, in seven cohorts. The first participant in each cohort served as the "Sentinel" subject. The first two dosing cohorts (0.4 µg and 1.0 µg) each included one subject. Subsequent cohorts proceeded with three subjects each (2, 4, 8, 15 and 25 µg).

Enrollment of dose escalation cohorts has completed, with 17 subjects receiving RTX. 65% were women and 35% were men. The median age was 58 years (range 28-82 years). The baseline numerical pain rating scale (NPRS) average score was a mean of 6.8 (standard deviation (S.D.) of 1.65), and the baseline NPRS worst score was a mean of 7.9 (S.D. of 1.26).

No dose-limiting toxicities were reported. Dose escalation was completed at 25 ug. The most frequently reported treatment-emergent adverse event was transient post-procedural pain that was described in 47.1% of subjects. Post-injection-associated pain was managed with traditional short-term pain medications on the day of RTX injection. Typically, the RTX-associated pain following injection subsided before the 8-hour post-injection assessment and resolved within 24 hours in all subjects. Transient and reversible adverse events reported in at least two RTX-treated subjects were nausea (17.6%), vomiting (17.6%), and headache (17.6%). A total of 15 serious adverse events (SAEs) were reported, but none were deemed by the investigator to be related to RTX treatment. Most adverse events were attributed to the underlying cancer diagnosis.

Clinical efficacy (CE) was assessed at three efficacy levels: CE30, CE50 and CE70, defined as a 30%, 50% and 70% decrease in pain, respectively, for three consecutive days from the original baseline NPRS score of ≥ 6/10.

A positive outcome was observed in the lowest dose of RTX administered (0.4 ug) at the CE30 efficacy point. A dose-response relationship was observed, with the majority of responders at the 15 ug and 25 ug dose levels. Of the 17 subjects, 11 achieved the CE30 prespecified efficacy end-point using NPRS scores. Day 90 results for all RTX doses pooled are shown below:

Percentage decrease in Pain Average Pain Worst Pain
> 30% reduction from Baseline 64.7 % 47.1 %
> 50% reduction from Baseline 35.3 % 29.4 %
> 70% reduction from Baseline 23.5 % 17.6 %
PK data revealed no detectable drug in plasma in 15 of the 17 subjects. Minimally detectable levels of RTX were seen in 2 of the 17 subjects, in each case only at the initial post-injection time point.

RTX administration was well-tolerated when given as a one-time epidural injection at doses up to 25 ug. Preliminary clinical pain improvement was observed in the dose-escalation phase. Based on the results, though the protocol allowed exploration of a 35 mcg dose for this indication, a dose beyond 25 mcg was not deemed necessary to qualify the safety and clinically meaningful efficacy of the drug. These preliminary data support further study of epidural RTX in a broader patient population with what would be considered moderate to severe pain associated with cancer in this orphan indication.

For access to the poster associated with the scientific presentation, please visit Sorrento Investor Relations Site

Sorrento intends to rapidly advance to larger scale trials and expects to submit a request to proceed with a multicenter, blinded, controlled Phase 3 trial to the FDA in the upcoming weeks.

About Resiniferatoxin (RTX)

A thousand times "hotter" than pure capsaicin (16 Billion Scoville units versus 16M), and with a high affinity for afferent pain nerves, resiniferatoxin binds to TRPV1 receptors and selectively ablates the nerve endings responsible for pain signals experienced by patients1. Delivered peripherally (into the joint space) the transient nerve ending ablation effect can have profound clinical benefits lasting for months to years (as shown in canine studies2).

RTX-001 was a multicenter, open-label dose escalation Phase 1b study to assess the safety and define the maximally tolerated dose of resiniferatoxin administered via the epidural route for the reduction of moderate to severe pain signal intensity associated with advanced cancer. The Phase 1b study was a dose-escalation protocol in which cohorts of patients received increasing doses of resiniferatoxin until the maximum tolerated dose was achieved. The primary objective of the study was to evaluate the safety of resiniferatoxin and identify the recommended Phase 3 dose. The secondary objective was to assess the preliminary efficacy of resiniferatoxin measured by assessing changes in the intensity of pain using the NPRS score, a widely used proprietary validated pain scale.

RTX is not approved for clinical use by regulatory authorities. Safety and efficacy have not been established.