On March 3, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that the European Medicines Agency (EMA) has validated the company’s Type II variation marketing authorization application for AYVAKYT (avapritinib) for the treatment of advanced systemic mastocytosis (SM) (Press release, Blueprint Medicines, MAR 3, 2021, View Source [SID1234576041]). Validation of the application confirms that the submission is sufficiently complete to begin the formal review process. The European Commission has granted orphan medicinal product designation to AYVAKYT for the treatment of mastocytosis.

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"AYVAKYT represents a promising approach to address the needs of patients with advanced systemic mastocytosis, a rare, life-threatening disease characterized by severe organ damage," said Paul Beresford, Senior Vice President and General Manager, International, at Blueprint Medicines. "In robust datasets across our registrational trials, AYVAKYT has shown complete and durable remissions not typically observed in this patient population, and a generally well-tolerated safety profile. We look forward to working closely with the EMA during the review process, and aim to bring the first precision therapy to patients in Europe that targets the primary driver of the disease."

AYVAKYT, a potent and selective inhibitor of D816V mutant KIT, is being developed to treat advanced and non-advanced forms of SM. Last month, Blueprint Medicines announced that the U.S. Food and Drug Administration (FDA) accepted the company’s supplemental new drug application and granted priority review for AYVAKIT (avapritinib) for the treatment of advanced SM.

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 aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival.

Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Currently, there are no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.

About AYVAKYT (avapritinib)

AYVAKYT (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation. This medicine was approved by the FDA for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, under the brand name AYVAKIT.

AYVAKIT/AYVAKYT is not approved for the treatment of any other indication, including SM, in the U.S. by the FDA or in Europe by the European Commission, 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 aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia, and for the treatment of moderate to severe indolent SM.

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.

On March 3, 2021 On Target Laboratories, Inc., a privately-held biotechnology company developing fluorescent markers to target and illuminate cancer during surgery, reported that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for Priority review for pafolacianine sodium injection as an adjunct for identifying ovarian cancer during surgery (Press release, On Target Laboratories, MAR 3, 2021, View Source [SID1234576040]). Many cancers, including over 95%1 of ovarian cancer, over-express folate receptors to enable the uptake of folic acid. Pafolacianine sodium injection is a novel molecule which is shown to bind to folate receptors and illuminate intraoperatively under near-infrared light, serving as an adjunct to provide greater certainty in a complete resection. It is administered via standard IV in as little as one hour before surgery.

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The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis or prevention of a serious condition. This designation shortens the review period from the standard 10 months to six months from the acceptance of the NDA. Pafolacianine sodium injection was investigated for use in ovarian cancer under Special Protocol Agreement and received both Fast Track and Orphan designations from the FDA. Additionally, pafolacianine sodium injection is being investigated in lung cancer under Fast Track designation.

Ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. The American Cancer Society estimates that in 2021 about 21,410 women in the United States will receive a new diagnosis of ovarian cancer and about 13,770 women will die from ovarian cancer2.

"Our NDA is supported by our positive Phase 2 and Phase 3 clinical trials. As we move closer to FDA approval for ovarian cancer, we are realizing our mission to intra-operatively visualize more cancer and extend the benefits of a complete resection to patients," said Christopher Barys, President and CEO of On Target Laboratories.

About Fluorescence-guided Surgery

To date, there have been limited ways for surgeons to confidently assess the location and full extent of cancerous tissue while operating. On Target Laboratories’ fluorescent markers are comprised of a near-infrared dye and a targeting molecule, or ligand, that binds to receptors overexpressed on cancer cells. These markers illuminate the cancerous lesions, enabling surgeons to see and remove more cancerous tissue that otherwise may have gone undetected.

On Target’s first novel compound, pafolacianine sodium injection, targets folate receptors commonly found on many cancers, including lung and ovarian cancers. A single dose of the compound is administered via intravenous infusion prior to surgery to help the surgeon identify additional malignant tissue during the operation using a near-infrared camera.

About Pafolacianine Sodium Ovarian Clinical Results

Clinical trials for the use of pafolacianine sodium as an intraoperative adjunct to aid in the surgical treatment of ovarian cancer have been conducted in the United States and Europe. Phase 2 results were previously published in the journal Gynecologic Oncology, which reported surgeons were able to detect additional lesions, regardless of tissue location, in 48.3% of patients. Further, pafolacianine sodium injection demonstrated a sensitivity of 97% when controlling for detection correlation of multiple lesions within a single patient3.

On March 3, 2021 Linnaeus Therapeutics, Inc. (Linnaeus), a privately held clinical-stage biopharmaceutical company focused on the development and commercialization of novel small molecule oncology therapeutics, reported that on March 2, 2021 the U.S. Patent and Trademark Office (USPTO) issued U.S. patent 10,934,277 (‘277 patent) covering various embodiments of the pharmaceutical composition of matter for the company’s lead compound, LNS8801 (Press release, Linnaeus Therapeutics, MAR 3, 2021, View Source [SID1234576039]).

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Linnaeus’s patent disclosure is directed toward the pharmaceutical composition of matter of LNS8801 and generally embodies (1) an enantiomerically purified compound SRR G-1, or a derivative thereof, including specific crystal forms, salts, and co-crystals, that modulates G protein-coupled estrogen receptor activity; (2) pharmaceutical and cosmetic compositions comprising an enantiomerically purified SRR G-1, or a derivative thereof; and (3) methods of treating or preventing disease states and conditions and cosmetic conditions mediated through these receptors and related methods thereof in humans and animals.

"We are extremely pleased that the USPTO has issued this first patent under its expedited review format," commented Patrick Mooney, MD, CEO of Linnaeus. "We believe that the issued claims will provide critical market protection for LNS8801 through at least 2038. As we continue to collect very promising data from our clinical trials of LNS8001, we plan to prosecute the ‘277 patent, both on a worldwide basis and an on-going effort in the U.S."

Linnaeus is testing LNS8801 in its phase 1/2 adaptive-design clinical trial as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients who had previous clinical benefit from immune checkpoint inhibitors and then subsequently progressed. This marks the first time any company has dosed a patient in a clinical trial specifically targeting the G protein-coupled estrogen receptor (GPER) in combination with pembrolizumab.

Linnaeus is currently finishing the dose-escalation portion of its phase 1/2 study assessing the safety, tolerability, pharmacokinetics, and preliminary efficacy in patients with advanced cancer, and the company has already identified the phase 2 dose for LNS8801 as a monotherapy and in combination with pembrolizumab.

About LNS8801
LNS8801 is an orally bioavailable and highly specific and potent agonist of GPER whose activity is dependent on the expression of GPER. GPER activation by LNS8801 rapidly and durably depletes c-Myc protein levels. In preclinical cancer models, LNS8801 displays potent antitumor activities across a wide range of tumor types, rapidly shrinking tumors and inducing immune memory.

In the ongoing phase 1/2 study in humans, LNS8801 monotherapy has been safe and well tolerated. Additionally, LNS8801 has demonstrated target engagement, c-Myc protein depletion, and clinical benefit in patients with advanced cancer. Data from the phase 1/2 study are anticipated to be presented in a peer-reviewed setting in 2021.

On March 3, 2021 Ampio Pharmaceuticals, Inc. (NYSE American: AMPE), a biopharmaceutical company focused on the advancement of immunology-based therapies for prevalent inflammatory conditions, reported that management will present at the virtual H.C. Wainwright Global Life Sciences Conference scheduled Tuesday, March 9, 2021 through Wednesday, March 10, 2021 (Press release, Ampio, MAR 3, 2021, View Source [SID1234576038]).

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The video webcast presentation will be available for viewing on-demand beginning Tuesday, March 9, 2021 at 7:00 am (EST) for those registered for the event and will be accessible on the Presentations & Media page in the Investors section of the Company’s website (View Source). The webcast replay will be archived for 90 days following the event.

On March 3, 2021 Geneos Therapeutics, a clinical stage company focused on the development of tumor neoantigen targeted personalized immunotherapies for cancer, reported that it has closed its Series A1 round, raising $12 million in financing (Press release, Geneos Therapeutics, MAR 3, 2021, View Source [SID1234576037]). The financing was led by Korea Investment Partners (KIP) – Global Bio Fund with strong participation from all existing Series A investors including, notably, Santé Ventures and Inovio Pharmaceuticals, Inc. (NASDAQ: INO). The new investment follows the previously announced initial financing of $10.5 million in February 2019. In conjunction with the financing, Mr. Sangwoo Lee, Managing Director Korea Investment Partners – USA Inc. joined Geneos’ Board of Directors.

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"Geneos has made great strides in advancing the novel and differentiated GT-EPIC platform for personalized cancer treatments," commented Mr. Lee. "KIP is pleased to join the company’s investment syndicate and help support the team with its development plans."

The proceeds will be used for the expansion of the company’s GT-30 Phase Ib/IIa clinical trial evaluating its personalized neoantigen-targeting vaccine, GNOS-PV02, for treating patients with advanced hepatocellular carcinoma (HCC), a type of liver cancer. GNOS-PV02, which is based on Geneos’ proprietary GT-EPIC platform, is a tumor-specific DNA plasmid product designed and manufactured for each patient based on their unique tumor mutations’ (neoantigens), identified by sequencing each patient’s tumor. In the trial, GNOS-PV02 is combined with a DNA plasmid encoded cytokine immunomodulator IL-12 (INO-9012) and PD-1 checkpoint inhibitor (pembrolizumab). Geneos has amended the trial protocol to expand enrollment from the initial 12 patients to 24 patients.

"The Series A1 financing demonstrates our investors’ confidence in the GT-EPIC platform to design and manufacture patient-specific personalized cancer vaccines," said Dr. Niranjan Y. Sardesai, Founder and Chief Executive Officer of Geneos Therapeutics. "Over the past two years we have demonstrated feasibility of the approach as a treatment modality – notably, the rapid biopsy to treatment turnaround time, which is so crucial when treating advanced cancer patients. This financing will allow us to expand our GT-30 clinical trial to a larger number of patients which we estimate will be sufficient to demonstrate efficacy in the 2nd line setting."

The company also announced the expansion of its management team with the addition of leadership team members who collectively bring decades of biopharmaceutical development expertise across early and late-stage clinical programs and pharma/biotech partnering. Joining the company’s leadership team are:

Ms. Joann Peters as Vice President, Clinical and Business Operations
Dr. Alfredo Perales-Puchalt, MD, Ph.D as Vice President, Research & Development
Dr. Hakim Hammach, Ph.D, MBA as Vice President, Business Development
Ms. Federica O’Brien as Strategic Finance Consultant/CFO