On Target Laboratories Announces U.S. FDA Acceptance and Priority Review of New Drug Application for pafolacianine sodium injection for Identification of Ovarian Cancer During Surgery

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.

Cerus Corporation to Participate in Jefferies Virtual Global Plasma Summit

On March 3, 2021 Cerus Corporation (Nasdaq: CERS) reported that William ‘Obi’ Greenman, Cerus’ president and chief executive officer, and Kevin D. Green, Cerus’ chief financial officer, are scheduled to participate in the Jefferies Virtual Global Plasma Summit on Thursday, March 11, 2021 at 11:00 am PT/ 2:00 pm ET (Press release, Cerus, MAR 3, 2021, View Source [SID1234575984]).

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A live webcast of the events will be available on the Investor Relations page of the Cerus web site at View Source A replay will be available for approximately two weeks following the completion of the event.

Avid Bioservices Declares Quarterly Dividend on Its Series E Convertible Preferred Stock

On March 3, 2021 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that its Board of Directors has declared a quarterly cash dividend payment on the Company’s 10.50% Series E Convertible Preferred Stock (the "Series E Preferred Stock") (Press release, Avid Bioservices, MAR 3, 2021, View Source [SID1234576005]).

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The quarterly dividend on the Series E Preferred Stock is payable on April 1, 2021 to holders of record at the close of business on March 15, 2021.

The quarterly dividend payment on the Series E Preferred Stock will be $0.65625 per share, which is equivalent to an annualized 10.50% per share, based on the $25.00 per share stated liquidation preference, accruing from January 1, 2021 through March 31, 2021. The Series E Preferred Stock is listed on the NASDAQ Capital Market and trades under the ticker symbol "CDMOP".

SQZ Biotech to Present at H.C. Wainwright Global Life Sciences Conference

On March 3, 2021 SQZ Biotechnologies (NYSE:SQZ), a cell therapy company developing novel treatments for multiple therapeutic areas, reported that Armon Sharei, PhD, chief executive officer of SQZ, will present a corporate overview at the upcoming virtual H.C. Wainwright Global Life Sciences Conference (Press release, SQZ Biotech, MAR 3, 2021, View Source [SID1234576023]). The presentation will be available through the virtual conference portal on Tuesday, March 9, 2021 at 7:00 am EST. The team is also taking individual meetings at the event.

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Registration for the webcast can be found here. Replays of the webcast will be available on the Investors & Media section of the SQZ website for 90 days following the event.

Blueprint Medicines Announces EMA Validation of Type II Variation Marketing Authorization Application for AYVAKYT® (avapritinib) for the Treatment of Advanced Systemic Mastocytosis

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.