On November 17, 2020 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Phase 1 MELANI-01 trial evaluating the UCARTCS1 product candidate for the treatment of patients with relapsed or refractory multiple myeloma (MM) (Press release, Cellectis, NOV 17, 2020, View Source [SID1234571264]).

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Cellectis worked closely with the FDA over the past months, to address the agency’s requests, which include adjustments to the MELANI-01 clinical protocol designed to enhance patient safety. Cellectis continues to work with the clinical site staff and investigators to efficiently obtain the required local approvals to reopen the trial and resume patient enrollment.

"We remain confident in the potential clinical benefit of UCARTCS1 product candidate for patients with relapsed/refractory multiple myeloma, a widely unmet medical need that Cellectis will continue to address. The safety of patients enrolled in our clinical trials remains our priority, and we are committed to resuming the clinical development of this promising program," said Carrie Brownstein, MD, Chief Medical Officer, Cellectis.

Patient enrollment is ongoing in Cellectis’ two other proprietary Phase 1 dose escalation trials: AMELI-01 evaluating UCART123 in relapsed and refractory acute myeloid leukemia and BALLI-01 evaluating UCART22 in relapsed and refractory B-cell acute lymphoblastic leukemia.

About MELANI-01
MELANI-01 is a Phase 1 open-label First-In-Human dose escalation clinical study evaluating UCARTCS1 product candidate for the treatment of patients with relapsed or refractory multiple myeloma (MM). UCARTCS1 is an allogeneic, off-the-shelf, gene-edited T-cell product candidate designed for the treatment of CS1/SLAMF7-expressing hematologic malignancies. CS1 (SLAMF7) is highly expressed on MM tumor cells. Learn more about the ongoing clinical trials at www.clinicaltrials.gov

About Multiple Myeloma (MM)

Multiple myeloma is a cancer that affects a type of white blood cells called plasma cells that are specialized mature B-cells, which secrete antibodies to combat infections. Multiple myeloma is characterized by the uncontrolled proliferation of neoplastic plasma cells in the bone marrow, where they overcrowd healthy blood cells. Although MM is a chronic disease and an exact cause has not yet been identified, researchers have made significant progress over the years in managing the disease through better understanding MM’s pathophysiology. The progress in finding a cure needs to be continued as The American Cancer Society estimates that 32,270 new cases of MM will be diagnosed, and 12,830 deaths are expected to occur in 2020 in the U.S. alone.

On November 17, 2020 Istari Oncology reported that UH Seidman Cancer Center became one of just six sites participating in a Phase II clinical trial to test the safety and efficacy of oncolytic polio/rhinovirus recombinant (PVSRIPO), a modified polio vaccine-based viral immunotherapeutic, in patients with recurrent malignant glioblastoma (rGBM) (NCT02986178) (Press release, Istari Oncology, NOV 17, 2020, View Source [SID1234571263]).

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The first-in-man Phase I trial, conducted at the Preston Robert Tisch Brain Tumor Center at Duke University Medical Center, showed encouraging results. The study found that survival rates were significantly higher in rGBM patients who received an intratumoral infusion of PVSRIPO immunotherapy compared to similar patients receiving standard treatment at the same institution. Overall survival among patients who received PVSRIPO plateaued at 21 percent at 24 to 36 months and appeared to last for up to five years based on a publication of the interim trial results (Desjardins, et al., 2018 NEJM). The overall survival rate was sustained at 36 months in these patients.

"We’ve never seen survival rates like this before," says Andrew E. Sloan, MD, FACS, Director of the Brain Tumor & Neuro-Oncology Center and the Center of Excellence in Translational Neuro-Oncology at UH Seidman Cancer Center and UH Neurological Institute, and Professor and Vice Chairman, Department of Neurosurgery at Case Western Reserve University School of Medicine, who serves as principal investigator for the trial. "The long-term survival rate is very exciting. We are proud that University Hospitals was selected as one of a handful of top brain tumor centers, including the Stephen E. and Catherine Pappas Center for Neuro-Oncology at Massachusetts General Hospital and UCSF Brain Tumor Center, to participate in the Phase II clinical trial based on our expertise in immunotherapy and reputation for treating brain tumors."

To date, Dr. Sloan has treated seven patients with rGBM at UH Seidman Cancer Center according to the trial protocol, all of whom are surviving. Building on this experience, he is now seeking to enroll patients with rGBM into a new Phase I/II trial of PVSRIPO, but this time with a difference. This trial combines PVSRIPO with the immune checkpoint inhibitor pembrolizumab (Keytruda). The hypothesis is that the combination of the two therapies will be able to generate a potent and specific anti-tumor response in rGBM patients, given their different but complimentary mechanisms of action. This new trial is now open at UH Cleveland Medical Center. When it adapts to a Phase II trial in the coming weeks, it will be offered at approximately six total sites nationwide.

"The tumor we’re targeting is one of the most aggressive tumors known to man," Dr. Sloan says. "The virus can counteract some of that, but we want to test whether we can perhaps give it a little boost. PVSRIPO and pembrolizumab together may be more effective than either on its own."

That is, in fact, the working theory. According to Istari Oncology, Inc., which is sponsoring the new trial, "pre-clinical data and limited clinical use suggest that PVSRIPO followed by pembrolizumab may be associated with synergistic anti-tumor responses."

As in the first trials, patients in the new trial will receive PVSRIPO via a catheter into the tumor, a process known as convection enhanced delivery (CED). The infusion will be performed in the NeuroIntensive Care Unit at UH Cleveland Medical Center. The PVSRIPO infusion will be followed by intravenous pembrolizumab at approximately 14 days post-procedure, and then every three weeks thereafter, for up to 24 months, provided no toxicity or disease progression.

Dr. Sloan says he’s hopeful that the combination of PVSRIPO and pembrolizumab will prove successful for his patients with rGBM. In fact, he says, approaches that seem to hold the most promise for this intractable cancer are those that seek to increase expression of tumor-specific neo-antigens, coupled with checkpoint inhibition. This clinical trial fits that bill.

"That’s likely what’s necessary to overcome the profound immune blockade associated with this disease," he says.

On November 17, 2020 Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company utilizing a proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics, with a focus on oncology and infectious diseases including COVID-19, reported third quarter 2020 financial results and recent highlights (Press release, Immunome, NOV 17, 2020, View Source [SID1234571261]).

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"The completion of our recent IPO marks a significant milestone for Immunome and positions us to further accelerate the output of new discoveries from our platform, and to rapidly advance our lead oncology and COVID-19 therapeutic antibody programs into the clinic," said Purnanand Sarma, PhD, President and CEO of Immunome. "We are on track to execute on our near-term clinical and strategic plans, including filing IND applications for two of our programs in 2021." Dr. Sarma further stated, "I am proud of the tremendous progress our team has made this year, particularly with regard to the notable effort and dedication supporting our accelerated development of antibody-based treatment targeting multiple viral antigens to treat the COVID-19 virus. As previously announced, Immunome was awarded a $13.3 million contract from the US Department of Defense to develop an antibody cocktail discovered by the interrogation of memory B cells from ‘super-responders’ to the SARS-CoV-2 virus (DoD Press Release)."

Recent Highlights

In October 2020, Immunome completed an IPO, including the full exercise of the underwriters’ option to purchase additional shares, resulting in gross proceeds of $44.9 million, before deducting underwriting commissions and offering expenses.
IMM-BCP-01: Unlike approaches solely directed at neutralizing the Spike protein by other companies, we aim to develop an antibody cocktail directed at multiple SARS-CoV-2 antigens. Unbiased interrogation of SARS-CoV-2 "super-responder" memory B cells using Immunome’s discovery engine has resulted in the following advances:
We discovered that more than 50% of the antibodies isolated from super-responders are directed at non-Spike antigens, suggesting non-Spike related antibodies may play a significant role in the effective immunological clearance of this virus.
We also identified multiple neutralizing antibodies with picomolar affinity directed at SARS-CoV-2 Spike protein.
We discovered that, in addition to affinity matured IgG antibodies, COVID-19 "super-responders" appear to mount robust affinity-matured IgA responses. IgA antibodies are naturally found at the surface of respiratory track and function to prevent initial viral infection.
IMM-ONC-01: Our further pre-clinical testing continues to demonstrate that IL-38 appears to be a novel immune checkpoint. In animal models, our inhibitory antibody demonstrates antitumor effects in IL-38 expressing tumors.
Third Quarter 2020 Financial Results:

Cash and cash equivalents: Cash and cash equivalents were $6.7 million as of September 30, 2020 compared to $2.5 million as of December 31, 2019.
IPO and net proceeds: On October 2, 2020 the Company’s common stock began trading on the Nasdaq Capital Market under the ticker (IMNM) and on October 6, 2020 the IPO closed with the Company receiving net proceeds of $41.7 million, after deducting underwriting discounts but before deducting other offering expenses. After deducting other offering costs of approximately $2.8 million, aggregate net IPO proceeds to the Company were $38.9 million.
Research and development (R&D) expenses: R&D expenses were $1.6 million for the quarter ended September 30, 2020 compared to $2.2 million for the quarter ended September 30, 2019 and $5.7 million for the nine month period ended September 30, 2020 compared to $6.4 million for the nine month period ended September 30, 2019.
General and administrative (G&A) expenses: G&A expenses were $1.2 million for the quarter ended September 30, 2020 compared to $0.5 million for the quarter ended September 30, 2019 and $2.5 million for the nine month period ended September 30, 2020 compared to $1.1 million for the nine month period ended September 30, 2019.
Net loss: Net loss attributable to common stockholders was $8.4 million or $7.52 per share for the quarter ended September 30, 2020 compared to $2.7 million or $2.50 per share for the quarter ended September 30, 2019 and $13.8 million or $12.44 per share for the nine month period ended September 30, 2020 compared to $7.5 million or $6.88 per share for the nine month period ended September 30, 2019.

On November 17, 2020 Five Prime Therapeutics, Inc. (Nasdaq: FPRX) reported the closing of its upsized underwritten public offering of 8,280,000 shares of its common stock, which includes 1,080,000 shares sold upon the underwriters’ full exercise of their option to purchase additional shares, resulting in aggregate gross proceeds of approximately $173.9 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Five Prime (Press release, Five Prime Therapeutics, NOV 17, 2020, View Source [SID1234571259]).

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Cowen and SVB Leerink acted as joint book-running managers for the offering. Wedbush PacGrow acted as co-manager for the offering.

The shares of common stock were offering pursuant to a "shelf" registration statement previously filed with and declared effective by the Securities and Exchange Commission (SEC). The offering is being made only by means of a prospectus supplement and accompanying prospectus, copies of which may be obtained from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at (833) 297-2926 or by email at [email protected], or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA, 02110, by telephone at (800) 808-7525, ext. 6132 or by e-mail at [email protected].

On November 17, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, reported it will participate in fireside chats at two investor conferences in December 2020 (Press release, Aeglea BioTherapeutics, NOV 17, 2020, View Source [SID1234571256]).

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Conference Details

Conference Name: Piper Sandler 32nd Annual Virtual Healthcare Conference
Conference Date: November 30 – December 3, 2020
Fireside Chat Date/Time: Available beginning November 23, 2020
Presenter: Anthony G. Quinn, M.B. Ch.B., Ph.D., Aeglea’s president and chief executive officer

Conference Name: Evercore ISI 3rd Annual HealthCONx Conference
Conference Date: December 1-3, 2020
Fireside Chat Date/Time: December 1, 2020 at 4:20 p.m. EST
Presenter: Anthony G. Quinn, M.B. Ch.B., Ph.D., Aeglea’s president and chief executive officer

To access the live and archived webcasts, visit the Presentations & Events section of the Company’s website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary. An archived version of the webcasts will also be available through the Company’s website for a limited time following the conferences.