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Sapience Therapeutics Receives FDA Fast Track Designation for ST101 for the Treatment of Recurrent Glioblastoma Multiforme (GBM)

On December 6, 2021 Sapience Therapeutics, Inc., a biotechnology company focused on the discovery and development of peptide therapeutics to address difficult-to-treat cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead program investigating ST101 for the treatment of recurrent glioblastoma (GBM) (Press release, Sapience Therapeutics, DEC 6, 2021, View Source [SID1234596504]). ST101 is currently being evaluated in an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors, which includes a GBM expansion cohort.

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In the ongoing Phase 1-2 study, ST101 demonstrated clinical proof-of-concept with a RECIST 1.1-confirmed partial response (PR) in a patient with cutaneous melanoma and evidence of long-lasting stable disease in several additional patients with refractory solid tumors.

"Glioblastoma is the most aggressive, fatal form of brain cancer with limited-to-no therapeutic options. With the efficacy data from solid tumors and its ability to cross the blood-brain barrier, ST101 could meaningfully improve outcomes for patients battling this debilitating disease and we look forward to advancing it into Phase 2," said Dr. Barry Kappel, Sapience’s CEO and President.

Dr. Gina Capiaux, PhD, Sapience’s Head of Regulatory Affairs added, "This is an exciting regulatory milestone for our program. Fast Track designation for ST101 highlights this unmet need and allows us to work closely with the FDA to deliver this novel therapy to people with GBM as soon as possible."

Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions that demonstrate the potential to address unmet medical needs. Sapience previously received Orphan Drug Designation from the FDA and European Commission for ST101 for the treatment of glioma.

About Glioblastoma
Glioblastomas are a class of malignant glioma and represent the most common and serious type of primary brain tumors. GBMs account for approximately 14% of all primary brain and Central Nervous System (CNS) tumors and 50% of all malignant brain tumors. Despite standard treatments including surgery, chemotherapy, and radiation, GBM has a rapid progression and high mortality with a median survival less than 8 months and a five-year survival rate of 6.6%.

About ST101
ST101, a peptide antagonist of C/EBPβ, is currently being evaluated in an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors (NCT04478279). In the ongoing study, ST101 has demonstrated clinical proof-of-concept with a RECIST 1.1-confirmed partial response (PR) in a patient with cutaneous melanoma and evidence of long-lasting stable disease in several additional patients. Following conclusion of the final dose-escalation cohort, Sapience plans to initiate four Phase 2 expansion cohorts in refractory, locally advanced and metastatic cutaneous melanoma, hormone-receptor-positive breast cancer, castrate-resistant prostate cancer, and glioblastoma starting in the second half of 2021. ST101 has been granted orphan drug product designation from the U.S. Food and Drug Administration and orphan medicinal product designation by the European Commission for the treatment of glioma.

Purple Biotech Announces Initiation of Part 2 of Ongoing Phase 1/2 Clinical Trial Evaluating NT219 for Treatment of Multiple Cancers Supporting Potential Expansion of Clinical Program

On December 6, 2021 Purple Biotech (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective and durable therapies by overcoming tumor immune evasion and drug resistance, reported the initiation of Part 2 of its ongoing Phase 1/2 clinical trial of NT219, a novel small molecule inhibiting simultaneously IRS1/2 and STAT3, for the treatment of multiple cancers (Press release, Purple Biotech, DEC 6, 2021, View Source [SID1234596503]). Part 2 is a dose escalation study of NT219, beginning with 6mg/kg, in combination with the standard dose of cetuximab (ERBITUX), in patients with recurrent or metastatic squamous cell carcinoma of head and neck cancer (SCCHN) or colorectal adenocarcinoma.

The monotherapy portion of the study, Part 1, is progressing as planned, and is now dosing patients with advanced solid tumors in the 24mg/kg cohort. To date, no drug limiting toxicities have been observed in the trial, and NT219 has been found to be well-tolerated with minimal serious adverse events.

Initial efficacy data previously reported from the first dose level in Part 1, and additional efficacy signals in the following two cohorts, support the immediate extension of the study to Part 2 of the study, dosing NT219 at its recommended Phase 2 dose in combination with cetuximab in patients with recurrent or metastatic SCCHN, as well as the potential expansion of the clinical program to new indications, which is anticipated to commence during the second half of 2022.

Additional preliminary efficacy data from Part 1 of the trial is expected to be presented at a medical meeting in the first half of 2022.

"The advancement of our ongoing Phase 1/2 clinical trial to Part 2 represents an important achievement for our NT-219 clinical development program," said Michael Schickler, Ph.D., Head of Clinical and Regulatory Affairs of Purple Biotech. "We are encouraged by the initial signals of efficacy, as well as safety trends, observed in Part 1 of the study to date. We look forward to the availability of additional preliminary efficacy data from Part 1 of the study in the first half of 2022, as well as to the further advancement of our development program to Part 2 of the study, and to the potential expansion of the clinical program into other unmet cancer indications, in the second half of 2022."

Bellicum Announces Positive Interim Data from Phase 1/2 GoCAR-T® Studies, Secures $35 Million Private Placement Equity Financing Priced at Market

On December 6, 2021 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported positive interim data from its ongoing Phase 1/2 GoCAR-T clinical trials, including a confirmed partial response (PR) in the first cohort of mCRPC patients treated in the clinical trial for BPX-601 (Press release, Bellicum Pharmaceuticals, DEC 6, 2021, View Source [SID1234596502]). Bellicum has also entered into an agreement for a $35 million private placement of equity securities with two biotechnology specialist investment funds. Proceeds from the financing will be used to support ongoing clinical development of BPX-601 and BPX-603.

"We are highly encouraged by the observation of anti-tumor activity of BPX-601 in the first mCRPC cohort, particularly in this heavily pre-treated patient population with limited therapeutic options," said Rick Fair, President and Chief Executive Officer of Bellicum. "The additional capital we raised through this private placement will support our ongoing clinical development of BPX-601 and BPX-603 to further pursue this early efficacy signal."

Clinical Trial Updates

BPX-601 GoCAR-T in Metastatic Castration-Resistant Prostate Cancer
Cell dose escalation and optimization of the lymphodepletion regimen was completed in the Phase 1/2 clinical trial of BPX-601 in metastatic pancreatic cancer patients. Earlier this year, enrollment was initiated in previously-treated mCRPC patients at the previously-cleared dose level.

In the first three-patient cohort of mCRPC treated at 5×106 cells/kg BPX-601 followed by single-dose rimiducid:

No dose-limiting toxicities were observed. Adverse events were comparable to those previously seen with BPX-601 and rimiducid in metastatic pancreatic cancer and typical of other cell therapy regimens.
A confirmed partial response was observed in one patient by RECIST v1.1 criteria on CT scan, accompanied by substantial reduction in PSA in response to treatment with BPX-601 and rimiducid.
Enrollment is ongoing in the second dose cohort in mCRPC of 5×106 cells/kg followed by weekly rimiducid, and results from these patients will be submitted for presentation at a medical meeting in 2022.

BPX-603 Dual-Switch GoCAR-T in HER2+ Solid Tumors
Enrollment is ongoing in the Phase 1/2 clinical trial for BPX-603 in patients with solid tumors that express human epidermal growth factor 2 (HER2), including breast, endometrial, ovarian, gastric, and colorectal cancers. BPX-603 is Bellicum’s first dual-switch GoCAR-T product candidate incorporating Bellicum’s iMC activation and CaspaCIDe safety switch technologies, which in tandem may serve to enhance cell proliferation, persistence, and anti-tumor effect while also enabling effective management of adverse events.

In the first patients treated in this trial at dose level 1 of 0.1×106 cells/kg BPX-603 alone or followed by weekly rimiducid:

No dose-limiting toxicities were observed.
Serious adverse events reported were pleural effusion and pneumonia. Neither was determined to be related to study drug.
Other Grade 3+ treatment-emergent adverse events reported were neutropenia, leukopenia, and anemia.
No CRS or ICANS events were reported.
Limited cell engraftment and expansion were observed.
Enrollment is ongoing at dose level 2 of 1×106 cells/kg alone or followed by weekly rimiducid, and results from these patients will be submitted for presentation at a medical meeting in 2022.

Stated Charity Scripture, PharmD, Chief Development Officer of Bellicum, "The early clinical activity we have observed with BPX-601 in mCRPC and the manageable safety profiles observed across both studies are highly encouraging. Given the very low starting cell dose in the BPX-603 trial, the lack of clinical efficacy at dose level 1 was in line with our expectations. I am thrilled to be re-joining Bellicum in a full-time capacity at such an exciting time in our programs’ development. We look forward to continued dose escalation to more fully evaluate the safety and potential efficacy of these product candidates."

Private Placement Completed

Bellicum will issue, in the private placement, pre-funded warrants to purchase up to 20,559,210 shares of common stock and accompanying warrants to purchase up to 2,055,920 shares of common stock. Each pre-funded warrant to purchase common stock is being sold together with one warrant to purchase one-tenth of one share of common stock at a combined unit price of $1.7024. The common stock warrants will be immediately exercisable at an exercise price of $1.69 per share of common stock and will expire seven years from the date of issuance. Each pre-funded warrant will have an exercise price of $0.0001 per share and will be exercisable immediately.

The aggregate offering size, before deducting placement agent commissions and other offering expenses, is expected to be approximately $35.0 million, excluding any proceeds that may be received upon exercise of the common warrants. Bellicum anticipates using the net proceeds from the offering, together with its existing capital resources, to fund ongoing and planned BPX-601 and BPX-603 clinical trials, and for general corporate purposes, including research and development and to fund working capital.

In conjunction with the equity financing announced today, the holders of the option to purchase securities associated with Bellicum’s August 2019 Private Placement have waived their rights, in full, to exercise their right to purchase Series 2 preferred stock and associated warrants, and Series 3 preferred stock and associated warrants, respectively.

Mr. Fair added, "We are pleased to have the support provided in the financing announced today. The waiver of the rights to exercise the Series 2 and Series 3 options is also a positive step in the simplification of our capital structure."

The private placement is expected to close on December 7, 2021. A Current Report on Form 8-K containing more detailed information regarding the financing will be filed with the Securities and Exchange Commission.

MTS Securities, LLC, an affiliate of MTS Health Partners, L.P., is acting as exclusive placement agent in the financing.

The Securities issued or to be issued to the purchasers in the Offering announced today, subject to the closing, were, or will be issued, as applicable, pursuant to an exemption from registration under the Securities Act of 1933 (the "Securities Act"). The securities have not been and will not be registered under the Securities Act or any state or other jurisdiction’s securities laws and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdiction’s securities laws.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the Securities or any other securities, nor shall there be any offer, solicitation or sale of the Securities or any other securities in any state or other jurisdiction in which such an offer, solicitation or sale would be unlawful.

Kaleido Biosciences Announces Expansion of Collaboration with Janssen to Explore Potential of Microbiome Metabolic Therapies (MMT™) to Prevent Childhood-Onset of Atopic, Immune and Metabolic Conditions

On December 6, 2021 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage biotech company with a differentiated, small-molecule approach to treating inflammatory conditions and diseases by selectively targeting the resident microbiome to restore gut-immune homeostasis, reported the extension and expansion of its research collaboration with Janssen Biotech, Inc. ("Janssen") (Press release, Kaleido Biosciences, DEC 6, 2021, View Source [SID1234596501]). The collaboration is designed to explore the potential for Microbiome Metabolic Therapies (MMT) to prevent childhood-onset of atopic, immune and metabolic conditions by promoting healthy function of the gut microbiome.

Since the beginning of the collaboration, Kaleido’s proprietary ex vivo screening platform has identified several potential MMTs that support the growth of specific beneficial microbes in the gut microbiome believed to be associated with the development of infant immune systems. Given this progress, the existing agreement with Janssen has subsequently been extended and expanded to optimize the identified MMTs for their ability to support growth of these advantageous microbes and their metabolites. Janssen will fund the additional costs associated with the expanded scope of the collaboration.

"We have made exciting progress to date in identifying relevant MMTs with desired effects on the infant microbiome communities," said Johan van Hylckama Vlieg, Ph.D., Chief Scientific Officer of Kaleido. "Our expertise in the microbiome combined with our unique platform positions us well to continue advancing our understanding of how glycans modulate the infant gut microbiome to support a developing immune system."

About Microbiome Metabolic Therapies (MMT)
Kaleido’s Microbiome Metabolic Therapies, or MMTs, are designed to drive the function and distribution of the microbiome’s existing microbes in order to decrease or increase the production of metabolites, or to advantage or disadvantage certain bacteria in the microbiome community. The Company’s initial MMT candidates are targeted, synthetic glycans that are orally administered, have limited systemic exposure, and are selectively metabolized by enzymes in the microbiome. Kaleido utilizes its discovery and development platform to study MMTs in microbiome samples to rapidly advance MMT candidates into clinical studies in healthy subjects and patients. These human clinical studies may be conducted under regulations supporting research with food, evaluating safety and tolerability and impact on the microbiome. For MMT candidates that are developed as therapeutics, the Company currently conducts and will conduct clinical trials under an Investigational New Drug (IND) or regulatory equivalent outside the U.S., often in Phase 2 or later development.

Greenwich LifeSciences Announces Commencement of the First Commercial Line Filling of GP2

On December 6, 2021 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, reported that manufacturing and clinical updates on Phase III clinical trial FLAMINGO-01 (Press release, Greenwich LifeSciences, DEC 6, 2021, View Source [SID1234596500]).

Over the past six months, the Company has been actively selecting and contracting partners to manufacture clinical and commercial lots of GP2 and to manage the Phase III clinical trial. An update on these activities follows:

Today, the Company ran its first commercial fill line of GP2 at its fill and finish partner’s production facility with sufficient scale to produce up to 80,000 doses of GP2 per lot. Three commercial lots of GP2 will be required to file a Biologics License Application (BLA) for GP2. The three lots combined should treat approximately 22,000 patients, and in the initial GP2 indication, approximately 17,000 new patients could be treated per year, saving up to 1,500 to 2,000 lives per year.
Clinical research organization (CRO) has been engaged to manage the Phase III clinical trial.
GP2 active ingredient manufacturer has been engaged to produce commercial scale GP2 lots.
Commercial testing laboratory has been engaged to release clinical and commercial GP2 lots and to manage GP2 stability program.
In addition to their operational role, these partners will play a key role in preparing the manufacturing and clinical information necessary to submit a BLA upon a successful interim analysis of the Phase III clinical trial.
Partners for HLA typing, blood test collection, GP2 storage and distribution, GM-CSF distribution, immune response testing, and blood sample storage have been selected to support the Phase III clinical trial.
CEO Snehal Patel commented, "Commencing commercial scale manufacturing is a major milestone for the Company. We are carefully selecting partners who are leaders in the industry with whom we hope to work with over the long term. At the same time, the Company is developing a back-up strategy by location and function to reduce risk and provide multiple options going forward. In addition, all of the various parties required to operate the Phase III clinical trial testing, storage, and distribution activities have been integrated well through our management team and CRO. The expertise of our partners has served to enhance the quality and accelerate the progress of our manufacturing and clinical plans."

About FLAMINGO-01 and GLSI-100

The Phase III clinical trial will be called FLAMINGO-01 and the combination of GP2 + GM-CSF will be called GLSI-100. The Phase III trial is comprised of 2 blinded, randomized, placebo-controlled arms for approximately 500 HLA-A*02 patients and 1 open label arm of up to 100 patients for all other HLA types. An interim analysis has been designed to detect a hazard ratio of 0.3 in IDFS, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater. The trial is currently being registered on clinicaltrials.gov and the link and trial identifier will be published shortly. For future updates about FLAMINGO-01 please visit the Company’s clinical trial tab at View Source

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 282,000 new breast cancer patients and 3.8 million breast cancer survivors in 2021. HER2/neu (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.