On March 8, 2022 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), reported the execution of an asset purchase agreement (the Agreement) for the sale of BridgeBio’s NULIBRY (Fosdenopterin) for Injection (Press release, BridgeBio, MAR 8, 2022, View Source [SID1234609754]). NULIBRY is approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening pediatric genetic disorder. The closing of the asset purchase is subject to customary closing conditions.

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"Sentynl’s focus on meaningful treatments for serious rare diseases is further enhanced by the acquisition of Fosdenopterin. We will leverage our existing platform of ultra-rare pediatric disease initiatives to facilitate early diagnosis and treatment by enhancing awareness, newborn screening, genetic testing and patient support across multiple products and rare diseases. By partnering with BridgeBio, we hope to reach even more patients born with MoCD Type A as quickly as possible with the hope of reducing the risk of mortality and progression of this devastating disease," said Matt Heck, CEO of Sentynl.

Under the Agreement, Sentynl will acquire global rights to NULIBRY and will be responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing and commercializing Fosdenopterin globally. BridgeBio will share development responsibilities for Fosdenopterin through approval of the marketing authorization application already under accelerated assessment with the European Medicines Agency and through approval of its regulatory submission with the Israeli Ministry of Health. Sentynl will provide cash payments upon the achievement of certain regulatory milestones. BridgeBio will be eligible to receive commercial milestone payments as well as tiered royalties on adjusted net sales of NULIBRY.

"Sentynl is an ideal partner given its expertise in the rare pediatric neurodevelopment space and its relationships with physicians who diagnose and treat children with MoCD Type A," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "Focused execution means reducing the scope of our internal activity. We will continue to advance high-quality programs in our pipeline while expanding our reach to patients in need of options."

About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity.1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.2

MoCD Type A is an ultra-rare disease. The incidence and prevalence of MoCD Type A in the United States are not known, but the estimated incidence is 1 per 342,000 to 411,000 live births (0.24 and 0.29 per 100,000).3 Based on these estimates, MoCD Type A is likely to be underdiagnosed, with an estimated 22 to 26 missed diagnoses per year in the United States and European Union.

The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

About NULIBRY (Fosdenopterin) for Injection
NULIBRY (Fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.

On March 8, 2022 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported an upcoming poster presentation at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place in Atlanta, GA on April 8-13, 2022 (Press release, Bio-Path Holdings, MAR 8, 2022, View Source [SID1234609753]).

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Dr. Maria Gagliardi, a Research Scientist at Bio-Path Holdings, will discuss pre-clinical studies of BP1003 (liposomal STAT3 antisense) in combination with paclitaxel or fluorouracil as a potential treatment against breast and ovarian cancer cells.

Details for the poster presentation are as follows:

Date: April 12, 2022
Presentation Time: 1:30-5 pm Eastern Time
Session: Experimental and Molecular Therapeutics
Abstract Number: 3285
Title: Targeting STAT3 with novel liposome-incorporated antisense oligonucleotide technology enhances the efficacy of paclitaxel (taxol) or 5-fluorouracil (5-FU) in breast and ovarian cancer cells

On March 8, 2022 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported abstracts highlighting the company’s lead RAF kinase inhibitor program, KIN-2787, have been accepted for presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, to be held April 8-13, in New Orleans, Louisiana (Press release, Kinnate Biopharma, MAR 8, 2022, View Source [SID1234609744]).

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Abstracts accepted for poster presentation include:

Occurrence of BRAF class II and III alterations is common across solid tumors and is associated with inferior clinical outcomes in NSCLC and melanoma (PAN# 4122). The poster will be presented by Paul Severson, Ph.D., Senior Director of Translational Medicine & Bioinformatics at Kinnate.
Design and rationale of a first in human (FIH) Phase 1/1b study evaluating KIN-2787, a potent and highly selective pan-RAF inhibitor, in adult patients with BRAF- and NRAS-mutation positive solid tumors (PAN# CT248). The poster will be presented by Meredith McKean, M.D., M.P.H., Director, Melanoma and Skin Cancer Research Program, Sarah Cannon Research Institute at Tennessee Oncology.
Antitumor activity of KIN-2787, a next-generation pan-RAF inhibitor, in preclinical models of human RAF/RAS mutant melanoma (PAN# 2674). The poster will be presented by Nichol Miller, Ph.D., Senior Director of Translational & Discovery Biology at Kinnate.
"We are honored that these abstracts focused on our RAF program were selected for presentation at this year’s AACR (Free AACR Whitepaper) annual meeting," said Richard Williams, MBBS, Ph.D., Chief Medical Officer at Kinnate. "The majority of oncogenic BRAF alterations are Class II or III alterations that function as dimers to drive cancer cell growth. In certain cancers, outcomes for patients with BRAF Class II or III alterations are inferior when treated with available therapies. In preclinical studies, KIN-2787 has demonstrated its potential as a promising next-generation RAF inhibitor with unique properties that have demonstrated potent activity against a variety of oncogenic BRAF-driven cancers, including those where Class II and III alterations are present."

KIN-2787, is an orally available small molecule pan-RAF inhibitor being developed for the treatment of patients with lung cancer, melanoma, and other solid tumors. KIN-2787 has been designed to target both monomeric and dimeric forms of the mutant BRAF kinase and minimize paradoxical activation, a liability often observed with other RAF inhibitors that can adversely impact tolerability and require addition of a MEK inhibitor to suppress pathway activation. Unlike currently available treatments that target only Class I BRAF kinase alterations, KIN-2787 targets Class II and Class III BRAF alterations, where it has the potential to be a first-line targeted therapy, in addition to covering Class I BRAF alterations. The ongoing KN-8701 clinical trial (NCT# 04913285) of KIN-2787 is actively enrolling patients across multiple centers in the United States.

Additional information about AACR (Free AACR Whitepaper) 2022 is available at: www.aacr.org/meeting/aacr-annual-meeting-2022.

On March 8, 2022 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing next-generation neoantigen immunotherapies, reported that it will present clinical, preclinical, and manufacturing data at next month’s American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 to be held from April 8-13 in New Orleans (Press release, Genocea Biosciences, MAR 8, 2022, View Source [SID1234609742]). The findings include late-breaking clinical data from the TiTAN clinical trial for the neoantigen-targeted peripheral T cell therapy product candidate GEN-011, results demonstrating successful production of GEN-011 using Genocea’s PLANET manufacturing process, and new preclinical data on Inhibigens, antigens of suppressive immune responses uniquely identifiable by Genocea’s ATLAS platform.

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The TiTAN trial is an open-label, multi-center Phase 1/2a study of GEN-011, which is comprised of neoantigen-targeted peripherally-derived T cells (NPTs) that are screened for patient-specific neoantigens by Genocea’s ATLAS platform. The platform identifies, through a bioassay, each patient’s anti-tumor neoantigens and pro-tumor Inhibigens. The PLANET manufacturing process selectively expands T cells specific to the ATLAS-identified neoantigens. Of the patient samples entering PLANET, 100% have either successfully yielded a released drug product or are in process.

"The preclinical data strengthen our conviction that using ATLAS to identify and selectively include T cells that drive anti-tumor responses and exclude Inhibigens whose T cells promote tumor growth enhances the potential of GEN-011 to treat of solid tumor cancers," said Thomas Davis, MD, Chief Medical Officer of Genocea. "By taking a comprehensive approach to neoantigen identification, we are creating new and potentially more effective cell therapies for patients."

Details on poster presentations for the TiTAN clinical trial patient data, the PLANET manufacturing process and Inhibigens are shared below.

AACR POSTER SESSION CATEGORY: Phase 1 Adult Clinical Trials
Poster #CT153
Title: TiTAN: a phase 1 study of GEN-011, a neoantigen-targeted peripheral blood-derived T cell therapy with broad neoantigen targeting
Presenter: Maura Gillison, MD, PhD, MD Anderson Cancer Center
Date: Monday, April 11, 2022
Time: 1:30 p.m. – 5:00 p.m. CT

AACR POSTER SESSION CATEGORY: Inflammation, Immunity, and Cancer
Poster #2088
Title: The PLANET manufacturing process reproducibly generates high-quality neoantigen-targeted peripheral T cells (NPTs) for adoptive T cell therapy in the TiTAN clinical trial
Presenter: Harshal Zope, PhD, Genocea Biosciences
Date: Monday, April 11, 2022
Time: 1:30 p.m. – 5:00 p.m. CT

AACR POSTER SESSION CATEGORY: Clinical Research Excluding Trials
Poster #2745
Title: ATLAS-identified Inhibigen-specific responses accelerate tumor growth in mouse melanoma and pancreatic cancer
Presenter: Jessica Flechtner, PhD, Genocea Biosciences
Date: Tuesday, April 12, 2022
Time: 9:00 a.m. – 12:30 p.m. CT

Posters will be available on-demand on the AACR (Free AACR Whitepaper) website at www.aacr.org beginning at the start of the poster session until July 13, 2022.

About GEN-011
GEN-011 is a neoantigen-targeted peripherally derived T cell therapy candidate comprised of autologous CD4+ and CD8+ T cells that are specific for up to 30 ATLAS-identified neoantigens to limit tumor escape. NPTs have minimal bystander, non-tumor-specific cells, and are devoid of Inhibigen-specific cells which may be detrimental to clinical response.

About the GEN-011 TiTAN clinical trial
TiTAN is an open-label, multi-center Phase1/2a trial evaluating safety, tolerability, T cell persistence and proliferation and clinical efficacy. The TiTAN clinical trial is testing two dosing regimens, a repeated fractional dose regimen of GEN-011 without lymphodepletion and a single dose administration of GEN-011 after lymphodepletion. Both groups will receive interleukin-2 after GEN-011 dosing to maximize the tumor-killing potential of the infused cells.

On March 8, 2022 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company reported full year 2021 financial results and provided a company update (Press release, Cumberland Pharmaceuticals, MAR 8, 2022, View Source [SID1234609741]). Net revenues totaled $36 million, resulting in $6 million in cash flow from operations.

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As of December 31, 2021, the company’s total assets were $84 million, including $27 million in cash. Total liabilities were $42 million, including $15 million on its credit facility and total shareholders’ equity was $43 million.

"Despite the continued challenges of operating during a pandemic, our diversified product portfolio of FDA-approved brands has helped mitigate its negative effects and allowed us to deliver another steady performance in 2021," said A.J. Kazimi, CEO of Cumberland Pharmaceuticals. "We closed out the year announcing our acquisition of Sancuso, an oncology-support drug that we believe will be favorable to our financial performance and provide significant benefits to oncology patients being treated for their cancers. We look forward to the year ahead and welcome the return to a more traditional operating environment."

Cumberland acquired the U.S rights to Sancuso from the U.S. subsidiary of Kyowa Kirin, a specialty pharmaceutical company based in Japan. Sancuso is the first and only FDA-approved prescription patch for the prevention of nausea and vomiting in cancer patients receiving certain types of chemotherapy treatment.

The active drug in Sancuso, granisetron, slowly dissolves in the thin layer of adhesive that sticks to the patient’s skin and is released into their bloodstream over several days, working continuously to prevent chemotherapy-induced nausea and vomiting (CINV). It is applied 24 to 48 hours before receiving chemotherapy and can prevent CINV for up to five consecutive days. Alternative oral treatments must be taken several times – day and night – to deliver the same therapeutic doses.

In the fourth quarter of 2021, Cumberland extended its bank line of credit for a new three-year term and expanded the facility to provide up to $20 million in capital to fund the Sancuso acquisition.

In November 2021, Cumberland received FDA approval of expanded labeling for its Caldolor product. The intravenously delivered formulation of ibuprofen is now approved for use prior to surgery. Orthopedic surgeon Dr. Stephen Southworth, who has published extensively on intravenous ibuprofen, supported this development stating that "when administered immediately prior to surgery, patients given Caldolor experience less postoperative pain and a decrease in their opioid use."

Also during 2021, Cumberland implemented the national launch of RediTrex, its FDA-approved line of injectable methotrexate products. RediTrex features an innovative delivery system for easy handling and dosing accuracy. It is approved for patients with severe, active rheumatoid arthritis and polyarticular juvenile idiopathic arthritis who have difficulty tolerating or responding to orally delivered methotrexate.

Other highlights from the year:

Cumberland signed an agreement with Verity Pharmaceuticals to license and commercialize Vibativ in Puerto Rico. Verity has a strong presence in Puerto Rico, which is in need of a product with Vibativ’s features, as it has a large number of residents living with chronic diseases, like diabetes, that increase the risk of hospitalization and infections. Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections, including hospital-acquired and ventilator-associated bacterial pneumonia and complicated skin infections.

SciClone Pharmaceuticals, which has licensed Cumberland’s Vibativ product for China, submitted an application for the product’s approval in that market in early 2021. In September, the submission was accepted for review, which is expected to occur over a 12-month period.

Enrollment in Cumberland’s clinical studies resumed after interruption due to the pandemic. Cumberland is sponsoring three Phase II clinical programs evaluating its ifetroban product candidates. These studies involve patients with cardiomyopathy associated with 1) Duchenne Muscular Dystrophy, a fatal, genetic neuromuscular disease; 2) Systemic Sclerosis, a debilitating autoimmune disorder; and 3) Aspirin-Exacerbated Respiratory Disease, a severe form of asthma.

Cumberland issued its second annual Sustainability Report, which details the company’s activities pertaining to its environmental, social and governance matters. The 2020 Sustainability Report notes that Cumberland provided nearly 2.5 million patient doses of its products, safely disposed of over 4,000 pounds of expired and damaged products, and had no product recalls. Cumberland also had no company brands listed on the FDA’s MedWatch Safety Alerts for Human Medical Products, no company product issues identified by FDA from its Adverse Event Reporting System and no clinical trials terminated due to failure to practice good clinical standards.

The 2020 Sustainability Report also highlights several initiatives Cumberland implemented as part of its commitment to delivering high-quality pharmaceutical products to improve patient care. For example, the company continued a program to serialize all commercial products sold in the U.S., allowing it to track every unit distributed, which helps to prevent counterfeit drugs from entering the market under the Cumberland brand.

The company also announced its upcoming move to the new Broadwest campus in Nashville’s West End/Vanderbilt corridor. The new location will keep Cumberland close to the internationally recognized Vanderbilt Medical Center, with whom it regularly collaborates. It will also provide Cumberland a long-term home with increased efficiency and convenience for its overall operations.

Cumberland also announced several publications in support of its products as well as a series of patient case studies outlining real-world instances in which Vibativ was used to effectively and safely treat COVID-19 patients.
FINANCIAL RESULTS:

Net Revenue: For the three months ended December 31, 2021, net revenues from continuing operations were $8.3 million. The company also recorded an additional $0.5 million in revenue during the fourth quarter associated with divested rights to products that the company no longer distributes.

Annual net revenues were $36 million, with an additional $2 million in revenue associated with divested product rights.

Net revenue by product for the fourth quarter of 2021 included $3.7 million for Kristalose, $2.9 million for Vibativ, $1.2 million for Caldolor, $0.2 million for Acetadote, $0.1 million for Omeclamox, $0.1 million for RediTrex and a break-even point for Vaprisol.

Annual net revenue by product included $16 million for Kristalose, $11.7 million for Vibativ, $5 million for Caldolor, $1.9 million for Vaprisol, $0.9 million for Acetadote, $0.1 million for RediTrex and Omeclamox reporting current year deductions over sales.

Operating Expenses: Total operating expenses for the fourth quarter were $12.7 million and $43.7 million for the full year 2021.

Adjusted Earnings: Adjusted earnings for the fourth quarter of 2021 were $(1.9) million compared to a net loss of $(4.4) million. For the full year, adjusted earnings were $(1.2) million, or $(0.08) per share compared to a net loss of $(5.6) million, or $(0.37) per share.

The annual 2021 adjusted earnings calculation does not include the benefit of the $2 million in payments received for the two products returned. It also does not include the benefit of the $3.4 million of Vibativ cost of goods associated with sales of the product during the year, which was received with the product acquisition. Cumberland recorded an additional $1.1 million in a one-time write-off of expired inventory, also received as part of the acquisition.

Cash Flow: Cash flow from operations for the year ended December 31, 2021 was $6.3 million.

Balance Sheet: At December 31, 2021, Cumberland had $84 million in total assets, including $27 million in cash and cash equivalents. Total liabilities were $42 million, including $15 million outstanding on the company’s revolving line of credit. Total shareholders’ equity was $43 million.

CONFERENCE CALL & WEBCAST

A conference call and live Internet webcast will be held today March 8, 2022 at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please dial (877) 303-1298 (for U.S. callers) or (253) 237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing (855) 859-2056 (for U.S. callers) or (404) 537-3406 (for international callers). The Conference ID for the rebroadcast is 1497637. Both the live webcast and rebroadcast can be accessed via Cumberland’s website at View Source