On November 4, 2021 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the third quarter ended September 30, 2021 and provided an update on the Company’s operations (Press release, BridgeBio, NOV 4, 2021, View Source [SID1234594564]).

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BridgeBio has more than 30 programs in its pipeline for patients living with genetic diseases and cancers and 20 ongoing clinical trials underway across more than 450 sites around the world. Earlier this year BridgeBio received its first two U.S. Food and Drug Administration (FDA) drug approvals and has successfully filed 15 Investigational New Drug (IND) applications since the Company’s founding in 2015.

BridgeBio’s four core value drivers:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): Topline results from Part A of the Phase 3 ATTRibute-CM trial are expected in late 2021 and from Part B in 2023. The primary endpoint at Part A is the change from baseline in a 6-minute walk distance (6MWD) in trial participants receiving acoramidis or placebo after 12 months. If the change from baseline in 6MWD in Part A is highly statistically significant, BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA. The study enrolled more than 600 subjects with either wild-type or variant TTR across more than 80 sites in 18 countries. ATTR-CM is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): BridgeBio presented updated Phase 2b data for encaleret in an oral presentation at the American Society of Bone and Mineral Research (ASBMR) 2021 Annual Meeting in October. Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion. Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of current standard-of-care. If approved, encaleret could be the first therapy on the market for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor (CASR) gene estimated to be carried by 12,000 individuals in the United States alone. BridgeBio plans to initiate a Phase 3 registrational trial of encaleret in patients with ADH1 in 2022.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the first half of 2022. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with a prevalence of greater than 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known product candidate in clinical development for achondroplasia that is designed to target the disease at its genetic source and the only orally administered product candidate in clinical-stage development.
BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): Received Fast Track designation from the FDA in May 2021. IND cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in mid-2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases estimated in the United States and European Union. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol production. BridgeBio’s AAV5 gene therapy candidate is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.
Recent pipeline progress and corporate updates:

Stock repurchases: BridgeBio repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program, demonstrating the Company’s confidence in the long-term prospects of its pipeline.
LianBio IPO and partnership: China-based partner LianBio raised $325 million in its initial public offering on November 1. BridgeBio is estimated to own approximately 4.7% post-IPO. BridgeBio and LianBio announced in August that the first patient was treated in a Phase 2a trial of infigratinib in patients with gastric cancer and other advanced solid tumors. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.
RAS cancer portfolio: BridgeBio announced the discovery of its next-generation KRAS G12C dual inhibitors, the first-known compounds that directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations, and PI3ka:RAS breakers, small molecules that block RAS driven PI3Ka activation – a novel approach with the potential to inhibit oncogenic PI3Ka signaling without adverse effects on glucose metabolism. RAS is one of the most well-known oncogenic drivers with approximately 30% of all cancers being driven by RAS mutations, including large proportions of lung, colorectal and pancreatic tumors. BridgeBio expects to select a RAS development candidate in 2022.
BBP-812 – AAV9 gene therapy candidate for Canavan disease: BridgeBio announced that the first patient was dosed in its Phase 1/2 trial of BBP-812 for Canavan disease. If successful, BridgeBio’s gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and life-threatening condition. An initial Phase 1/2 data readout is expected in 2022.
BBP-818 – AAV gene therapy candidate for classic galactosemia (severe GALT deficiency): BridgeBio announced a new gene therapy program for classic galactosemia, which is caused by a severe deficiency of the enzyme galactose-1-phosphate uridylyltransferase (GALT), affecting approximately 7,000 patients in the United States and the European Union. Preclinical studies in a mouse model of classic galactosemia have shown that BridgeBio’s BBP-818 therapy restored up to 72% of wild-type levels of GALT enzyme in the brain following a single dose.
BBP-398 – SHP2 inhibitor: First publication of preclinical data for BridgeBio’s potentially best-in-class SHP2 inhibitor designed for the treatment of resistant cancer. Data demonstrated activity in non-small cell lung cancer (NSCLC) driven by RAS or other MAPK-pathway activating mutations. The results were featured in a poster presentation shared at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i): Fast Track designation granted by the FDA. The Phase 2 trial was initiated in patients with LGMD2i in the first quarter of 2021. If successful, BBP-418 could be the first approved therapy for patients with LGMD2i. With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein. A Phase 2 data readout is expected in 2022.
BBP-711 – Glycolate oxidase (GO) inhibitor for hyperoxaluria: BridgeBio announced preliminary Phase 1 data in which BBP-711 was well-tolerated and resulted in maximal increases in plasma glycolate exceeding those achieved by any GO-targeting agents reported in healthy adult volunteers. BBP-711 is being developed for the treatment of primary hyperoxaluria type 1 (PH1) and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers. A full readout of Phase 1 data in healthy adult volunteers is expected in 2022, to be followed by initiation of a Phase 2/3 trial in PH1 and a Phase 2 proof-of-concept trial in recurrent kidney stone formers.
TRUSELTIQ (infigratinib): Health Canada approved TRUSELTIQ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement. BridgeBio’s partner Helsinn Group has exclusive commercial rights for TRUSELTIQ in Canada with BridgeBio eligible for tiered royalties as a percentage of net sales as part of the global collaboration and license agreement entered into between the two companies in March 2021.
BridgeBio Pharma R&D Day: Held a virtual R&D Day on October 12, 2021. Presentation replay can be found on BridgeBio’s investor website here.
Four new independent directors added to BridgeBio’s board:
Hannah Valantine, M.D., a leader in organ transplant genomics and workforce diversity who is a professor of medicine at Stanford University School of Medicine
Fred Hassan, a pharmaceutical and financial industry leader who is the former CEO of Schering-Plough and former chairman of Bausch & Lomb
Andrea Ellis, a consumer technology innovator and the chief financial officer of Lime
Douglas Dachille, an investment management veteran and the former chief investment officer of American International Group, Inc. (AIG)
Third Quarter 2021 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $599.6 million as of September 30, 2021, compared to $607.1 million as of December 31, 2020. Over the past three quarters, the Company repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program and $50.0 million in BridgeBio common stock in conjunction with its issuance of the 2029 convertible notes, paid $61.3 million for capped call options related to the issuance of its 2029 convertible notes and $35.0 million of regulatory-related milestone payments in connection with its approved products. Earlier during the year, BridgeBio paid $21.3 million to Eidos shareholders who elected for cash settlement in exchange for their Eidos shares and $63.8 million of direct transaction costs arising from the merger with Eidos. These were offset by cash receipts of $731.4 million in net proceeds from the issuance of BridgeBio’s 2029 convertible notes, $65.1 million from collaboration partner, Helsinn Group, and $25.0 million in net proceeds from Hercules Capital, Inc. under an amended loan agreement. The remaining change primarily related to payments of interest and operating costs and expenses.

Cash, cash equivalents and marketable securities, excluding restricted cash decreased by $298.8 million when compared to balance as of June 30, 2021, which was $898.4 million. During the quarter, the Company repurchased $143.1 million of BridgeBio common stock and paid $35.0 million of regulatory-related milestone payments in connection with its approved products.

Operating Costs and Expenses

Operating costs and expenses for the quarter increased by $23.7 million to $151.8 million in the current quarter as compared to $128.1 million for the same period in the prior year. The increase in operating costs and expenses was due to an increase in personnel and external costs to support the progression in BridgeBio’s research and development programs and staged buildout of its commercial organization as part of commercial launch readiness activities. This increase in personnel and external costs was offset by $12.2 million in reimbursement of expenses from the cost sharing arrangement recognized under BridgeBio’s License and Collaboration Agreement with Helsinn Group. Stock-based compensation for the quarter was $16.1 million as compared to $17.7 million for the same period in the prior year.

The Company’s research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While BridgeBio experienced some delays in certain of its clinical enrollment and trial commencement activities, it continues to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with its contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on BridgeBio’s operating costs and expenses is currently unknown.

On November 4, 2021 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Jounce Therapeutics, NOV 4, 2021, View Source [SID1234594563]).

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"Jounce has had another very productive quarter as we have made significant progress across all areas of the company and continue to build momentum as we head into the new year. We were pleased to report that we are now actively recruiting patients in the monotherapy and combination therapy expansion portion of the INNATE study, testing JTX-8064 +/- pimivalimab, our own PD-1 inhibitor, in 8 distinct cohorts across 7 tumor types. We also continue to advance patient enrollment in our randomized SELECT study which employs our stringent patient selection strategy and have continued our commitment to build our discovery pipeline," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "We believe our focus on translational science, biomarker approaches, and targeting new immune mechanisms leads us closer to bringing the right immunotherapies to the right patients."

Pipeline Update:

JTX-8064 (LILRB2 / ILT4)

Completed monotherapy and combination therapy dose escalation 3 week safety review of INNATE: Jounce has completed the monotherapy and pimivalimab (PD-1 Inhibitor) combination therapy dose escalation 3 week safety review of the Phase 1 trial of JTX-8064, and a preliminary recommended phase 2 dose (RP2D) of 700 mg has been established. To date, JTX-8064 has been well tolerated with no dose limiting toxicities.

Enrollment initiated in tumor-specific monotherapy and pimivalimab combination expansion cohorts of INNATE: Jounce previously announced the initiation of patient enrollment in INNATE tumor-specific expansion cohorts for both JTX-8064 monotherapy and combination therapy of JTX-8064 with pimivalimab in August and October, respectively. The expansion cohorts will study JTX-8064 in three subsets of patients in order to identify the most rapid development paths for JTX-8064 alone or in combination with PD-1 inhibitors:

Patients who have progressed on PD-1 inhibitors and are now PD-1 inhibitor resistant,
PD-1 inhibitor naïve patients with tumors that do not typically respond to PD-1 inhibitors, and
PD-1 inhibitor naïve patients with tumors for which PD-1 inhibitors are approved, but there is still room for improvement.

Vopratelimab (ICOS) and Pimivalimab (PD-1)

Continued enrollment in Phase 2 SELECT trial of vopratelimab: Enrollment continues in SELECT, a randomized Phase 2 trial to evaluate vopratelimab in combination with pimivalimab versus pimivalimab alone in immunotherapy naïve, TISvopra biomarker-selected, second line non-small cell lung cancer (NSCLC) patients. The SELECT trial also aims to provide additional important single agent data for pimivalimab in a new biomarker selection paradigm. Jounce is on track to report data from the SELECT trial in 2022.

Gilead Sciences begins Phase 1 clinical trial of GS-1811 (formerly JTX-1811)

The clinical study of GS-1811 (formerly JTX-1811) was initiated by Gilead Sciences. GS-1811, which Jounce discovered and progressed through to IND, is out licensed to Gilead and is the fourth internally developed candidate to enter the clinic.

Corporate Update:

Jigar Raythatha Appointed to the Board of Directors

On September 15th, 2021, Jounce announced the appointment of former chief executive officer of Constellation Pharmaceuticals and former Jounce chief business officer, Jigar Raythatha, to its board of directors.

Third Quarter 2021 Financial Results:

Cash position: As of September 30, 2021, cash, cash equivalents and investments were $249.0 million, compared to $213.2 million as of December 31, 2020. The increase was primarily due to receipt of $90.9 million in net proceeds from the follow-on public offering and sales under Jounce’s at-the-market ("ATM") offering program completed first quarter of 2021 and receipt of a $25.0 million milestone from Gilead in the third quarter of 2021, offset by operating expenses incurred.

License and collaboration revenue: No license or collaboration revenue was recognized in the third quarter of 2021 or 2020.

Research and development expenses: Research and development expenses were $23.3 million for the third quarter of 2021, compared to $18.0 million for the same period in 2020. The increase in research and development expenses was primarily due to increased clinical and regulatory costs for INNATE and SELECT, increased manufacturing activities performed for Jounce’s development programs and payroll and stock-based compensation expense.

General and administrative expenses: General and administrative expenses were $6.9 million for the third quarter of 2021, compared to $7.1 million for the same period in 2020. The decrease in general and administrative expenses was primarily a result of decreased professional fees offset by increases in other administrative costs.

Net loss: Net loss was $30.1 million for the third quarter of 2021, resulting in basic and diluted net loss per share of $0.59. Net loss was $24.9 million for the same period in 2020, resulting in a basic and diluted net loss per share of $0.73. The increase in net loss is attributable to increased operating expenses, the decrease in the net loss per share is attributable to increased number of shares outstanding as compared to the same period in 2020.

Financial Guidance:

Based on its current operating and development plans, Jounce is narrowing its financial guidance on gross cash burn on operating expenses and capital expenditures for the full year 2021 to $100.0 million to $110.0 million. Jounce expects to end fiscal year 2021 with approximately $220.0 million in cash, cash equivalents and investments.

Given the strength of its balance sheet, Jounce continues to expect its existing cash, cash equivalents and investments to be sufficient to enable the funding of its operating expenses and capital expenditure requirements through the third quarter of 2023.

Conference Call and Webcast Information:

Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 6873343. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. The webcast will be archived and made available for replay on Jounce’s website approximately two hours after the call and will be available for 30 days.

About JTX-8064

JTX-8064 is a humanized IgG4 monoclonal antibody designed to specifically bind to Leukocyte Immunoglobulin Like Receptor B2 (LILRB2/ILT4) and block interactions with its ligands. JTX-8064 is the first tumor-associated macrophage candidate developed from Jounce’s Translational Science Platform. Preclinical data presented at the 2020 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting and the 2019 and 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meetings support the development of JTX-8064 as a novel immunotherapy to reprogram immune-suppressive macrophages and enhance anti-tumor immunity. A Phase 1 clinical trial named INNATE (NCT04669899) of JTX-8064 as a monotherapy and in combination with Jounce’s internal anti-PD-1 inhibitor, pimivalimab (formerly JTX-4014) is currently enrolling patients with advanced solid tumors into tumor-specific expansion cohorts.

About Pimivalimab

Pimivalimab (formerly JTX-4014) is a well-characterized fully human IgG4 monoclonal antibody designed to block binding to PD-L1 and PD-L2. Pimivalimab demonstrated a 17% durable overall response rate in a Phase 1 trial of 18 heavily pre-treated PD-(L)1 inhibitor naïve patients, which excluded all tumor types for which PD-(L)1 inhibitors were approved. In this Phase 1 trial, pimivalimab was shown to have an acceptable safety profile. Pimivalimab is currently being assessed in the INNATE Phase 1 trial (NCT04669899) in combination with JTX-8064, a LILRB2 (ILT4) inhibitor. Pimivalimab is also being assessed in the SELECT Phase 2 clinical trial (NCT04549025) in combination with vopratelimab, a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO-Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors.

About Vopratelimab

Vopratelimab is a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO-Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors. Vopratelimab is currently being assessed in the SELECT Phase 2 clinical trial (NCT04549025) in combination with Jounce’s internal investigational PD-1 inhibitor, pimivalimab (formerly JTX-4014), compared to pimivalimab alone. The SELECT trial is currently enrolling approximately 75 immunotherapy naïve NSCLC patients who have been pre-selected with the TISvopra predictive biomarker, an 18 gene RNA tumor inflammation signature which predicted the emergence of ICOS hi CD4 T cells and clinical benefit in the ICONIC trial of vopratelimab alone and in combination with a PD-1 inhibitor. SELECT is powered to demonstrate the statistical superiority of the combination of vopratelimab plus pimivalimab compared to pimivalimab.

On November 4, 2021 Enterome, a clinical stage biopharmaceutical company developing first-in-class immunomodulatory drugs based on its unique ability to decode the gut microbiome’s interaction with the immune system, reported the launch of its new corporate brand and visual identity supported by a new website (Press release, Enterome, NOV 4, 2021, View Source [SID1234594562]).

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These changes reflect the significant progress that Enterome has made with its two advanced, fully owned, OncoMimics and EndoMimics pipelines of transformational medicines for cancer and immune diseases, respectively.

Enterome’s most advanced OncoMimics drug candidates are:

EO2401, a therapeutic cancer vaccine candidate currently in clinical development for recurrent glioblastoma and adrenal tumors. EO2401 has already shown strong immunogenicity data in brain and adrenal cancer patients with clinical proof of concept data expected in H1 2022, and
EO2463, a second OncoMimic cancer vaccine, in clinical development for the treatment of indolent non-Hodgkin lymphoma with clinical proof of concept data planned for H1 2023
In addition, the Company’s most advanced EndoMimics candidate is:

EB1010, a new orally delivered bioactive and a potent local inducer of IL-10, which has been selected to provide improved therapeutic outcomes for patients with inflammatory bowel disease (IBD). EB1010 is expected to enter clinical trial in 2023
The Enterome name was created using a combination of the Greek prefix "ENTER", which means ’from the gut’ and the suffix of gen’OME’. This name highlights Enterome’s world-leading ability to interrogate the collective gut microbiome genome to identify and characterize bioactive proteins and peptides as a foundation to generate novel therapeutic vaccines and biological drugs.

Enterome’s drug discovery platform leverages a unique combination of biocomputational tools and bioassays, to explore new therapeutic solutions from the world’s largest database of 20 million functionally annotated full-length gut microbiome proteins. This use of cutting-edge tools means that Enterome’s platform is more efficient than well established, labor-intensive drug discovery approaches. Enterome’s platform has already demonstrated it is highly productive and has generated multiple pipelines of transformative drug candidates, targeting a broad range of therapeutic areas.

On November 4, 2021 Morphic Therapeutic (Nasdaq: MORF), a biotechnology company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the third quarter of 2021 (Press release, Morphic Therapeutic, NOV 4, 2021, View Source [SID1234594561]).

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Recent Highlights and Outlook

Presented positive phase 1 data at the European Crohn’s and Colitis Organisation (ECCO) Virtual Congress 2021, fully supporting MORF-057’s target product profile as an oral inhibitor of the α4β7 integrin
MORF-057 was well tolerated in all dose cohorts with no safety signals identified
Pharmacodynamic data, including α4β7 receptor saturation at 100 mg BID administered orally, strongly supported MORF-057’s ability to replicate the mechanism of approved therapeutic vedolizumab, administered via IV infusion
MORF-057’s dose-dependent and consistent pharmacokinetic data support twice daily dosing profile and enable plans to evaluate once-daily dosing in phase 2b
Lymphocyte subset changes strongly demonstrate early in-human evidence for MORF-057’s small molecule α4β7 inhibition to replicate the published data on mechanism of action from approved biologic therapeutics in inflammatory bowel disease (IBD)
Planned initiation of MORF-057 phase 2a remains on track for first quarter 2022
All phase 2 readiness work, including chronic toxicology studies, on track and scheduled for completion in fourth quarter 2021
Study to include 30-35 patients with moderate to severe ulcerative colitis treated with 100 mg BID (twice daily) with 12-week induction phase and rollover to 40 additional weeks maintenance phase in patients
The primary endpoint is change in Robarts Histopathologic Index (RHI) at 12 weeks
Global phase 2b randomized controlled trial of MORF-057 to commence after phase 2a initiation and then run in parallel
Welcomed Nisha Nanda, Ph.D., to the Morphic Board of Directors, an experienced leader in preclinical and clinical-stage development strategy across multiple therapeutic areas
Received acceptance to present preclinical data from our αvβ8 inhibitor program at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting
Announced the planned resignation of Peter G. Linde, M.D., Chief Medical Officer, effective December 31, 2021. Dr. Linde has notified the Company that he is departing for personal reasons but will remain with the Company through year end to assist with an orderly transition of responsibilities
"MORF-057’s performance in phase 1 studies exceeded our internal modelling across all key criteria and we are now finalizing preparations for the initiation of the MORF-057 phase 2 program in patients with moderate-to-severe ulcerative colitis," said Praveen Tipirneni, M.D., President and Chief Executive Officer of Morphic Therapeutic. "Developing MORF-057 with our MInT platform has generated an array of learnings that feed into our earlier proprietary pipeline, driving advances in Morphic’s programs in immuno-oncology and other indications. In particular, we are looking forward to the presentation of new preclinical data from our αvβ8 program demonstrating the strong anti-tumor activity of a Morphic αvβ8 small molecule inhibitor dosed in combination with a checkpoint inhibitor and radioimmunotherapy in a tumor that is unresponsive to respective monotherapies or conventional radioimmunotherapy."

Financial Results for the Third Quarter 2021

Net loss for the quarter ended September 30, 2021 was $25.0 million or $0.69 per share, basic and diluted compared to net income of $5.4 million or $0.17 per share, diluted for the same quarter last year
Revenue was $3.1 million for the quarter ended September 30, 2021, compared to $25.8 million for the same quarter last year. The decrease was primarily due to the receipt of a $20 million payment triggered by AbbVie exercising their option to Morphic’s αvβ6 program during the quarter ended September 30, 2020
Research and development expenses were $21.0 million for the quarter ended September 30, 2021 as compared to $16.0 million for the same quarter last year. The increase was primarily due to clinical trial costs associated with MORF-057, along with manufacturing costs associated with the upcoming phase 2 clinical trial
General and administrative expenses were $7.3 million for the quarter ended September 30, 2021, compared to $4.8 million for the same quarter last year. The increase was due to an increase in headcount and higher professional and consulting costs associated with Morphic operating as a public company
As of September 30, 2021, Morphic had cash, cash equivalents and marketable securities of $427.6 million, compared to $228.3 million as of December 31, 2020. Morphic believes its cash, cash equivalents and marketable securities as of September 30, 2021, will be sufficient to fund operating expenses and capital expenditure requirements until the end of 2024.

On November 4, 2021 Vaxart, Inc. (NASDAQ: VXRT) reported its business update today for the third quarter of 2021, reporting strong forward momentum in developing its oral vaccine platform consisting of tablet vaccines that it believes may revolutionize public health (Press release, Vaxart, NOV 4, 2021, View Source [SID1234594560]).

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During the third quarter, Vaxart started the first Phase II trial of its investigational COVID-19 oral tablet vaccine and dosed its first subjects. Vaxart expects the full data set from this study to be available in Q1 2022.

Duke University published the results of its study of Vaxart’s COVID-19 vaccine candidate, finding that Vaxart’s oral tablet vaccine reduced the airborne transmission of SARS-CoV-2 virus in a hamster preclinical model.

The Company also expanded its research and manufacturing capabilities, enhancing its ability to advance its pipeline of vaccine candidates.

"Vaxart made significant progress this quarter toward its goal of developing a next-generation oral tablet COVID-19 vaccine," said Andrei Floroiu, Vaxart’s Chief Executive Officer. "We have started our United States Phase II trial and anticipate beginning international trials in the near future. This is an important milestone, as our candidate is the only oral COVID-19 vaccine to progress to Phase II trials in the U.S."

"Our view has been that an oral tablet vaccine could transform how the world is protected from COVID-19 and other infections because they are easy to distribute and administer. Now we added evidence suggesting the differentiated mucosal mechanism of action could be yet another improvement over injectable alternatives," added Mr. Floroiu.

"The progress we made in our vaccine research this quarter was significant," said Dr. Sean Tucker, Vaxart’s founder and Chief Scientific Officer. "We already learned from an earlier human influenza challenge study that our oral flu vaccine candidate inhibited the shedding of viral RNA better than injectable vaccines. Our published hamster study showed that our vaccine candidate can reduce transmission of SARS-CoV-2, even when there is infection breakthrough in a vaccinated subject."

"The implications are significant because existing injected vaccines do not always protect against viral shedding and transmission to other people. A vaccine that reduces shedding and reduces the probability of infection could make a big difference in protecting lives and public health."

Recent Business Highlights

Corporate Developments

Vaxart recently brought online its own GMP manufacturing facility and is now producing vaccines at two GMP plants. This has allowed the Company to manufacture all of the COVID-19 vaccine oral tablets for the clinical trials started and planned to start this year, and to begin manufacturing vaccines for its upcoming norovirus Phase II trials.
Vaxart scaled up its research, quality and manufacturing capabilities, increasing its R&D employee headcount by more than 35% during the quarter.
The Company created two advisory boards, the Scientific and Clinical Advisory Board and the Manufacturing and Quality Advisory Board, to provide expertise as the Company grows its scientific and manufacturing capabilities.
Vaxart named Dr. James F. Cummings as its Chief Medical Officer. Dr. Cummings is a board-certified infectious disease physician with extensive experience in vaccine, drug and diagnostics development, and will help guide the Company’s development of its vaccines.
Widely respected biotech executive Dr. Julie Cherrington joined Vaxart’s Board of Directors, deepening the pool of expertise on its board.
Preclinical and Clinical

Clinical COVID-19 Vaccine Developments

Vaxart made significant progress in Q3 2021 to advance its transformative approach to vaccines.

Vaxart began dosing subjects in October for its Phase II clinical trial in the United States, which is expected to enroll 96 subjects.
The Company expects data from this study to be available during Q1 2022.
The study will be conducted with subjects split evenly between COVID-19 naïve and mRNA vaccinated subjects.
During Q4 2021, Vaxart expects to start Phase Ib clinical testing in India.
The Company expects to launch additional international clinical trials in 1H 2022.
Preclinical COVID-19 Vaccine Developments

A Duke University-led study published in bioRxiv in October showed that Vaxart’s investigational oral tablet vaccine candidate reduced the airborne transmission of SARS-CoV-2 virus in a hamster model.
These results are consistent with those from Vaxart’s Phase II human flu challenge study, which showed that Vaxart’s oral tablet flu vaccine was better at reducing shedding than the injectable flu vaccine comparator.
Vaxart’s preclinical study also demonstrated that the Company’s oral vaccine platform induces robust systemic and mucosal responses.
The study suggested that mucosal vaccines may protect not only vaccinated, but also unvaccinated animals.
Vaxart will submit the results of its nonhuman primate study for publication in Q4 2021. Results from that study prompted Vaxart to move forward with its S-only vaccine candidate in Phase II clinical trials. This S-only vaccine candidate produced better antibody responses and cross-reactivity against all variants tested than Vaxart’s S+N vaccine candidate.
Norovirus Vaccine Developments

Norovirus is a highly infectious illness that affects around 20 million Americans annually, with an annual economic impact of approximately $10.5 billion in the United States.

Vaxart has almost completed enrollment in its Phase Ib placebo-controlled, dose-ranging, repeat dose trial investigating its oral norovirus vaccine candidate in elderly subjects aged 55 to 80 years. This study is designed to evaluate the safety and immunogenicity of Vaxart’s vaccine candidate.
The Company anticipates completing enrollment in its norovirus VXA-G1.1-104 study in Q4 2021.
Vaxart is also conducting an additional norovirus vaccine study to evaluate the optimal timing for boost administration under VXA-NVV-105. This study has completed enrollment.
The Company expects initial data to be available from these studies in Q1 2022 and more complete data by Q2 2022.
Vaxart is preparing to launch its norovirus challenge study in Q1 2022 to evaluate the safety and clinical efficacy of its oral vaccine candidate.
Manufacturing Updates

As noted above, Vaxart is now producing vaccines at two GMP manufacturing plants in California, including its own GMP manufacturing facility.
As a result, Vaxart has produced the tablets for its Phase II and Phase Ib COVID-19 studies and is starting to produce norovirus vaccine for additional clinical studies.
Financial Results for the Three Months Ended September 30, 2021

Vaxart ended the quarter with cash, cash equivalents and available-for-sale debt securities of $204.0 million, compared to $198.9 million as of June 30, 2021. The increase was primarily due to net receipts of $20.3 million from the Company’s $250 million at-the-market facility entered into in October 2020 and $0.3 million from the exercise of warrants and options, partially offset by $14.2 million of cash used in operations and $1.3 million spent on property and equipment.
The Company reported a net loss of $17.6 million for the third quarter of 2021, compared to $8.1 million for the third quarter of 2020. Net loss per share for the third quarter of 2021 was $0.14, compared to a net loss of $0.08 per share in the third quarter of 2020. The increase in net loss per share was primarily due to the increase in net loss, resulting from a significant increase in research and development expenses.
Revenue for the third quarter of 2021 was $200,000, compared to $265,000 in the third quarter of 2020. The decrease was due to lower royalty revenue from sales of Inavir in Japan.
Research and development expenses were $12.4 million for the third quarter of 2021, compared to $4.6 million for the third quarter of 2020. The increase was mainly due to increases in headcount and related costs and in manufacturing and clinical trial expenses related to our COVID-19 and norovirus vaccine candidates.
General and administrative expenses were $5.0 million for the third quarter of 2021, compared to $4.2 million for the third quarter of 2020. The increase was mainly due to an increase in headcount and related costs.
About Vaxart
Vaxart is a clinical-stage biotechnology company developing a range of oral recombinant vaccines based on its proprietary delivery platform. Vaxart vaccines are designed to be administered using tablets that can be stored and shipped without refrigeration and eliminate the risk of needle-stick injury. Vaxart believes that its proprietary tablet vaccine delivery platform is suitable to deliver recombinant vaccines, positioning the company to develop oral versions of currently marketed vaccines and to design recombinant vaccines for new indications. Vaxart’s development programs currently include tablet vaccines designed to protect against coronavirus, norovirus, seasonal influenza, and respiratory syncytial virus (RSV), as well as a therapeutic vaccine for human papillomavirus (HPV), Vaxart’s first immune-oncology indication. Vaxart has filed broad domestic and international patent applications covering its proprietary technology and creations for oral vaccination using adenovirus and TLR3 agonists.