On November 4, 2021 Primmune Therapeutics reported that it has received $8.4 million in a second tranche of the Company’s Series A financing. The total proceeds for the equity raised in the Series A was $31.4 million (Press release, Primmune Therapeutics, NOV 4, 2021, View Source [SID1234594568]). These funds will be used to support the further clinical development of PRTX007 as a TherAjuvant for acute viral diseases, pre-cancerous lesions, and advanced cancer. PRTX007 is a novel orally-administered, small molecule toll-like receptor 7 (TLR7) agonist that has both therapeutic and adjuvant properties.

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"Given the initial results from our Phase 1 study in healthy volunteers, we achieved our target clinical milestone that triggered a second tranche of $8.4 million from our existing investors. These funds will be used to set the stage for Primmune’s expansion into multiple definitive efficacy studies," said Charlie McDermott, Chairman and Chief Executive Officer of Primmune Therapeutics. "In 2022, we intend to study PRTX007 in ambulatory respiratory syncytial virus (RSV), outpatient SARS-CoV-2, human papilloma virus (HPV) driven high-grade squamous intraepithelial lesions (HSIL) of the cervix, and in the neo-adjuvant setting in combination with checkpoint inhibitors in advanced cancer."

About TherAjuvants
Primmune Therapeutics coined the term TherAjuvants to reference its lead candidate PRTX007, a toll-like receptor 7 (TLR7) agonist with a combination of therapeutic and adjuvant mechanisms of action. PRTX007 is designed to provide immediate benefit to patients through controlled stimulation of the innate immune response while also potentiating long-term effective innate and adaptive immune responses. PRTX007 uniquely engages TLR7 and targeted immune cells without exacerbating inflammation, a critical feature in treating respiratory viral infections. TherAjuvants differ from therapeutic vaccines in that the source of the antigens presented to the patient’s immune system come from the treated pathology. Additionally, TherAjuvants differ from most small molecule approaches in that they target the patient’s immune system and not tumor cells or virally encoded targets.

On November 4, 2021 Plexium Inc., (Plexium), a leading next-generation targeted protein degradation (TPD) company, reported the appointment of experienced biopharmaceutical leader Percival Barretto-Ko as President and Chief Executive Officer and a member of the Board of Directors (Press release, Plexium, NOV 4, 2021, View Source [SID1234594567]).

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Mr. Barretto-Ko joins Plexium following a sixteen-year tenure with Astellas Pharma Inc., a global pharmaceutical company, where he held several prominent roles and was most recently Chief Business Officer and a member of the Executive Committee where he led the execution of Astellas’ Corporate Strategic Plan.

"Percival is a highly accomplished veteran with the leadership, track record and breadth of experience that make him uniquely suited to lead Plexium," said Mike Grey, Executive Chairman of Plexium. "I look forward to working with Percival to advance the company’s promising drug discovery programs against challenging targets utilizing Plexium’s best-in-class technology."

"I am delighted to join Plexium at this exciting stage, and help achieve its aspiration to be the premier, next generation TPD company. Plexium is well-positioned for success, with its comprehensive proprietary platform, novel approach, top-tier investors, and a world-class team, to create TPD therapies that address the limitations of PROTACs and cereblon," said Mr. Barretto-Ko. "I look forward to advancing the important and innovative work we’re doing to advance protein degraders as a new class of medicines with the potential to treat a wide range of diseases."

Prior to serving as CBO for Astellas Pharma, Mr. Barretto-Ko was President, Astellas Pharma US, where he was responsible for United States operations with annual revenues of over $4.5 billion and more than 3,000 employees. Mr. Barretto-Ko’s earlier roles at Astellas include Senior Vice President, Head of International, and Senior Vice President, Corporate Strategy, Business Development & Government Affairs. Mr. Barretto-Ko joined Astellas in 2005 in London where he served in several roles including EMEA Head of Corporate Strategy, Business Development, and Corporate Communications, and EMEA Head of Immunology and Transplant. Before joining Astellas, Mr. Barretto-Ko held increasingly senior commercial positions with the Roche Group in the United States and United Kingdom, across various therapeutic areas including Oncology, Hematology, Virology, Immunology, and Infectious Diseases.

Mr. Barretto-Ko earned a Bachelor of Science in Biological Sciences with Honors from Cornell University and a Master of Business Administration from Yale University. In addition, Mr. Barretto-Ko served as a Sloan Fellow for Innovation and Global Leadership at the MIT Sloan School of Management, where he was awarded a Master of Science degree in Management. He is a board member of Americares, a health-focused relief and development organization that serves people affected by poverty or disaster with life-changing medicine and health programs.

On November 4, 2021 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported that Maurice Zauderer, Ph.D., President and Chief Executive Officer, is scheduled to present virtually at the 12th Annual Jefferies London Healthcare Conference and invites investors to participate in virtual one-on-one meetings on Thursday and Friday, November 18th and 19th (Press release, Vaccinex, NOV 4, 2021, View Source [SID1234594566]). Please see Presentation details below:

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12th Annual Jefferies London Healthcare Conference
Date: November 18th to 19th, 2021
Time: Virtual presentation available Thursday, November 18 at 8:00am GMT/3:00am ET through Friday, November 19 at 5:00pm GMT / 12:00pm ET
Presenter: Maurice Zauderer, Ph.D., President and Chief Executive Officer
Webcast Link: View Source;group=TbkBHEif
Replay: The presentation and a replay of the webcast will be available on Vaccinex’s website shortly after the event at: View Source
Please contact your Jefferies representative or Vaccinex if you would like to participate in a virtual one-on-one meeting during the conference.

For more information about the Jefferies London Healthcare Conference, please refer to the Jefferies conference website. Vaccinex’s presentation will also be available on the Jefferies website for 30 days after the conference.

On November 4, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported financial results for the third quarter ended September 30, 2021, and recent operational highlights (Press release, Intellia Therapeutics, NOV 4, 2021, View Source [SID1234594565]).

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"Intellia continues to make important progress toward advancing its full-spectrum genome editing pipeline. Today, we are pleased to share that we remain highly encouraged by the safety profile, consistency of TTR reduction and ongoing effect of NTLA-2001 in the Phase 1 study. We believe we are closing in on identifying the recommended therapeutic dose for NTLA-2001 in patients with ATTR amyloidosis with polyneuropathy for further evaluation in Part 2, a single-dose expansion cohort. Additionally, while it has long been our plan to develop NTLA-2001 for all forms of ATTR amyloidosis, we have decided to accelerate the evaluation of NTLA-2001 in patients with ATTR amyloidosis whose primary clinical manifestation is cardiomyopathy. Based on the strength of our interim dataset, we are now seeking regulatory feedback for inclusion of the ATTR-CM patient population in our current Phase 1 study. We look forward to sharing updates from this program in Q1 2022," said Intellia President and Chief Executive Officer John Leonard, M.D. "Beyond our lead program, we remain focused on advancing NTLA-2002 for HAE and NTLA-5001 for AML. We received regulatory clearance for both programs to initiate first-in-human studies and we expect those studies to begin later this year. Finally, we nominated two gene insertion development candidates – NTLA-3001, our wholly owned AATD program, and a Factor 9 gene insertion candidate for Hem B in collaboration with Regeneron. Our rapidly expanding pipeline is yet one more example of the benefits of our modular platform in generating novel and potentially curative treatment options for the patients we aim to serve."

Third Quarter 2021 and Recent Operational Highlights

In Vivo Program Updates

NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first systemically delivered CRISPR-based therapy to be dosed in a patient and has the potential to be a curative treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
NTLA-2001 is completing the dose-escalation portion of the Phase 1 study to determine the recommended dose for evaluation in Part 2 of the study, a single-dose expansion cohort. During the third quarter, to more fully elucidate the dose-response relationship, Intellia began dosing of subjects in Cohort 4, evaluating NTLA-2001 in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) at the 0.7 mg/kg dose level.
Intellia accelerated the evaluation of NTLA-2001 for the treatment of patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The Company is in discussions with regulatory authorities on a protocol amendment to expand the Phase 1 trial population to include patients with ATTR-CM.
Intellia now plans to present interim data from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
In October, Intellia announced that NTLA-2001 received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of ATTR amyloidosis.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of hereditary angioedema (HAE). This approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In October, Intellia announced the authorization of its Clinical Trial Application (CTA) by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002. Additionally, a CTA has been subsequently authorized by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) for the first-in-human study of NTLA-2002.
Intellia intends to enroll the first patient in the first-in-human study by year-end. The Phase 1/2 study will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE, and will leverage insights gained from the development of NTLA-2001 enabling this study to begin evaluating NTLA-2002 at a higher initial dose.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s first and wholly owned CRISPR/Cas9-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to permanently restore expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or transplant in severe cases.
Today, Intellia announced the nomination of a new development candidate, NTLA-3001 for treatment of AATD. At the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), Intellia presented data showing that insertion of a healthy form of the SERPINA1 gene led to normal human A1AT levels in non-human primates (NHPs) which were durable through 52 weeks in an ongoing study. The Company is advancing towards Investigational New Drug (IND)-enabling activities for this program and continues to explore additional editing strategies for AATD.
Ex Vivo Program Updates

NTLA-5001 for AML: NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML).
In September, Intellia announced that the U.S. FDA accepted its IND application for NTLA-5001.
Intellia intends to initiate patient screening by year-end for a Phase 1/2a study evaluating NTLA-5001 in adults with persistent or recurrent AML who have previously received first-line therapy.
Research and Corporate Updates

Modular Platform and Pipeline Expansion: Intellia is advancing its modular platform technologies to broaden the in vivo and ex vivo applications of genome editing. This includes progressing capabilities for innovative CRISPR/Cas9-mediated targeted transgene insertion, in vivo editing in multiple tissue types and an allogeneic approach for the development of "off-the-shelf" T cell therapies. These efforts support new therapeutic candidates and strategic business development partnerships for advancing treatments for genetic diseases, cancers and autoimmune diseases.
ESGCT Annual Congress: In October, Intellia presented new preclinical data demonstrating key platform capabilities.
Intellia shared the first data highlighting its proprietary allogeneic cell engineering platform, demonstrating it can prevent immune rejection of allogeneic T cells for application in TCR-T and CAR-T cell therapy. Intellia’s proprietary approach leverages a novel combination of sequential gene edits to shield the engineered cell therapy from host T and NK cell attack. It does not rely on long-term, aggressive immune suppression of patients or the knockout of a protein required for HLA class I expression, which are approaches currently employed by others to address the challenge of immune rejection of the allogeneic cell product. Intellia intends to nominate its first allogeneic cell therapy development candidate by the first half of 2022.
Intellia shared new data on its proprietary cell engineering process, demonstrating LNP-based delivery of CRISPR/Cas9 ex vivo allows for sequential editing of T cells with high efficiency, faster expansion and minimal translocations as compared to electroporation. The data support the ability of this platform to be used for a variety of targeting modalities, including CAR and TCRs, and to support both autologous and allogeneic T cell candidates. This LNP-based approach is being used for NTLA-5001.
Collaboration Updates:
In the third quarter, Intellia and Regeneron nominated a Factor 9 (F9) gene insertion development candidate for its Hemophilia B (Hem B) program, leveraging their jointly developed targeted transgene insertion capabilities to insert F9. This candidate is part of a co-development/co-funding agreement between Intellia and Regeneron, the lead party for this program. F9 is a gene that encodes for Factor IX (FIX), a blood-clotting protein that is missing or defective in Hem B patients. In preclinical studies, the companies demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of NHPs, which resulted in circulating FIX levels at or above those found in normal human plasma.
In October, the Company announced a strategic collaboration with SparingVision to develop novel genomic medicines utilizing Intellia’s proprietary CRISPR/Cas9 technology for the treatment of ocular diseases. As part of the collaboration, Intellia will receive an equity stake in SparingVision, option for exclusive U.S. commercialization rights for product candidates arising from two of three collaboration targets, and eligibility for development and commercial milestone payments as well as royalties on potential future sales of products arising from the collaboration. The companies will additionally research and develop novel self-inactivating AAV vectors and LNP-based approaches to address delivery of CRISPR/Cas9 genome reagents to the retina.
In July, the Company completed the formation of a new universal CAR-T cell therapy company, which Intellia launched in collaboration with Blackstone Life Sciences and Cellex Cell Professionals GmbH. The new company will combine clinical-stage universal CAR-T platforms with Intellia’s differentiated allogeneic cell engineering platform to develop therapies for immuno-oncology and autoimmune diseases.
Upcoming Events

The Company will participate in the following events during the fourth quarter of 2021:

Barclays Gene Editing & Gene Therapy Summit, November 15, Virtual
Evercore ISI 4th Annual HealthCONx Conference, December 1, Virtual
63rd ASH (Free ASH Whitepaper) Annual Meeting & Exposition, December 11-14, Atlanta, GA and Virtual
Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR:
Report additional interim clinical data from Phase 1 study of NTLA-2001 in Q1 2022
Initiate Part 2, a single-dose expansion cohort, of Phase 1 study of NTLA-2001 in Q1 2022
HAE: Initiate enrollment in the first-in-human study of NTLA-2002 by year-end
AML: Initiate patient screening in the first-in-human study of NTLA-5001 by year-end
Pipeline Expansion: Nominate the Company’s first allogeneic development candidate by 1H 2022
Third Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,148.7 million as of September 30, 2021, compared to $597.4 million as of December 31, 2020. The increase was driven by net proceeds of $648.3 million from a follow-on offering in Q3 2021, $45.3 million of net proceeds from the Company’s "At the Market" (ATM) agreement, $40.8 million in proceeds from employee-based stock plans, and $4.2 million of funding for a cost-sharing agreement received from Regeneron. These increases were offset in part by cash used to fund operations of approximately $187.3 million.
Collaboration Revenue: Collaboration revenue decreased by $15.0 million to $7.2 million during the third quarter of 2021, compared to $22.2 million during the third quarter of 2020. The decrease in collaboration revenue in 2021 was primarily driven by $15.3 million related to the transfer of control of the license to develop the Factor VIII target for hemophilia A that was recorded during Q3 2020.
R&D Expenses: Research and development expenses increased by $20.7 million to $60.5 million during the third quarter of 2021, compared to $39.8 million during the third quarter of 2020. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs, and expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $8.1 million to $18.7 million during the third quarter of 2021, compared to $10.6 million during the third quarter of 2020. This increase was primarily related to employee related expenses, including stock-based compensation of $3.8 million.
Net Loss: The Company’s net loss was $71.6 million for the third quarter of 2021, compared to $27.8 million during the third quarter of 2020.
Conference Call to Discuss Third Quarter Earnings

The Company will discuss these results on a conference call today, Thursday, November 4, at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, beginning on November 4 at 12 p.m. ET.

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.