News

• $40 million upfront including $25 million cash and $15 million in equity in Nuance Biotech (parent company)
• Up to $179 million in potential clinical, regulatory, and commercial milestone payments plus tiered double-digit royalties
• Nuance Pharma is responsible for all costs related to development and commercialization of ensifentrine in China

LONDON and RALEIGH, N.C. and SHANGHAI, China - June 10, 2021 - Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma”) and Nuance Pharma Limited (“Nuance Pharma”), today announce that the companies have entered into an agreement granting Nuance Pharma, a Shanghai-based specialty pharmaceutical company, the rights to develop and commercialize ensifentrine in Greater China (mainland China, Taiwan, Hong Kong and Macau). Ensifentrine is an investigational, first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 (“PDE3” and “PDE4”). This dual inhibition enables it to combine both bronchodilator and anti-inflammatory effects in one compound. Verona Pharma is currently conducting a global Phase 3 program evaluating ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease (“COPD”), with sites in the US, Europe and South Korea.

“We are extremely excited about the formation of this strategic partnership with Nuance Pharma to further the development and future commercialization of ensifentrine in Greater China,” said David Zaccardelli, Pharm. D., President and CEO of Verona Pharma. “Nuance Pharma’s highly talented leadership team has deep experience developing and commercializing respiratory products across China and we look forward to working with them to bring ensifentrine to this important market.”

• TI-1777 Achieved Statistically Significant Result in the Primary Efficacy Endpoint at Week 4
• Minimal Systemic Exposure Supports “Soft” Topical JAK Inhibitor Approach
• ATI-1777 was Generally Well Tolerated
• Data Support Progression to Phase 2b

WAYNE, PA -  June 08, 2021 - Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced positive preliminary topline results from its first in human Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-group clinical trial to evaluate the efficacy, safety, tolerability and pharmacokinetics of ATI-1777, an investigational topical “soft” JAK 1/3 inhibitor, in 50 subjects with moderate to severe atopic dermatitis (AD) (ATI-1777-AD-201). ATI-1777 is the second compound generated from Aclaris’ proprietary KINect^® drug discovery platform to demonstrate positive proof of concept in clinical trials. “We are very pleased that we achieved positive results in this trial of ATI-1777 in subjects with moderate to severe AD with minimal systemic exposure to drug,” said Dr. David Gordon, Chief Medical Officer at Aclaris. “Our approach to treating patients with moderate to severe atopic dermatitis is particularly relevant in light of some of the potential safety concerns with oral therapies.   We look forward to advancing ATI-1777 into the next phase of clinical development.” In the trial, which consisted of a 4-week treatment period and a 2-week follow-up period during which no treatment was given, 50 subjects with moderate to severe AD were randomized in a 1:1 ratio into one of two arms: ATI-1777 topical solution 2.0% w/w or vehicle applied twice daily. One of the key objectives of this first in human trial was to assess the “soft” aspect of this topical JAK inhibitor compound in subjects with moderate to severe atopic dermatitis. A preliminary analysis of pharmacokinetic plasma samples in the ATI-1777 arm showed greater than 86% of the plasma samples had concentrations below 1 ng/ml and mean drug levels in the ATI-1777 arm (as a group) were not greater than 5% of the IC50 of ATI-1777. The primary efficacy endpoint of this trial was the percent change from baseline in the modified Eczema Area and Severity Index (mEASI) score at week 4. The mEASI is a modified measure of EASI which excludes evaluation of the body areas that were not treated in the trial (i.e., the head, palms of hands, soles of feet, groin, or genitalia). Only the primary efficacy endpoint was powered to detect a statistically significant outcome.

• Pivotal study demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response in patients with previously-treated advanced cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement
• BridgeBio, through its affiliate QED (“BridgeBio”), and Helsinn will co-commercialize TRUSELTIQ in the U.S.
• TRUSELTIQ is BridgeBio’s first FDA approved therapeutic in oncology and second approved therapeutic this year

PALO ALTO, CA and LUGANO, Switzerland -  May 28, 2021 -  BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., and Helsinn Group today announced that the US Food and Drug Administration (FDA) has approved TRUSELTIQ™ (infigratinib) under the accelerated approval program for the treatment of patients with previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. TRUSELTIQ is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. In the pivotal trial of patients with advanced, unresectable CCA, an aggressive malignancy with poor prognosis, TRUSELTIQ led to cases of tumor shrinkage. CCA is known to affect approximately 20,000 people in the United States and European Union each year and has a median five-year survival rate of only 9%.¹ “This is an important milestone for patients diagnosed with FGFR2-fusion-driven cholangiocarcinoma who have recurred after first-line therapy and are in need of targeted options for further treatment,” said Susan Moran, M.D., M.S.C.E., Chief Medical Officer for QED. “Based on the efficacy seen to date, our team believes infigratinib possesses promise for a range of FGFR-driven conditions, including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need.”

• Preclinical Data for Congenital Adrenal Hyperplasia and Canavan Disease Programs Shared at American Society of Gene & Cell Therapy Annual Meeting, Enabling Anticipated Clinical Trials This Year

PALO ALTO, CA – May 14, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to BBP-631, an investigational adeno-associated virus 5 (AAV5) gene therapy designed for the treatment of congenital adrenal hyperplasia (CAH).Fast Track designation, granted by the FDA, is designed to facilitate the development and to expedite the review of new therapies hoping to treat or prevent serious conditions and fill an unmet medical need. Additionally, BBP-631 was granted Rare Pediatric Disease Designation by the FDA and has received Orphan Drug Designation by the FDA and European Medicines Agency (EMA).“The standard-of-care for CAH patients has not changed significantly over the last 50 years, and a gene therapy offers for the first time the possibility that patients may be able to make their own cortisol and aldosterone, at the right times and in the right amounts. The FDA’s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible. We are grateful to have received Fast Track along with other key designations granted by the FDA and the EMA,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy, which is focused on developing gene therapy treatment options for patients in need. “We are eager to initiate our first-in-human Phase 1/2 study. The Investigational New Drug application has been cleared by the FDA and site activation for the study is ongoing, and is expected to begin in the coming months.”CAH is one of the most prevalent genetic diseases with more than 75,000 cases estimated in the United States and Europe. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol and aldosterone production. This lack of production causes patients with CAH to be unable to form physiological responses to illnesses and stressors, which can be life-threatening, especially for children. BBP-631 is designed to provide a functional copy of the 21-hydroxylase-encoding gene to help patients produce their own cortisol and aldosterone. Additionally, BridgeBio Gene Therapy presented preclinical data from its CAH program on May 13, 2021 at the American Society of Gene & Cell Therapy (ASGCT) annual meeting, being held virtually May 11-14, 2021. The presentation covered three key Investigational New Drug (IND)-enabling studies that informed the anticipated upcoming clinical trial for BBP-631, which is expected to begin in late 2021. The IND application has been 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 late 2021 or early 2022.  At ASGCT, BridgeBio Gene Therapy today will also be presenting preclinical data from its program designed for the treatment of Canavan disease, which is an extremely rare genetic disease starting in infancy with an incidence of approximately one in 100,000 births worldwide. “ASGCT is an important opportunity to share the robust preclinical data package underlying our programs. We hope that by rapidly advancing our investigational therapies into the clinic, we will be one step closer to providing CAH and Canavan patients with potential therapeutic relief,” said Clayton Beard, senior vice president of research and development at BridgeBio Gene Therapy.

BOSTON and LOUISVILLE, KY - May 11, 2021 - Talaris Therapeutics, Inc. (Nasdaq: TALS), a late-clinical stage cell therapy company developing therapies with the potential to transform the standard of care in solid organ transplantation, certain severe autoimmune diseases, and certain severe non-malignant blood, immune and metabolic disorders, today announced the closing of its initial public offering of 8,825,000 shares of its common stock at a price to the public of $17.00 per share. The gross proceeds of the offering were approximately $150.0 million, before deducting underwriting discounts and commissions and other offering expenses. The shares began trading on the Nasdaq Global Market on May 7, 2021 under the symbol “TALS.” All shares in the offering were offered by Talaris Therapeutics. In addition, the underwriters have a 30-day option to purchase up to an additional 1,323,750 shares of common stock at the initial public offering price less underwriting discounts and commissions.

Morgan Stanley, SVB Leerink, Evercore ISI and Guggenheim Securities are acting as joint book-running managers for the offering. A registration statement relating to these securities has been filed and was declared effective by the Securities and Exchange Commission on May 6, 2021. This offering was made only by means of a written prospectus. Copies of the final prospectus relating to the initial public offering can be obtained, when available, from: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com; Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, or by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com; and Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-5548, or by email at GSEquityProspectusDelivery@guggenheimpartners.com.  

REDWOOD CITY, CA - April 20, 2021 - Biomea Fusion, Inc. (“Biomea”) (Nasdaq: BMEA), a preclinical-stage biopharmaceutical company focused on the discovery, development and commercialization of irreversible small molecules to treat patients with genetically defined cancers, today announced the closing of its initial public offering of 9,000,000 shares of its common stock at a public offering price of $17.00 per share. All of the shares of common stock were offered by Biomea. Biomea’s common stock began trading on The Nasdaq Global Select Market on April 16, 2021 under the ticker symbol “BMEA.” The gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Biomea, were $153.0 million. In addition, Biomea has granted the underwriters a 30-day option to purchase up to an additional 1,350,000 shares of common stock at the initial public offering price, less the underwriting discounts and commissions and offering expenses.

MENDHAM, N.J. - April 12, 2021  – Antios Therapeutics, Inc. (“Antios”) today announced the successful completion of a $96 million Series B financing. The proceeds from this financing will support the ongoing Phase 2 clinical program which is evaluating the potential for the Company’s lead clinical candidate, ATI-2173, to be a backbone of a once-daily curative regimen for chronic hepatitis B. ATI-2173 is a novel, orally administered, liver-targeted Active Site Polymerase Inhibitor Nucleotide (ASPIN) that can shut down HBV polymerase activity and viral replication. The financing was led by Soleus Capital with participation from new major investors RA Capital Management, Adage Capital Management LP, Pontifax and Aisling Capital as well as other healthcare focused funds, Altium Capital, Amzak Health, Granite Point Capital Management, LP, and LifeSci Venture Partners and participation from all of the original Series A investors including Lumira Ventures, CAM Capital, Delos Capital, Domain Associates and Sixty Degree Capital. David Canner, Partner at Soleus Capital and Iyona Rajkomar, MBBS, CFA, Partner at Pontifax, are joining the Board of Directors. “ATI-2173 has already demonstrated potent on-treatment and durable off-treatment effects in our Phase 1b study in patients with chronic hepatitis B,” said Greg Mayes, Chief Executive Officer of Antios. “Those results will be presented at an upcoming major medical conference. With this financing we are now well positioned to continue to highlight how ATI-2173 may become the backbone of a curative regimen for HBV which remains a significant unmet global public health need. We look forward to working with new and existing investors to advance ATI-2173 forward in development.” Guy Levy, Chief Investment Officer of Soleus Capital said, “Despite significant progress in recent years, HBV remains an area of high unmet medical need. Given the mechanistic rationale and impressive early data, we believe ATI-2173 could play an essential role in developing curative regimens for the millions of people suffering from chronic HBV infection. We are excited to partner with the experienced team at Antios to help bring this therapy to patients.”

About ATI-2173

ATI-2173 is a novel, orally administered, liver-targeted Active Site Polymerase Inhibitor Nucleotide (ASPIN) molecule designed to deliver the 5’-monophosphate of clevudine to the liver. This L-nucleoside’s active 5’-triphosphate has unique antiviral properties as a non-competitive, non-chain terminating HBV polymerase inhibitor that distorts the active site of HBV polymerase resulting in potent HBV antiviral activity and extended off-treatment suppression of HBV DNA. ATI-2173 targets the liver, delivering high levels of the unique 5’- triphosphate while limiting systemic exposure to the parent L-nucleoside. ATI-2173 has the potential to become an integral part of a curative combination regimen for chronic hepatitis B.

SAN DIEGO, CA - April 08, 2021 - Reneo Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare, genetic, mitochondrial diseases, today announced the pricing of its initial public offering of 6,250,000 shares of its common stock at a public offering price of $15.00 per share, for total gross proceeds of approximately $93.8 million, before deducting underwriting discounts and commissions and offering expenses. All of the shares are being offered by Reneo. The shares are expected to begin trading on the Nasdaq Global Market on April 9, 2021 under the symbol “RPHM.” In addition, Reneo has granted the underwriters a 30-day option to purchase up to an additional 937,500 shares of common stock at the public offering price less underwriting discounts and commissions. The offering is expected to close on April 13, 2021, subject to satisfaction of customary closing conditions. Jefferies, SVB Leerink and Piper Sandler are acting as joint book-running managers for the offering. A registration statement relating to these securities has been filed with the Securities and Exchange Commission and became effective on April 8, 2021. The offering is being made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained, when available, from: Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388 or by e-mail at prospectus_department@jefferies.com; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA, 02110, by telephone at (800) 808-7525, ext. 6105 or by e-mail at syndicate@svbleerink.com; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by e-mail at prospectus@psc.com. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. About Reneo Pharmaceuticals Reneo is a clinical stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, which are often associated with the inability of mitochondria to produce adenosine triphosphate (ATP). Reneo is developing REN001 to modulate genes critical to metabolism and generation of ATP, which is the primary source of energy for cellular processes. REN001 has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation, and may increase production of new mitochondria.

BOSTON, MA - March 12, 2021 - Monte Rosa Therapeutics, a biotechnology company focused on discovering and developing precision medicines that degrade disease-causing proteins, announced today the closing of a $95 million Series C financing. The proceeds will be used to advance its lead development candidate into the clinic, accelerate pipeline growth and bolster its platform capabilities to rationally design and develop small molecule degraders (also known as molecular glues) that hijack the body’s innate ability to degrade proteins. Through this approach, Monte Rosa aims to eradicate undruggable proteins that cause or drive the progression of genomically-defined diseases intractable to standard care, including cancer. The financing was led by Avoro Capital Advisors with participation from additional new investors Fidelity Management & Research Company LLC, funds and accounts managed by BlackRock, funds and accounts advised by T. Rowe Price Associates, Inc., and RTW Investments, LP. Monte Rosa’s founding investor Versant Ventures participated in the round, as did additional existing investors New Enterprise Associates, Aisling Capital, Cormorant Asset Management, HBM Healthcare Investments, GV, Amzak Health, Sixty Degree Capital, Casdin Capital and Cambridge Asset Management. “By leveraging the power of nature’s protein degradation pathways, our next-generation approach is purpose-built to target the undruggable proteome and eliminate disease-driving proteins with high precision,” said Markus Warmuth, M.D., CEO of Monte Rosa. “With extensive and compelling in vivo data in hand, we are confident in the potential of our molecular glue-based targeted protein degradation platform to deliver breakthrough small molecule therapeutics. As we prepare to initiate IND-enabling studies on our lead candidate later this year, and with additional programs transitioning into lead optimization, we are thrilled to have the continued support of such a strong group of new and existing investors who share in our vision. We are excited to build on our early successes to further expand our platform, targeting disease-causing proteins that have been impossible to drug across multiple therapeutic indications.” “Molecular glue degraders represent a powerful and differentiated approach to eradicating disease-causing proteins,” said Behzad Aghazadeh, Ph.D., Managing Partner, Avoro Capital Advisors. “Coupled with Monte Rosa’s world-class team and drug discovery platform enabled by their machine learning-based prediction of degrons, the company is poised to rapidly expand the previously undruggable target universe and foster a new generation of therapeutics.”

FREMONT, CA - February 01, 2021 - Zosano Pharma Corporation (NASDAQ:ZSAN), a clinical-stage biopharmaceutical company, today announced that the company completed its Type A meeting with the U.S. Food and Drug Administration (“FDA") Division of Neurology II (the “Division”) regarding the requirements for resubmission of the Qtrypta™ (zolmitriptan transdermal microneedle system) 505(b)(2) New Drug Application (“NDA”) following the Complete Response Letter received on October 20, 2020.

Based on feedback from the Type A meeting held with the Division, the company plans to conduct an additional pharmacokinetic (“PK”) study for inclusion in an NDA resubmission package. During the meeting, the Division did not request that the company conduct any further clinical efficacy studies to support the resubmission. Prior to initiating the PK study, the company plans to submit the study protocol to the Division for additional comment and review. The Division indicated willingness to review the study protocol and provide comments prior to the initiation of the study. The company’s plans for resubmitting the NDA are based on the discussions between the company and the Division during the Type A meeting and may be subject to change upon receipt of the FDA’s official meeting minutes from the Type A meeting. “We appreciate the FDA’s willingness to discuss our application, and the Division providing confirmation that an additional PK study will be necessary to resubmit the NDA for Qtrypta,” said Steven Lo, President and CEO of Zosano. “We are working to have a protocol for the PK study finalized this quarter. We look forward to initiating and completing the study, and ultimately resubmitting our NDA application. Qtrypta leverages our proprietary microneedle technology that has been supported by data from five trials in 774 subjects, and we are encouraged by its potential to treat patients with debilitating migraines.”