News

• Proceeds to accelerate clinical development of first-in-class bifunctional EGFR/TGF-β inhibitor, BCA101, for multiple cancer types, including 1L HPV-negative recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC)
• Presented positive interim clinical data at 2023 ASCO Annual Meeting and ESMO Congress 2023 from ongoing Phase 1/1b dose expansion study of BCA101 in combination with pembrolizumab demonstrating clinically meaningful anti-tumor activity and tolerable safety profile in 1L HPV-negative R/M HNSCC
• Financing co-led by Braidwell LP and TPG, with participation from other new and existing leading healthcare investors

• Transaction led by healthcare specialist investors, TCGX and Aisling Capital
• $125 million financing upfront with up to an additional $160 million tied to exercise of warrants
• Net proceeds from financing expected to extend cash runway into late 2025 

  London, UK - August 16, 2023 - COMPASS Pathways plc (Nasdaq: CMPS),  a biotechnology company dedicated to accelerating patient access to evidence-based innovation in mental health, today announced that it has entered into a securities purchase agreement with a select group of healthcare specialist investors for the private placement of (i) 16,076,750 American Depositary Shares (“ADSs”) representing 16,076,750  ordinary shares, and (ii) warrants to purchase up to 16,076,750 ADSs (representing 16,076,750  ordinary shares) (the “Warrants”) at a purchase price of approximately $7.78 per ADS and accompanying Warrant to purchase one ADS. Each Warrant will have an exercise price of $9.93 per ADS, representing a 30% premium to the last sale price. The financing is expected to close on August 18, 2023, subject to customary closing conditions. The transaction is being led by TCGX and Aisling Capital with participation from new and existing institutional investors, including Vivo Capital, RA Capital, Surveyor Capital (a Citadel company), Paradigm BioCapital Advisors LP, Soleus Capital, Armistice Capital, Logos Capital, PFM Health Sciences and Laurion Capital Management, among others. Kabir Nath, Chief Executive Officer of COMPASS Pathways, said, “We are grateful for the support of this group of leading healthcare investors for our work to bring potentially transformative treatment for mental health conditions to patients urgently in need of better options. We thank these investors for their confidence in our rigorous approach to building a strong base of evidence for the potential of COMP360 psilocybin treatment to help people with treatment-resistant depression (TRD), post-traumatic stress disorder and anorexia nervosa. We expect that the net proceeds will allow us to advance our pivotal phase 3 program in TRD and achieve important milestones in the development of COMP360. We view this investment as a validation of the potential of psychedelic medicine and the importance of a rigorous and evidence-based approach.”

•  Highly statistically significant result observed on primary endpoint with a Win Ratio of 1.8 (p<0.0001)
• 58% of ties in Finkelstein-Schoenfeld (F-S) primary analysis broken by all-cause mortality and frequency of cardiovascular-related hospitalization; statistical significance also achieved on an F-S test with those two parameters alone (p=0.0182)
• Clinically meaningful and consistent separation observed on all measures of mortality, morbidity, function, and quality of life
• On-treatment survival rate of 81% versus placebo survival rate of 74% (absolute risk reduction of 6.43%; relative risk reduction of 25%)
• Highly statistically significant relative risk reduction of 50% (p<0.0001) observed on frequency of cardiovascular-related hospitalization
• Highly statistically significant and clinically meaningful treatment benefit observed at 30 months on the secondary endpoints of NT-proBNP (p<0.0001), KCCQ (p<0.0001), and 6-minute walk distance (p<0.0001)
• In comparative exploratory post hoc analyses enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed 42% greater increase in serum TTR levels and a 92% improvement in median NT-proBNP relative to placebo + tafamidis
• No safety signals of potential clinical concern identified
• Company intends to file a New Drug Application (NDA) with the U.S. Food and Drug Administration by end of 2023; late-breaker presentation has been accepted for annual meeting of the European Society of Cardiology

Palo Alto, CA - July 17, 2023 - BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), is a commercial-stage biopharmaceutical company focused on genetic diseases and cancers. Today, alongside the dedicated physicians and courageous patients who participated, the Company reports positive results from ATTRibute-CM, its Phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). BridgeBio will host an investor call on July 17, 2023 at 8:00 am ET to discuss these results. “The outstanding results of the ATTRibute-CM study provide new hope to patients living with transthyretin amyloid cardiomyopathy, or ATTR-CM”, said Dr. Daniel Judge, Professor of Medicine and Cardiology at the Medical University of South Carolina, and Co-Chair of the ATTRibute-CM Steering Committee. “The consistent and clinically meaningful benefits on survival, hospitalization, and additional measures of illness severity are truly remarkable.” “ATTR-CM is an increasingly recognized cause of heart failure. The results from BridgeBio’s ATTRibute-CM trial are very exciting and bring much hope to amyloidosis patients and their loved ones,” said Muriel Finkel, President of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers.

• Acquisition of Spyre and concurrent oversubscribed $210 million private investment positions the company to advance a leading inflammatory bowel disease (IBD) portfolio
• Spyre, the second spinout from Paragon Therapeutics, is advancing a pipeline of product candidates, led by SPY001 and SPY002, that are potentially best-in-class antibodies targeting a 4 b 7 and TL1A, respectively, and which are expected to enter the clinic in 2024
• Combined company expected to have approximately $220 million in cash or cash equivalents at close: expected to fund operations into 2026

AUSTIN, TX and WALTHAM, MA - June 22, 2023 - Aeglea BioTherapeutics, Inc. ("Aeglea") (NASDAQ: AGLE), today announced it has completed the acquisition of Spyre Therapeutics, Inc. ("Spyre"), a privately held biotechnology company advancing a robust pipeline of antibody therapeutics with the potential to transform the treatment of inflammatory bowel disease (IBD). Concurrent with the acquisition of Spyre, Aeglea entered into a definitive agreement for the sale of Series A non-voting convertible preferred stock (the "Series A preferred stock") in a private placement to a group of institutional accredited investors led by Fairmount Funds Management LLC ("Fairmount Funds"), with participation from Fidelity Management & Research Company, Venrock Healthcare Capital Partners, Commodore Capital, Deep Track Capital, Perceptive Advisors, RTW Investments, Cormorant Asset Management, Driehaus Capital Management, Ecor1 Capital, RA Capital Management, Surveyor Capital (a Citadel company), and Wellington Management Company LLP, as well as additional undisclosed institutional investors. The private placement is expected to result in gross proceeds to Aeglea of approximately $210 million before deducting placement agent and other offering expenses. The proceeds from the private placement are intended to be used to advance Spyre's portfolio of potentially best-in-class IBD products through multiple data milestones and are expected to fund operations into 2026.

Redwood City, CA -- March 28, 2023 -- Biomea Fusion, Inc. (“Biomea”) (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, today announced initial positive topline data for the first two cohorts of patients with type 2 diabetes mellitus (T2DM) enrolled in the Phase II portion of its ongoing Phase I/II clinical study (COVALENT-111) of BMF-219, the company’s novel, investigational covalent menin inhibitor.

• In Cohort 3, after 4 weeks of once-daily 100 mg dosing with the investigational, oral covalent menin inhibitor, BMF-219, 89% of patients achieved a reduction in A1c, 78% of patients achieved at least a 0.5% reduction in A1c, and 56% achieved at least a 1% reduction in A1c.
• Initial observations of continued glycemic control were seen in follow up visits in patients that had already reached week 8 in the study (4 weeks after the last BMF-219 dose) at the time of this publication.
• BMF-219 demonstrated a well-tolerated safety profile. No patients on BMF-219 discontinued dosing and all patients completed 4 weeks of treatment.
• Biomea continues dose escalation of BMF-219 in COVALENT-111 and plans to explore additional dosing periods greater than 4 weeks in order to evaluate the optimal duration of glycemic control.
• Biomea plans to explore the potential clinical utility of BMF-219 in other diabetic patient populations, including type 1 diabetes.
• Biomea to host conference call on Tuesday, March 28^th at 8:30 AM EDT.

Palo Alto, CA -- March 06, 2023 -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive results from PROPEL2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating potential best-in-class efficacy and a clean safety profile. Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source. BridgeBio will also host an investor call on March 6, 2023, at 7:30 am ET to discuss the results from the Phase 2 study. To date, key results from the clinical trial include:

• At the highest dose level evaluated to date (Cohort 5, 0.25 mg/kg once daily), the mean increase from baseline in annualized height velocity (AHV) for the 10 children that have had six-month visits was +3.03 cm/yr (p = 0.0022). Individual data can be found in Figure 1 below
• The baseline AHV for the 10 children with six-month visits was in the expected range for children with achondroplasia at 3.73 cm/yr, rising to 6.77 cm/yr after treatment
• The two remaining children who have not yet had six months of follow-up have a mean change from baseline in AHV of +8.8 cm/yr at three months. The mean age for the cohort was 7.24 years
• 80% of the 10 children with six-month visits were responders, with a change from baseline AHV of at least 25%. Among the responders, the average change from baseline in AHV was +3.81 cm/yr
• Preliminary analysis of Collagen X (CXM) levels also saw a statistically significant increase from baseline in Cohort 5 (p=.03). CXM is the gold-standard biomarker of chondrocyte-driven growth and further validates the robust response to infigratinib
• Combined with the previously reported Cohort 4 change from baseline in AHV value of +1.52 cm/yr, the Cohort 5 data demonstrate a strong dose response for infigratinib
• Median follow-up across all cohorts is 71.1 weeks. To date, the study has shown a well-tolerated safety profile, with no study drug related treatment emergent adverse events (TEAEs) in Cohort 5. No serious adverse events (SAEs) or discontinuations due to AEs were reported in any cohort

South San Francisco, CA -- Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today announced that it has entered into a definitive securities purchase agreement for a private placement that is expected to result in gross proceeds of approximately $53.6 million, before deducting offering expenses. The private placement includes participation from new and existing investors, including 5am Ventures, Abingworth, Armistice Capital, HealthCap, Novo Holdings A/S, RiverVest Venture Partners, and Rock Springs Capital.

CAMBRIDGE, MA.-- Garuda Therapeutics (Garuda), a company creating off-the-shelf, durable blood stem cell-based cellular therapies, today announced a $62 million Series B financing led by Northpond Ventures, OrbiMed Advisors, Cormorant Asset Management, and Aisling Capital, along with participation from Sectoral Asset Management, Mass General Brigham Ventures, Alexandria Venture Investments, and other elite investors and individuals. This brings the ​​total amount of funding for the company to $134 million. The company also announced the appointment of Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant Asset Management, to its Board of Directors. Like bone marrow transplants, Garuda’s technology has the potential to address, and possibly cure, more than 120 diseases for a diverse population of patients with unmet medical need. Proceeds from the financing will be used to further advance the development of Garuda’s platform for generation of off-the-shelf, self-renewing blood stem cells towards the clinic as potential treatment for patients with hematological diseases. The Series B funding will also enable Garuda to continue the advancement of its proprietary HSC-derived off-the-shelf, durable immune cell program for treating patients with oncologic disorders. The funding will also further advance Garuda’s continued development of HLA-matched pluripotent stem cells greatly broadening the eligibility of patients and increasing access to blood and other cellular therapies in regenerative medicine.

San Francisco, CA & TOKYO - Jan 5, 2023 -  Spruce Biosciences, Inc. (Nasdaq: SPRB) and Kaken Pharmaceutical Co. Ltd (Tokyo Stock Exchange: 4521) today announced that the companies have entered into an exclusive licensing agreement for the development and commercialization of Spruce’s product candidate, tildacerfont, for the treatment of congenial adrenal hyperplasia (CAH) in Japan. Under the terms of the agreement, Spruce will receive an upfront payment of $15 million from Kaken and will be eligible to receive additional payments upon the achievement of future development and commercial milestones, as well as tiered double-digit royalties on net sales in Japan. Kaken will be responsible for the clinical development and commercialization of tildacerfont in Japan, and Spruce will retain all rights to tildacerfont in all other geographies. As part of the agreement, Kaken will have the first right of negotiation to expand the scope of the agreement to include China (including Hong Kong, Taiwan, and Macau), South Korea and other specified Southeastern Asian (ASEAN) countries. Kaken will also be responsible for securing and maintaining regulatory approvals necessary to market and sell tildacerfont in Japan.

LONDON and RALEIGH, NC - December 20, 2022 - Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), today announces positive results of its Phase 3 ENHANCE-1 trial evaluating nebulized ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease (“COPD”). The ENHANCE-1 trial successfully met its primary and key secondary endpoints demonstrating significant improvements in lung function, symptoms and quality of life measures. In addition, ensifentrine substantially reduced the rate and risk of COPD exacerbations. Ensifentrine was well tolerated over 24 and 48 weeks.

Ensifentrine is a first-in-class, selective dual inhibitor of the enzymes phosphodiesterase 3 and 4 combining bronchodilator and non-steroidal anti-inflammatory activities in one compound.

Highlights

• Study population (n=763):
  • Subject demographics and disease characteristics were well balanced between treatment groups.
  • Approximately 66% of subjects received background COPD therapy, either a long-acting muscarinic antagonist (“LAMA”) or a long-acting beta-agonist (“LABA”). Additionally, approximately 21% of all subjects received inhaled corticosteroids (“ICS”) with concomitant LAMA or LABA.
• Primary endpoint met (FEV₁* AUC 0-12 hr):
  • Placebo corrected, change from baseline in FEV₁ area under the curve 0-12 hours post dose at week 12 was 87 mL (p<0.0001) for ensifentrine.
  • Demonstrated consistent improvements in all subgroups including gender, age, smoking status, COPD severity, background medication, ICS use, chronic bronchitis, FEV₁ reversibility and geographic region.
• Key secondary endpoints of lung function, symptoms and quality of life measures met:
  • Placebo corrected, increase in peak FEV₁ of 147 mL (p<0.0001) 0-4 hours post dose at week 12.
  • Daily symptoms as measured by E-RS** Total Score in the ensifentrine group improved from baseline to greater than the minimal clinically important difference (“MCID”) of -2 units with a statistically significant improvement compared to placebo at week 24. Improvements in symptoms were early and sustained with statistical significance versus placebo at weeks 6, 12 and 24.
  • Quality of Life (“QOL”) as measured by SGRQ** Total Score in the ensifentrine group improved from baseline to greater than the MCID of -4 units with a statistically significant improvement compared to placebo at week 24. Improvements in QOL were early and sustained with statistical significance versus placebo at weeks 6, 12 and 24.
  • Placebo corrected, increase in morning trough FEV₁ of 35 mL (p=0.0421) at week 12, supporting twice daily dosing regimen.
• Exacerbation rate and risk reduced:
  • Subjects receiving ensifentrine demonstrated a 36% reduction in the rate of moderate to severe COPD exacerbations over 24 weeks (p=0.0505) compared to those receiving placebo.
  • Treatment with ensifentrine significantly decreased the risk of a moderate/severe exacerbation as measured by time to first exacerbation when compared with placebo by 38% (p=0.0378).
  • Pooled exacerbation data from ENHANCE-1 and ENHANCE-2, ensifentrine demonstrated a 40% reduction in the rate of moderate to severe COPD exacerbations over 24 weeks (p=0.0012) compared to those receiving placebo. Additionally, ensifentrine significantly decreased the risk of a moderate/severe exacerbation as measured by time to first exacerbation when compared with placebo by 41% (p=0.0008).
• Favorable safety profile:
  • Ensifentrine was well-tolerated with very few events occurring in more than 1% of subjects and greater than placebo over 24 and 48 weeks.