News

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.

Waltham, MA -- December 14, 2022 -- Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced the closing of its previously announced underwritten public offering of 7,840,909 shares of its common stock at a price to the public of $22.00 per share. This includes the exercise in full by the underwriters of their option to purchase up to 1,022,727 additional shares of common stock. The aggregate gross proceeds to Syndax from this offering were $172.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Syndax. Following the closing of the Offering, Syndax has 68,100,918 shares issued and outstanding as of December 14, 2022.

WALTHAM, MA - December 5, 2022 - Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for revumenib for the treatment of adult and pediatric patients with relapsed or refractory (R/R) acute leukemia harboring a KMT2A rearrangement (KMT2Ar). Revumenib is the Company's highly selective, oral menin inhibitor. "The Breakthrough Therapy Designation underscores revumenib's potential as a first- and best-in-class therapy to meaningfully change the treatment paradigm for patients with R/R KMT2Ar acute leukemia, whether it presents clinically as acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL), in adults or children," said Michael A. Metzger, Chief Executive Officer. "Revumenib has the potential, if approved, to be the first drug to address the significant unmet need in KMT2Ar leukemia believed to occur in up to 10% of all acute leukemias, including in approximately 80% of infant acute leukemias. Syndax is committed to bringing revumenib to these patients as quickly as possible and we look forward to working collaboratively with the FDA to expedite a potential approval of revumenib." The BTD is supported by Phase 1 data from the AUGMENT-101 trial. Ten of 37 patients (27%) with age and phenotype agnostic KMT2Ar acute leukemia treated at doses meeting the protocol defined criteria for the recommended Phase 2 dose (RP2D) and evaluable for efficacy as of the March 2022 data cutoff achieved a complete remission as measured by a CR/CRh. Included in this analysis were patients treated in Arm A (226 and 276 mg q12 hours not receiving a strong CYP3A4 inhibitor) and Arm B (113 and 163 mg q12 hours receiving a strong CYP3A4 inhibitor).

BOSTON, MA -- November 1, 2022 -- Monte Rosa Therapeutics, Inc. (NASDAQ: GLUE), a biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today announced the company has dosed the first patient in its Phase 1/2 clinical trial evaluating MRT-2359 for the treatment of MYC-driven solid tumors, including lung cancer. MRT-2359 is a potent, selective and orally bioavailable GSPT1-directed MGD, designed to disrupt protein synthesis in MYC-driven tumors, and lead to anti-tumor activity.

“MYC transcription factors are well-defined drivers of human cancers, and disrupting protein translation has emerged as a promising path to address this highly prevalent disease pathway. Dosing the first patient is a significant milestone in developing MRT-2359 as a therapeutic option for MYC-driven cancers,” said Filip Janku, M.D., Ph.D., Chief Medical Officer of Monte Rosa. “We have designed our Phase 1 trial to move efficiently through dose escalation and, by focusing on tumor types where MYC expression is prevalent, to improve our chances of seeing early signals of clinical activity. We are encouraged by the strong preclinical data and hope that MRT-2359 will make a meaningful difference in the lives of patients.”

REDWOOD CITY, CA - Oct. 31, 2022 -- 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 the dosing of the first patient with type 2 diabetes in the Phase II portion of COVALENT-111, a Phase I/II clinical trial underway in Canada. Biomea has completed the Phase I portion of the trial in healthy volunteers. “With the dosing of our first patient with BMF-219, we have reached an important milestone for the nearly 500 million patients worldwide with type 2 diabetes. We are pursuing BMF-219 with the aim to cure this disease. Beta cell preservation, reactivation and regeneration are the core components in providing diabetes patients with long term benefit,” stated Thomas Butler, Biomea Fusion’s Chief Executive Officer and Chairman of the Board. “The burden of diabetes is rapidly increasing, and we are faced with a global unmet need for treatments with novel mechanisms of action. With BMF-219, a potentially first-in-class, covalent menin inhibitor, we are aiming to address and reverse the root cause of this epidemic.”

October 17, 2022

"Homo sapiens is a storytelling animal that thinks in stories rather than in numbers or graphs, and believes that the universe itself works like a story, replete with heroes and villains, conflicts and resolutions, climaxes and happy endings." — Yuval Noah Harari, 21 Lessons for the 21st Century Last year, we provided a founders’ letter that told a story. 15 months into the Treeline journey, we find ourselves in between story and “numbers and graphs.” While we’ve made significant progress in building the pipeline we envisioned at company formation, we are a team that wants our data to do the talking. We are excited for the day when we provide scientific disclosure that has clear implications for patients with cancer and other serious diseases. That day is not today. Nevertheless, a new and exciting financing deserves mention.

COLUMBUS, OH -- September 27, 2022 -- Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced the new availability of plasmid DNA manufacturing to its suite of scalable manufacturing services for gene therapy programs, complementing Forge’s existing AAV process development, analytical development, cGMP manufacturing, and automated final fill capabilities. Forge’s plasmid manufacturing services encompass three grades to accommodate a phase-appropriate approach: Research-Grade, suitable for discovery, research and development; GMP-Pathway, suitable for early-stage clinical trial cGMP AAV manufacturing, and cGMP, suitable for late-stage clinical and commercial cGMP manufacturing. Forge’s plasmid production services utilize single-use systems that seamlessly integrate into the Company’s platform AAV manufacturing process, allowing continuity of manufacturing for clients and streamlined vendor management. Research-Grade and GMP-Pathway plasmid grades are currently available, with cGMP plasmid production available in 2023. All plasmid production will occur at Forge’s manufacturing facility, the Hearth, in Columbus, OH, and is available exclusively to clients performing their AAV manufacturing at Forge.

• Total capital raised of $330 million since the Company’s launch in 2020 • Financing expands planned usage of Company’s 20 cGMP suites containing multiple 50L, 500L, 1,000L and 5000L bioreactors for research-to-commercial gene therapy manufacturing COLUMBUS, OH – September 12, 2022 – Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that it has raised $90 million in a Series C financing, bringing its total funding raised to date to $330 million. The current round was co-led by Drive Capital and Aisling Capital with an additional undisclosed strategic investor. “We continue our ambitious mission to enable access to life changing gene therapies by fulfilling a key goal of bringing them from concept to reality,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics. “To deliver on that mission, we’ve made great strides in our facility development, technology advancement, and hiring expansion. These new funds will help support this next measured phase of our growth focused on delivering new services to our clients so that we can support them from idea to impact. Each advancement we make goes towards serving our clients and their patient communities, and we are grateful to our investors for their confidence in our mission.” The Company will use the funding to expand client offerings, including proprietary technologies, manufacturing systems, cell lines, and additional services to bolster its end-to-end manufacturing platform. Through the Company’s 200,000 square foot cGMP facility dedicated to AAV viral vector manufacturing, Forge will expand the number of offerings available to clients to help scale gene therapy programs from research to clinical and commercial manufacturing in its 20 cGMP suites utilizing its operational 50L, 500L, 1,000L and 5,000L bioreactors.

LONDON and RALEIGH, NC -- August 15, 2022 -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announced today the closing of its upsized public offering of 14,260,000 American Depositary Shares (“ADSs”), each representing eight ordinary shares of Verona Pharma, nominal value £0.05 per share, at a price to the public of $10.50 per ADS, which includes the exercise in full by the underwriters of their option to purchase an additional 1,860,000 ADSs. The aggregate gross proceeds from the offering were approximately $150 million before deducting underwriting discounts and commissions and estimated offering expenses payable by Verona Pharma. All ADSs in the offering were offered by Verona Pharma.