News

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.

LONDON and RALEIGH, N.C., Aug. 09, 2022 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), today announces its top-line Phase 3 ENHANCE-2 (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) trial results evaluating nebulized ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease (“COPD”). The ENHANCE-2 trial has successfully met its primary endpoint, as well as secondary endpoints demonstrating improvements in lung function, and significantly reduced the rate and risk of COPD exacerbations.

Ensifentrine is a first-in-class, dual inhibitor of the enzymes phosphodiesterase 3 and 4 (“PDE3” and “PDE4”) combining bronchodilator and anti-inflammatory activities in one compound.

Highlights

• Study population (n=789):
  • Subject demographics and disease characteristics were well balanced between treatment groups.
  • Approximately 52% of subjects received background COPD therapy, either a long-acting muscarinic antagonist (“LAMA”) or a long-acting beta-agonist (“LABA”). Additionally, approximately 15% 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 average FEV₁ area under the curve 0-12 hours post dose at week 12 was 94 mL (p<0.0001) for ensifentrine.
  • Statistically significant and clinically meaningful improvements with ensifentrine demonstrated across all subgroups including gender, age, smoking status, COPD severity, background medication, ICS use, chronic bronchitis, FEV₁ reversibility, and geographic region.
• Secondary endpoints of lung function met:
  • Placebo corrected, increase in peak FEV₁ of 146 mL (p<0.0001) 0-4 hours post dose at week 12.
  • Placebo corrected, increase in morning trough FEV₁ of 49 mL (p=0.0017) at week 12, confirming twice daily dosing regimen.
• Exacerbation rate reduced:
  • Subjects receiving ensifentrine demonstrated a 42% reduction in the rate of moderate to severe COPD exacerbations over 24 weeks compared to those receiving placebo (p=0.0109).
  • Treatment with ensifentrine significantly decreased the risk of a moderate/severe exacerbation as measured by time to first exacerbation when compared with placebo by 42% (p=0.0088).
• COPD symptoms and Quality of Life (“QOL”):
  • Daily symptoms and QOL as measured by E-RS** Total Score and SGRQ** Total Score in the ensifentrine group improved from baseline to greater than the minimal clinically important difference (“MCID”) of -2 units and -4 units, respectively, at week 24. Improvements in these measures were seen as early as 6 weeks and showed continued improvement at 12 and 24 weeks, numerically exceeding placebo at each measurement. Statistical significance was not achieved due to improvements observed in the placebo group over time.
• Favorable safety profile:
  • Ensifentrine was well tolerated with safety results similar to placebo, including occurrence of pneumonia, gastrointestinal and cardiovascular adverse events.

• Leveraging Poseida's novel approach to cell therapy and Roche's expertise in developing and commercializing therapies to transform cancer care, the collaboration is focused on advancing multiple existing and additional next generation allogeneic CAR-T programs directed to hematologic malignancies
• Poseida will receive $110 million upfront, could receive up to $110 million in near-term milestones and other payments, and is eligible for future development and commercial milestones and tiered royalty payments
• Poseida to host a brief conference call today at 8:30 a.m. ET

SAN DIEGO, CA -- August. 3, 2022 -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today announced it has entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. The global collaboration covers the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications. Under the agreement, Roche will receive from Poseida either exclusive rights or options to develop and commercialize a number of allogeneic CAR-T programs in Poseida's portfolio that are directed to hematologic malignancies, including P-BCMA-ALLO1, an allogeneic CAR-T for the treatment of multiple myeloma and for which a Phase 1 study is underway, and P-CD19CD20-ALLO1, an allogeneic dual CAR-T for the treatment of B-cell malignancies with an IND expected in 2023. Building on complementary expertise and capabilities, the parties will also collaborate in a research program to create and develop next-generation features and improvements for allogeneic CAR-T therapies, from which they would jointly develop additional allogeneic CAR-T product candidates directed to existing and new hematologic targets. For a subset of both the Poseida portfolio programs licensed or optioned to Roche and the parties' future collaboration programs, Poseida will conduct the Phase 1 studies and manufacture clinical materials before transitioning the programs to Roche for further development and commercialization. Roche will be solely responsible for the late-stage clinical development and global commercialization of all products that are subject to the collaboration. Under the agreement, Poseida will receive $110 million upfront and could receive up to $110 million in near-term milestones and other payments in the next several years. In addition, Poseida is eligible to receive research, development, launch, and net sales milestones and other payments potentially up to $6 billion in aggregate value, as well as tiered net sales royalties into the low double digits, across multiple programs.