Pfizer Breakthrough Growth Initiative

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

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 Cardiff Oncology is a California-based clinical-stage precision oncology startup that develops treatment combinations targeting gene mutations associated with drug resistance.
 
 
 Its flagship drug candidate onvansertib is a third-generation PLK1 (Polo-like Kinase 1) inhibitor and is in Phase 2 clinical trials. Its development process combines tumor genomics and biomarker technology to assess patient response to treatment. In Q2 2023, the company announced a new lead program in RAS-mutated metastatic colorectal cancer (mCRC) and expects to release interim topline data mid 2024.&nbsp;
 
 
 Funding and financials
 
 
 In November 2021, the company received a USD 15 million equity investment from Pfizer&rsquo;s Breakthrough Growth Initiative program to accelerate the development of onvansertib for treating KRAS-mutated metastatic colorectal cancer, metastatic pancreatic cancer, and metastatic castrate-resistant prostate cancer, which are in Phase II trials. It also plans to advance the preclinical development of onvansertib for treating other indications. The pharma giant was also given the right of first access to data from Cardiff&rsquo;s development programs.
 
 
 <a href="https://sp-edge.com/updates/16828">
 For the year 2022
 </a>
 , revenue rose 7.5% YoY to USD 0.4 million mainly due to higher royalty revenue while net losses for the same period increased by 36.8% YoY to USD 38.7 million. For the six months ended June 2023, the company achieved a revenue of USD 0.2 million (down by 15.8% YoY) and net loss of USD 11.2 million (up by 7.7% YoY). Its cash and cash equivalent as of June 2023 was at USD 19.4 million, compared to USD 16.3 million in December 2022, which the company believes will be adequate to fund operations till 2025. 
 
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 Akero Therapeutics is focused on developing treatments for serious metabolic diseases, particularly non-alcoholic steatohepatitis (NASH). NASH is a chronic liver disease that is characterized by the buildup of fat in the liver, inflammation, and damage to liver cells.&nbsp;
 
 
 Its lead product candidate, efruxifermin (EFX), is a once-weekly injection dosing of the fibroblast growth factor 21 fusion protein, which, as of August 2023, was in Phase IIb clinical trials for EFX in patients with pre-cirrhotic NASH and cirrhotic NASH and thus was being developed as a multi-modal investigational drug for NASH. Furthermore, the company claims it was the first investigational drug for NASH to receive the most designations; 1) Prime (from European Medicines Agency in 2020), 2) Fast Track (from FDA in 2021), and 3) Breakthrough Therapy (from FDA in 2022). In addition, the company has several research programs with undisclosed indications.&nbsp;
 
 
 Key Customers and Partnerships
 
 
 In October 2019, the company partnered with InSphero to use InSphero&rsquo;s 3D InSight Human Liver Disease Platform to investigate the effects of&nbsp; Akero&rsquo;s drug candidate, AKR-001, in various types of liver cells.&nbsp;
 
 
 Funding and Financials
 
 
 The company began trading on Nasdaq in June 2019 under the ticker symbol &ldquo;AKRO&rdquo; and raised USD 92 million at IPO. In May 2023, the company announced an 
 <a href="https://sp-edge.com/updates/18639">
 underwritten public offering
 </a>
 of USD 200 million in gross proceeds. The funds raised were used for the clinical development of EFX, to support future clinical trials, potential in-licensing for pipeline diversification, and working capital and general corporate purposes.
 
 
 The company did not generate any revenue in the three months ended March 2023. However, it reported a net loss of USD 25.8 million, which had improved slightly by 1% YoY. Its cash and cash equivalent position as of March 2023 was at USD 343.2 million, which extended the company&rsquo;s cash runway until 2025.
 
 
 
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Reinventing gene therapies with next-generation technologies

Human Gene Editing

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 Homology Medicines develops novel gene and gene editing therapies for the treatment of a range of genetic diseases starting off with monogenic diseases&mdash;diseases caused by defects in a single gene. The company&nbsp; leverages its proprietary human hematopoietic stem cell-derived adeno-associated virus (AAVHSC) vector technology based on homologous recombination, a naturally occurring process to ensure DNA repair. This process deals with gene correction and does not require nucleases unlike common methods such as CRISPR/Cas9 that perform gene cutting. According to the company, the process enables precise on-target editing without unnecessary off-target DNA modifications.&nbsp;
 
 
 As of July 2022, Homology had seven programs in its pipeline, consisting of four gene therapies and three gene editing therapies. The company&rsquo;s most advanced program is a gene therapy for adult Phenylketonuria (PKU)&mdash;a rare inherited disorder&mdash;in the Phase II clinical trials. As for its gene editing pipeline, as of 2022, one program was in the Phase I clinical trials for pediatric PKU and the remaining two were in the preclinical stages.&nbsp;
 
 
 Key customers and partnerships
 
 
 The company has secured a number of big pharma partnerships that include 
 <a href="https://www.homologymedicines.com/news-story/novartis-collaboration?/novartis-collaboration">
 Novartis
 </a>
 in November 2017 to leverage Homology&rsquo;s gene editing technology to develop therapies for select ophthalmic targets and a hemoglobinopathy disease and 
 <a href="https://www.homologymedicines.com/news-story/homology-medicines-announces-60-million-equity-investment-from-pfizer-inc">
 Pfizer
 </a>
 in November 2020 with focus on the company&rsquo;s PKU therapy, which also came with a USD 60 million equity investment.&nbsp;
 
 
 The company also launched a new AAV manufacturing and innovation business in January 2022 called ​​
 <a href="https://www.biospace.com/article/symbiotic-deal-snags-cash-for-homology-and-aav-manufacturing-for-oxford-biomedica-/#:~:text=Britain's%20Oxford%20BioMedica%20and%20Bedford,Homology's%20current%20chief%20operating%20officer.">
 Oxford Biomedica Solutions
 </a>
 , in partnership with British company Oxford Biomedica. Homology owned 20% of the company&rsquo;s stake, while Oxford Biomedica held the majority.&nbsp;
 
 
 Funding and financials
 
 
 Homology Medicines, listed on the Nasdaq Global Select market since April 2018, raised gross proceeds of USD 50 million in its latest 
 <a href="https://www.homologymedicines.com/news-story/homology-medicines-announces-pricing-of-public-offering-of-common-stoc">
 post-IPO
 </a>
 public offering in April 2021. The proceeds were earmarked to advance its clinical development pipeline, expand its manufacturing capacity, and to fuel working capital expenses. For 2021, the company generated USD 34 million in collaboration revenue. 
 
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Targeting complex diseases with complex molecules

Large-molecule Therapeutics

AgomAb Therapeutics is a Belgian biotech company pioneering and developing novel treatments that aim to resolve fibrosis, repair tissue structure, and restore organ function. The company's pipeline comprises growth factor-targeting antibodies and small molecule compounds, including its lead candidate AGMB-129, a gastrointestinal tract restricted ALK5 inhibitor. In February 2023, AgomAb announced positive Phase 1 results showing that single- and multiple-dose oral AGMB-129 was safe and well-tolerated for the treatment of Fibrostenosing Crohn's Disease (FSCD). It has recently initiated a Phase 2a clinical trial (STENOVA) evaluating AGMB-129 in patients with symptomatic FSCD and received US FDA Fast Track Designation for the drug candidate in October 2023.
AgomAb's second TGF-β targeting pipeline candidate is AGMB-447, a Phase 1-ready lung-restricted ALK5 inhibitor for the treatment of Idiopathic Pulmonary Fibrosis, which received Orphan Drug Designation from the FDA in June 2024. AGMB-101 is an HGF-mimetic cMET receptor agonist in IND-enabling development for treating fibrotic disorders. In October 2023, AgomAb closed a USD 100 million (EUR 94.9 million) Series C financing led by Fidelity Management & Research Company to advance its pipeline.

Cour Pharmaceuticals Development Co Inc operates in the pharmaceutical sector. Cour Pharmaceuticals Development Co Inc was founded in 2012 and is based in Elmhurst, Illinois.

Anokion, a spin-off from the Ecole Polytechnique Fédérale de Lausanne (EPFL), is focused on applying the company’s antigen-specific immune tolerance technology to reduce the immunogenicity of therapeutic proteins and to treat autoimmune and allergic diseases. As a platform technology, Anokion’s approach to antigen-specific tolerance can be translated to virtually any protein in numerous clinical indications.

AgomAb is developing molecular therapies for the regeneration of damaged tissues. Their unique agonistic monoclonal antibody molecules (“agomAbs”) stimulate molecular and cellular repair mechanisms that can potentially restore organ function in patients with fibrotic, inflammatory, autoimmune, and degenerative diseases. The company is focusing on biologically validated pathways, such as Transforming Growth Factor β and Hepatocyte Growth Factor, and leveraging specialized capabilities in organ-specific small molecules and high-affinity antibodies. With a diversified clinical pipeline across multiple fibrotic conditions, end-to-end research and development expertise, a successful business development track record, and a robust investor base.

Akero Therapeutics is committed to providing meaningful treatment options for people with serious metabolic diseases like non-alcoholic steatohepatitis (NASH), a disease marked by inflammation in the liver and liver cell damage resulting in fibrosis, cirrhosis, liver failure, and death. The company is developing pioneering medicines designed to restore metabolic balance and halt NASH progression.  Akero's lead clinical program, AKR-001, is a long-acting Fc fusion modified FGF21 protein and potential best-in-class treatment for NASH. The AKR-001 builds upon two decades of work on FGF21 biology. It is designed to affect both the liver and adipose tissue, reducing liver fat and suppressing inflammation and fibrosis.

Cardiff Oncology is a clinical-stage oncology therapeutics company developing drugs that target cell division for the treatment of leukemias, lymphomas, and solid tumor cancers. It has a broad intellectual property and proprietary technology to measure circulating tumor DNA (ctDNA) in urine and blood to identify and quantify clinically actionable markers for predicting response to cancer therapies. The company offers its PCM technology at its CLIA/CAP-accredited laboratory and plans to continue to vertically integrate its PCM technology with precision cancer therapeutics. Cardiff Oncology (formerly known as Trovagene) was founded in 1999 and is headquartered in San Diego, California.

Vedanta Biosciences is a developer of immunotherapies designed to treat immune-mediated and infectious diseases. It includes a suite of proprietary assays to select pharmacologically potent strains, vast proprietary datasets from human interventional studies, and facilities for cGMP-compliant manufacturing of rationally defined bacterial consortia in powder form to help physicians have access to live bacteria drugs to treat autoimmune and inflammatory diseases easily.

ESSA is a pharmaceutical company focused on the development of small molecule drugs for the treatment of cancer, with a focus on advanced prostate cancer. ESSA is developing drugs that selectively block the N-terminal domain of the androgen receptor (AR). The AR is required for the growth and survival of most prostate cancer; therefore, the AR N-terminal domain is an ideal target for next-generation hormone therapy.

Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases and a robust intellectual property portfolio with issued composition of matter patents in the United States for its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines.

Trillium Therapeutics Inc. operates as a biopharmaceutical research and development company. It focuses on the immune system and its regulation. The company specializes in the discovery and development of protein therapeutics, such as monoclonal antibodies, secreted proteins, and peptides. It involves in therapeutic areas, including autoimmune disease, cancer, necrotizing enterocolitis, and transplantation. Trillium Therapeutics Inc. was formerly known as Transplantation Technologies Inc. and changed its name in May 2003. The company was founded in 1996 and is based in Toronto, Canada.

The Pfizer Breakthrough Growth Initiative will focus on making non-controlling equity investments in clinical-stage public companies, with a primary focus on companies with small- to medium-sized market capitalizations across a range of therapeutic categories that are consistent with Pfizer’s core areas of focus: Internal Medicine, Inflammation & Immunology, Oncology, Rare Disease, Vaccines and Hospital.

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