Biotech Metropolitan Women 2023 Year in Review: New York Edition
Applied Therapeutics Received Orphan Medical Designation
Shoshana Shendelman has led Applied Therapeutics as CEO since its inception in 2016 and has been gathering significant momentum in 2023. Her company prioritizes developing drugs for diseases thatdo not currently have accessible or successful treatment options.
This past year, Applied Therapeutics received the orphan medical product designation for one of their medications, called Govorestat, which is in development. Orphan Medical products receive monetary and regulatory government assistance to incentivize developing drugs for rare diseases since the return on investment would be much lower than with a disease affecting more of the population. Govorestat addresses SORD deficiency, which is peripheral neuropathy caused by the buildup of unoxidized sorbitol in the blood, affecting 1 in 100,000 people. The designation as an orphan medical product enables Applied Therapeutics to devote more resources to advancing its regulatory initiatives and getting this product on the market as soon as possible.
Aquavit Pharmaceuticals Began Clinical Trials
Founded in New York City by Sobin Chang, Aquavit Pharmaceuticals primarily focuses on developing new drug delivery systems like their AQUAGOLD microchannel delivery system. Sobin Chang previously served as Chair of the Portfolio Committee for BOTOX, carrying what she learned there over into her own company. Aquavit is currently pursuing a novel Botulinum Toxin delivery system for the treatment of axillary and palmar hyperhidrosis. In April, Aquavit received authorization to initiate clinical trials for these applications, nicknamed SWEAT I and SWEAT II. Aquavit is the first company to receive regulatory approval for intradermal delivery of BOTOX for palmar hyperhidrosis.
Since Aquavit recently received a license to commercialize BOTOX in 2021, in the US and Canada, they have been expanding their target uses of BOTOX to include treatment for Chronic Migraines, Cerebral Palsy, Depression, and Overactive Bladder, to name a few. Aquavit has submitted another IND proposal to the FDA this year, pending approval in 2024.
Celmatix Made Strides in Treating Infertility, PCOS, and Endometriosis
Piraye Yurttas Beim has a Ph.D. in Oncology and completed post-doc work in mammalian reproduction. Her work inspired her to pursue women’s health and go on to found Celmatix, a pre-clinical organization focused on ovarian health. 1 in 3 women experience problems related to poor ovarian health, spurring Celmatix to pursue several initiatives related to PCOS, Ovarian Cancer, Endometriosis, and Infertility.
Celmatix has had a busy year marked by new drug program launches, landmark genetic studies, and breakthroughs in current investigations. For their PCOS Drug Program, Celmatix reached the hit stage of drug discovery in January, with independent validation of their PCOS drug target. Armed with this knowledge, they launched a new PECOS Drug Program in April that focuses on the identified target melatonin receptors existing in the peripheral nervous system, and how they influence reproductive health. Since then, they partnered with a Brazilian pharmaceutical company called Aché Laboratórios to develop and commercialize a medication to treat PCOS via peripheral melatonin receptors.
Other notable advances by Celmatix in 2023 include their discovery of a genetic basis for Endometriosis in relation to chronic pain and migraines. This is the largest study of its kind on Endometriosis and presents interesting treatment and target options to pursue in their next research initiative on this subject. Celmatix also made a breakthrough discovery in the treatment of infertility, identifying an FSH receptor that can be targeted via an oral medication, eliminating the need for constant injections in IVF treatments. Celmatix has a bright future ahead, and we can’t wait to see what they accomplish in 2024.
ConSynance Therapeutics Moved Into Stage 2 Clinical Trials
Shuang Liu is a seasoned scientist with 25+ years of experience working in research and development of pharmaceuticals, founding Consyance in 2014. Consyance is a pharmaceutical organization that focuses on treating central nervous system diseases, particularly those related to the thalamus. Their current focus is Prader-Willi Syndrome (PWS), a genetic neurodevelopmental disorder that encompasses psychiatric issues and growth problems. The treatment options are sparse, with growth hormones only addressing height issues, there are currently no medications that address the other symptoms of PWS.
Consyance is exploring the applications of an antidepressant discovered in 2015, the Triple Monoamine Reuptake Inhibitor (TRI) for PWS. This TRI is only medication of its kind that targets all three neurotransmitter reuptake types implicated in mood disorders; serotonin, dopamine, and norepinephrine. Consyance has achieved positive results and no adverse events, measured by positron emission tomography, in the first phase of their clinical trial, and are ready to move into phase two to establish if PWS patients respond to treatment with TRI. They aim to begin this study in 2024, using their tailored individualized dosing system.
COTA Releases New Generative AI Tool for Oncology
COTA is a data organization and collection company that uses data to inform research for pharmaceutical companies. Miruna Sasu, among other doctors, engineers, and data scientists, founded COTA in 2011 to solve the issue of inaccessible or incomplete datasets in the medical feild to inform drug discovery and testing. This year, COTA announced a new generative AI platform called Vista, a game-changing tool for cancer research and AI-driven care. Vista taps into automated extraction with expert oversight, delving into electronic health records to extract insights for cancer patients. Vista will propel future oncology research by using data to aid patient matching to clinical trials. Addressing the challenge of unreliable data, Vista ensures transparency and trustworthiness, making it a pivotal tool for advancing generative AI in oncology.
Serving as a precursor to COTA’s existing datasets, Vista allows researchers to seamlessly transition from broad hypothesis generation to specific clinical studies. This comprehensive dataset includes co-morbidity information beyond oncology conditions, offering a portrayal of real-world cancer populations and marking a significant advancement in precision, patient-centric, cancer care. This may change the process of clinical trials and oncology research in 2024, creating AI-generated treatment plans rooted in large-scale, broad-spectrum data from cancer patients.
Curemark Makes Autism Treatment Breakthrough
Joan Fallon’s company, Curemark, takes a novel approach to treating neurological disorders, like Autism, Schizophrenia, and Parkinson’s disease, with enzyme replacement. The experts at Curemark believe that maladaptive behaviors like irritation, agitation, and hyperactivity are due to a lack of certain fundamental enzymes. So far, the enzyme intervention for autism has gone through two phase III trials and has received positive results.
In December 2023, Curemark released the results of their long-term trials on children with Autism, which was published in the Journal of the American Medical Association. The publication explains that children with Autism Spectrum Disorder have 60% of the circulating amino acids in their blood that neurotypical children do, and that may explain many of the behavioral problems. Those amino acids are used to manufacture serotonin, a key neurotransmitter in social interactions, as it can not cross the blood-brain barrier after the age of 2.
Supplementation with these enzymes decreased unfavorable behaviors in all groups, but this study set out to prove that the enzymes don’t just change behavior, but the development of the brain. Using two groups with different enzyme therapy start ages, the study proved that delaying the start of treatment resulted in developmental changes in the early start group that were not seen in the delayed start group. This revelation totally changes the approach to treating Autism, putting early intervention modalities on the table as a possible preventative measure.
ENB Therapeutics Tests New Approaches on Resistant or Recurring Cancers
Sumayah Jamal, co-founder and president of ENB Therapeutics, is a dermatologist with a PhD in biochemistry. ENB Therapeutics focuses on a specific receptor, the selective endothelin B receptor, implicated in melanoma and some other cancers. They have developed a molecule that utilizes the patient’s own immune system to attack cancer cells.
In 2023, ENB announced positive results in phase I of clinical trials treating refractory solid tumors and ovarian cancer. The new drug enhances the efficacy of other drugs in recurring or resistant cancer. The clinical trial revealed successful treatment or unchanged response in concert with Keytruda, another immune-system-based drug for cancer treatment, indicating that clinical trials can proceed to the next stage. ENB is interested in collaborating with Merck on a second phase of trials, where they will assess the effect on real patients.
Epibone Enters Phase I Clinical Trials for Lab-Grown Cartilage
Epibone’s technology allows them to grow bone out of a patient’s stem cells. Led by Nina Tandon, a tissue engineering researcher with a Ph.D. in bioengineering, Epibone has entered the IND stage, with clinical trials beginning imminently. Epibone’s process involves a full 3D CT scan of the bone to be replaced, along with a harvest of stem cells from abdominal fat. Next, they create a scaffold and a perfect environment–a bioreactor–for the bone to grow into the desired shape, inducing the stem cells to proliferate and fill in the matrix they created. After the growth, the bone is ready for implantation. This has advantages over foreign material grafts because it does not require immunosuppressants to prevent potential rejection since it is made of the patient’s cells.
The approval from the FDA allows Epibone to pursue a clinical trial for the replacement of knee cartilage that was grown in their lab. This has applications for those with sports injuries, trauma, or arthritis. The process that they use for creating cartilage is different than their ossification process, employing a mesenchymal condensation technique in order to induce mesenchymal stem cells treated with TGF-β to deposit tenascin and commence building cartilaginous tissue. Epibone is set to start phase one of clinical trials in early 2024.
Evvy Secures $14 million in Series A Funding
Evvy offers the first-ever consumer product to test the vaginal microbiome and genetic markers related to vaginal health. Their product was expanded in 2023 to include not only the initial testing, but also a clinical care plan that involves coaching, prescription medication management, educational materials, and the opportunity to participate in community discussions with similar individuals. After treatment, Evvy has introduced a platform for proactive management, ensuring that the problem that the patient experiences does not recur. This solution is an end-to-end treatment avenue that will revolutionize women’s health.
The CEO and Co-Founder of Evvy, Priyanka Jain, aims to close the gender gap in accessible treatment, destigmatizing seeking care for vaginal discomfort, which is a leading cause of women seeking medical treatment. Evvy received Series A funding in the amount of 14 million dollars from venture capital firms, including Left Lane Capital, to advance their research in linking the vaginal microbiome with infertility, STIs, cancers, and birth problems. They plan to add STI testing, antibiotic resistance, and PCR panels to their testing arsenal in 2024.
Kind Body Democratizing Fertility Treatment with Major Investments and Partnerships
“Only three things matter in healthcare: cost, experience, and outcomes, and only Kindbody, as the provider of care, can effectuate change in all three,” said Gina Bartasi, Founder and Chairwoman of Kindbody. Kindbody, led by CEO Annbeth Eschbach, is the fertility benefits provider for 112 large employers, covering more than 2.4 million lives. The company’s serves patients at 32 signature clinics and hundreds of partner clinics in the U.S. Kindbody’s clinically-managed program covers the full-spectrum of reproductive health, including both female and male fertility, which encompasses fertility assessments and education, fertility preservation, genetic testing, in vitro fertilization (IVF), donor and surrogacy services, and adoption, as well as a full continuum of reproductive care including physical, mental and emotional support.
In March of 2023, this hybrid fertility and reproductive care company Kindbody scored a whopping $100 million from life sciences investment firm Perceptive Advisors, bringing its total raise to $290 million and its valuation to $1.8 billion. Later that spring, the company partnered with JPMorgan Chase’s venture arm, Morgan Health to $25 Million Investment in Kindbody, expanding access to fertility and family-building care.
As the fertility benefits provider, technology platform, and direct provider of high-quality care, Kindbody delivers a seamless, integrated experience with superior health outcomes at lower cost, making fertility care more affordable and accessible for all. Add in 2023 strategic partnerships with Rightway and Quantum Health Kindbody is prepared to meet growing demand for employer fertility benefits.
Massive Bio Partners With 7 Organizations to Improve Clinical Trial Access
Massive Bio breaks down barriers to healthcare, providing equal access to cutting-edge therapies and emerging clinical trials for everyone, irrespective of their location or financial means. Massive Bio’s dedicated team and advanced technology collaborate to connect patients with the most effective treatments available. Stemming from CEO Selin Kurnaz’s personal journey of witnessing the challenges within the healthcare system, Massive Bio was born out of a passion to make a difference. The driving force behind this initiative is the belief that every individual deserves access to world-class healthcare to conquer and overcome the challenges posed by cancer.
In 2023, Massive Bio achieved significant milestones, propelling the landscape of cancer care and clinical trial matching to new heights. One of their biggest innovations of 2023 is their introduction of Chat GPT-4 AI chatbots, AskFiona AI and DrArturo AI, at ASCO 2023. These AI-driven solutions redefine patient and provider engagement, offering unprecedented access to clinical trials and personalized cancer treatments. The company formed several strategic partnerships with other companies to advance its offerings, like;
- The Oncology Institute revolutionizes cancer treatment through the integration of advanced AI technology and a nationwide clinical trial network. This collaboration empowers patients with precise treatment options and accelerated breakthroughs in cancer research.
- Precision Cancer Consortium serves to advance precision oncology. The collaboration optimizes clinical trial matching through AI analytical tools, ensuring efficient patient access to targeted next-generation sequencing testing and tailored interventions.
- The company’s work with CureMatch combines precision medicine with AI-driven clinical trial matching to reach more patients and provide the best possible treatment options.
- Health in Code offers advanced genomic testing. This partnership focuses on increasing patient access to clinical trials and personalized oncology, offering accurate and individualized treatments based on genomic data.
- Neogenomics has a large diagnostic library that will allow Massive Bio to improve drug discovery phases and clinical trial placement.
- The partnership with Asklepieia accelerates the application, selection, and approval processes for clinical trials in the country of Greece.
- Further Group is a global provider of serious illness solutions for the insurance industry. This collaboration streamlines the triage process and ensures smooth patient admission.
Looking ahead to 2024, Massive Bio envisions continued advancements in AI-driven clinical trial matching, personalized oncology solutions, and expanded global collaborations. The company remains dedicated to breaking down barriers to access cutting-edge therapies and clinical trials, transforming the landscape of cancer treatment, and offering hope to patients and their families.
Neurogene FDA Clearance of IND for children with Rett Syndrome
Neurogene, led by CEO Rachel McMinn, clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases. The company announced FDA Clearance of IND for NGN-401 Gene Therapy for children with Rett Syndrome in January 2023. In November, first Patients in Phase 1/2 Trial of NGN-401 were dosed for the treatment of female pediatric patients with Rett Syndrome.
Neurogene ended the year with the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”) and closed an oversubscribed $95 million private financing, led by new and existing healthcare-dedicated specialist and mutual fund institutional investors, including participation from Great Point Partners, EcoR1 Capital, Redmile Group, Samsara BioCapital, Janus Henderson Investors, funds and accounts managed by Blackrock, Casdin Capital, Avidity Partners, Arrowmark Partners, Cormorant Asset Management, Alexandria Venture Investments, and a healthcare investment fund. Neurogene’s cash, cash equivalents, and investments of approximately $200 million, before payment of final transaction-related expenses, are expected to fund operations and multiple potentially value-creating milestones into the second half of 2026.