January 18, 2017
FirstString Research receives the Tibbetts Award from the US. Small Business Administration at a ceremony at the White House. The award recognizes FirstString’s achievements in developing innovative therapeutics for indications with high unmet therapeutic needs.
The US. Small Business Administration (SBA) has awarded one of its highest honors, the Tibbetts Award, to FirstString Research at a ceremony in Washington, DC on January 10, 2017 at the White House. The Tibbetts Award honors the Small Business Innovation Research (SBIR) program participants “that have created a significant economic or social impact […] and are considered the best of the best from the thousands of firms that participate in the SBIR/STTR program”.
FirstString Research was founded on a vision to deliver clinically relevant and effective solutions for inflammation-based medical conditions through a better understanding of the molecular and cellular context that define the underlying pathology. FirstString Research is now advancing the development of its novel peptide-based therapeutic platform for several unmet medical needs, including radiation-induced injuries to the skin, chronic wounds, ophthalmology, burns, organ preservation, and oncology. Its lead drug, aCT1, mitigates excessive inflammation by reinforcing cell-cell contacts in endothelial cells (blood vessel wall), and restoring cell-cell communication that restores and enables cellular coordination and leads to improved injury response.
Support from the SBIR program has enabled FirstString to advance its aCT1-based lead product, Granexin® gel, through four human clinical studies. Pivotal trials are currently underway, green-lighted by the FDA. The product has demonstrated superior clinical benefit in radiation injuries to the skin, diabetic foot ulcers, and venous leg ulcers; making it a highly desirable therapeutic likely to become first-in-line treatment (i.e. standard of care).
“It is an honor to receive this prestigious award”, said Dr. Gautam Ghatnekar, President and CEO of FirstString Research. “The SBIR and STTR programs have helped immensely in advancing our product pipeline through preclinical and clinical testing”, Ghatnekar said. “FirstString is dedicated to the development of cutting edge therapeutic options for indications that have high unmet therapeutic needs and receipt of this award is in recognition of our long-term efforts and accomplishments with respect to this goal.”
May 1, 2016
The Journal of Visualized Experiments (JoVE)
Apr 29, 2015
The Original article was published online on April 29, 2015 and available in the Mar-Apr 2015 Issue.
A link to the full text is available below:
Topical administration of a connexin43-based peptide augments healing of chronic neuropathic diabetic foot ulcers: A multicenter, randomized trial
Nonhealing neuropathic foot ulcers remain a significant problem in individuals with diabetes. The gap-junctional protein connexin43 (Cx43) has roles in dermal wound healing and targeting Cx43 signalling accelerates wound reepithelialization. In a prospective, randomized, multicenter clinical trial we evaluated the efficacy and safety of a peptide mimetic of the C-terminus of Cx43, alpha connexin carboxy-terminal (ACT1), in accelerating the healing of chronic diabetic foot ulcers (DFUs) when incorporated into standard of care (SOC) protocols. Adults with DFUs of at least four weeks duration were randomized to receive SOC with or without topical application of ACT1. Primary outcome was mean percent ulcer reepithelialization and safety variables included incidence of treatment related adverse events (AEs) and detection of ACT1 immunogenicity. ACT1 treatment was associated with a significantly greater reduction in mean percent ulcer area from baseline to 12 weeks (72.1% vs. 57.1%; p = 0.03). Analysis of incidence and median time-to-complete-ulcer closure revealed that ACT1 treatment was associated with a greater percentage of participants that reached 100% ulcer reepitheliazation and a reduced median time-to-complete-ulcer closure. No AEs reported were treatment related, and ACT1 was not immunogenic. Treatment protocols that incorporate ACT1 may present a therapeutic strategy that safely augments the reepithelialization of chronic DFUs.
Dec 12, 2014
This Letter to the Editor is available in the Journal of Investigative Dermatology, Volume 135, Issue 1 (January 2015).
A link to the full editorial text is below:
Healing Refractory Venous Ulcers: New Treatments Offer Hope
Non-healing wounds are associated with an inflammatory and proteolytic wound environment, and recent therapeutic strategies have been focused on reversing these changes. Connexins, as members of gap junctions, are important in intercellular signaling and wound repair. Connexin 43 (Cx43) downregulation is associated with normal wound healing, and it has been found to be upregulated in non-healing venous leg ulcers (VLUs). Ghatnekar et al. (2014) report findings of a small phase II trial performed in Indian patients with chronic VLUs, reporting
that ACT1, a mimetic peptide of Cx43, accelerates healing in the treatment group. Despite standard care with compression therapy and adjuvant therapy for refractory wounds, at present in clinical practice a significant number of patients remain unhealed. The potential for ACT1 exists to help heal refractory VLUs, but it faces additional regulatory hurdles.
Dec 12, 2014
This Original Article was published online on September 11, 2014 and available in Volume 135, Issue 1 (January 2015).
A link to the full text is available below:
The Effect of a Connexin43-Based Peptide on the Healing of Chronic Venous Leg Ulcers: A Multicenter, Randomized Trial
The gap junction protein, connexin43 (Cx43), has critical roles in the inflammatory, edematous, and fibrotic processes following dermal injury and during wound healing, and is abnormally upregulated at the epidermal wound margins of venous leg ulcers (VLUs). Targeting Cx43 with ACT1, a peptide mimetic of the carboxyl-terminus of Cx43, accelerates fibroblast migration and proliferation, and wound reepithelialization. In a prospective, multicenter clinical trial conducted in India, adults with chronic VLUs were randomized to treatment with an ACT1 gel formulation plus conventional standard-of-care (SOC) protocols, involving maintaining wound moisture and
four-layer compression bandage therapy, or SOC protocols alone. The primary end point was mean percent ulcer reepithelialization from baseline to 12 weeks. A significantly greater reduction in mean percent ulcer area from baseline to 12 weeks was associated with the incorporation of ACT1 therapy (79% (SD 50.4)) as compared with compression bandage therapy alone (36% (SD 179.8); P¼ 0.02). Evaluation of secondary efficacy end points
indicated a reduced median time to 50 and 100% ulcer reepithelialization for ACT1-treated ulcers. Incorporation of ACT1 in SOC protocols may represent a well-tolerated, highly effective therapeutic strategy that expedites chronic venous ulcer healing by treating the underlying ulcer pathophysiology through Cx43-mediated pathways.
Jul 15, 2013
FirstString Research, Inc. Announces the Achievement of Primary Endpoints in Three Phase 2 Clinical Trials of Granexin™ Gel for the Treatment of Chronic Wounds and Scar Reduction
MOUNT PLEASANT, S.C.–(BUSINESS WIRE)–FirstString Research, Inc. today announced that it will present positive data from three Phase 2 trials of GranexinTM Gel for the treatment of diabetic foot ulcers (DFUs), venous leg ulcers (VLUs) and for scar reduction at the upcoming International Gap Junction Conference (IGJC), July 13-18, 2013, in Charleston, SC. In each of the Phase 2 trials, GranexinTM Gel was well-tolerated and achieved the primary endpoint of the study with statistical significance. Patients treated with GranexinTM Gel demonstrated highly statistically significant improvements in mean percent wound closure at four and twelve weeks, as well as in incidence and time to 100% wound closure for DFUs and VLUs. A significant reduction in scar formation of surgical incisions following laparoscopic surgery was observed at the study end-point of nine months post-surgery. GranexinTM Gel contains a synthetic peptide, ACT1, which enhances wound healing through a multi-effect mechanism that promotes a healthy regenerative state in tissues.
“Based on the favorable safety data and clinical activity demonstrated in these trials of GranexinTM Gel in DFUs and VLUs, we are in the process of finalizing plans to advance the Granexin program into pivotal Phase 3 studies,” said Gautam Ghatnekar, DVM, PhD, President and CEO of FirstString. “These data reinforce our belief that GranexinTM Gel has potential to become a first-in-class topical peptide for the treatment of chronic wounds and the reduction of scar formation.”
About the Granexin Gel™ Clinical Trials
FirstString Research is scheduled to present results from three separate Phase 2 trials of GranexinTMGel in chronic wounds and scar reduction at the International Gap Junction Conference (IGJC). Each of the Phase 2 studies was randomized, controlled, multi-center, and blinded to evaluate the safety and efficacy of GranexinTM Gel for the treatment of VLUs and DFUs; and the reduction of scarring following laparoscopic surgery. In the twelve-week studies for the treatment of VLUs and DFUs, GranexinTM Gel (100μM ACT1) was administered three times in the first week, followed by weekly application for the following eleven weeks. To date, over 300 patients have participated in Phase 1 and Phase 2 clinical evaluations of GranexinTM Gel. GranexinTM Gel application resulted in highly significant increases in mean percent wound closure at four and twelve weeks as well as incidence of 100% wound closure, along with significantly reduced time for complete wound closure. In the nine-month study for the reduction of scarring following laparoscopic surgery, GranexinTM Gel (100μM ACT1) was administered once at wound closure and 24 hours post-surgery. GranexinTM Gel demonstrated statistically significant reduction in scarring as measured by Vancouver Scar Scoring (VSS) consisting of scar vascularity, pigmentation, pliability, and height at the study end-point of nine months post-surgery. In all studies, GranexinTM Gel was safe and well-tolerated with no drug-related systemic or local adverse events. Detailed results for the Phase 2 clinical trials will be published in peer-reviewed journals.
Details of the oral presentation to be made at the IGJC are as follows:
Date: Tuesday, July 16, 2013
Time: 3:30-5:30pm ET
Location: Francis Marion Hotel, Charleston, SC
Title: ACT1 Scene 1: Clinical Benefits of the Connexin-Based Mimetic Peptide ACT1
Presenting Author: Gautam Ghatnekar
About Granexin™ Gel
Granexin GelTM contains ACT1, a synthetic peptide designed to mimic the C-terminus of the gap junction protein connexin 43 (Cx43), with high binding specificity to the tight junction associated protein zona occludens (ZO-1). Gap junctions and tight junctions are found in many cells throughout the body, including epithelial cells, which make up the skin and line other organs. Gap junctions connect the cytoplasms of adjacent cells, allowing free exchange of ions and molecules. Tight junctions form barriers between cells that are virtually impermeable by fluid. ACT1 is soluble, engineered to directly translocate within cells, and interacts with ZO-1, a known binding partner of the C-terminus of Cx43. The binding of the peptide to specific Cx43 C-terminus interaction domains such as PDZ-2 on ZO-1 serves to competitively inhibit its association with the Cx43 C-terminus, resulting in stabilized gap junctions and tight junctions. ACT1 also sequesters hemichannels (extracellular communicating channels) into gap junctions. This multi-effect mechanism – reducing hemichannel activity, promoting gap junctional communication, re-enforcing tight junction integrity – tempers excessive inflammation at the site of injury and promotes a healthy regenerative response. These activities result in the promotion of chronic wound healing and reduction of scarring. The clear, odorless, easy-to-apply topical formulation is also designed to provide a localized protective barrier against microbial colonization and a moist environment to promote natural autolytic debridement of necrotic tissue surrounding a wound. GranexinTM Gel has been evaluated in three Phase 2 trials for the treatment of diabetic foot ulcers, venous leg ulcers and the reduction of scarring following laparoscopic surgery.
About FirstString Research
FirstString Reseach, a clinical stage biotech company, is leading the translation of cell-cell communication and cell-cell contact/adhesion science into a pipeline of drugs and medical applications for wound healing, scar reduction, inflammation, and complex tissue regenerationࠀ The company is currently advancing GranexinTM Gel, a topical formulation containing the ACT1 peptide, for the treatment of chronic wounds and scar reduction, through clinical trials, with the goal to broaden the treatment options for patients suffering from morbidities associated with acute and chronic wounds. FirstString is also conducting preclinical studies with ACT1 for additional indications having high unmet therapeutic needs in a number of therapeutic segments that include ophthalmology, CNS injuries, cardiac injuries, organ transplants, acute lung injuries, and oncology.
Mar 18, 2012
Local tech firms can face uphill climb to win over investors
CHARLESTON, SC – March 8, 2012 – Dr. Gautam Ghatnekar sat before a crowd of Charleston business people recently and acknowledged that “it has not been that smooth sailing” since he launched FirstString Research nearly seven years ago.
Ghatnekar could’ve been talking about the scientific challenges associated with converting his peptide discovery into a safe, effective scar-preventing gel. But he wasn’t.
Instead, the former veterinarian and Medical University of South Carolina post-doc was talking about something local tech entrepreneurs understand all too well: how tough it can be to raise money.
“I mean, you’re asking people to bet on what you really believe in, and that’s never going to be easy. You have to convince them to part with their hard-earned money to invest in your bright idea. It doesn’t sound easy and it’s not easy and it shouldn’t be easy,” Ghatnekar said. “Good ideas typically survive, but there’s no guarantee of success, of course.”
Ghatnekar recalled that, even as early potential investors were presented a patented technology licensed from MUSC, they saw “this … young Indian kid” and said, “I’m not going to give you $100,000. What are you going to do with it?”
Since those early growing pains, the 34-year-old Mumbai native and his company have come a long way. FirstString has attracted several million dollars from investors and, despite executive changes and financial straits over the years, is running three advanced Phase II trials, which test efficacy.
In many ways, Ghatnekar’s experience hunting for capital as a Charleston tech start-up is typical. FirstString’s money has come from a variety of sources, including wealthy individuals, so-called angel investors who seek to get in early on promising young businesses, and government sources, both local and out-of-state.
He’s talked with venture capitalists but never took their money. He’s learned to recognize people who can bring value to his company and those who can’t. And he’s still adapting, pivoting and angling his company forward.
“It’s a miracle of sorts that we are still in business,” Ghatnekar said. “But somehow we’ve managed to stay afloat.”
A few local companies, such as human resources software firm PeopleMatter, have been able to attract — and have relied heavily on — venture capital. Others, such as fast-growing health care software company Benefitfocus and biotech firm Immunologix, which made a tidy profit for its owners when it was sold last fall, have succeeded without so-called institutional money.
Shawn Jenkins, chief executive officer of Daniel Island-based Benefitfocus, said the dearth of capital was a hot topic in the Charleston tech community a dozen years ago when he co-founded his company. He said Benefitfocus doesn’t need investor money now — Goldman Sachs bought out the early investors in 2007 — but that many of its smaller software peers do.
“I wouldn’t say that’s totally solved now,” Jenkins said of the capital problem. “But it’s definitely gotten better.”
In fact, Jenkins said both he and his company would consider investing in a promising local start-up.
Like the increase in talent, investment is another indication that the Charleston area tech sector is on the rise, if not in the same league as mature hubs such as Silicon Valley, Boston and Austin, Texas.
According to last year’s National Venture Capital Association Yearbook, between 1985 and 2010, South Carolina has ranked toward the top of the bottom third of states in terms of venture capital investment and number of deals. California is the clear leader, followed by Massachusetts and New York.
In 2010 and last year, 10 Palmetto State companies attracted $48.74 million in venture capital, with six software companies landing $38.74 million of that, said John Taylor, head of research at the NVCA.
“The Southeast, certainly at the moment, is quieter compared with some of the other regions,” he said.
Among the recent standouts are Charleston-based dermatology drug and device firm Innocutis, which scored a $6.5 million investment from Florida’s Ballast Point Ventures, and PeopleMatter, which closed both a $7.2 million round led by Noro-Mosely Partners of Atlanta and a $14 million round led by Silicon Valley heavyweight Morgenthaler Ventures.
But such deals are exceptional, a fact that Andrea Marshall knows well. She is executive administrator of Charleston Angel Partners, a consortium of about 30 wealthy individuals. It receives “hundreds” of applications each year and invests — usually $100,000 to $200,000 — in only one or two companies, generally tech firms in the Carolinas, Marshall said. The group has put in $4.9 million over its almost eight years in operation. FirstString Research, which has received two rounds of funding, is one of its 10 portfolio companies.
Marshall said Charleston’s tech companies, seeking that elusive “critical mass,” contend with a version of “the chicken and the egg problem,” wherein they have to grow up to gain market acceptance and investment but can’t do that without early stage capital.
Even in the best of times, there’s not enough money out there for all the promising companies, she said. Add in the recent macroeconomic conditions and Charleston’s relative youth in software and biotech, and obtaining investment capital is no small feat.
“In late 2008 and most of 2009, I would say things were dark and scary,” Marshall said. “Since that time, we’re seeing a gradual re-engagement with early stage investment. … Now the sun’s just starting to peek out from behind the clouds.”
Speaking at a November biotech conference in Charleston, venture capitalist Ed Snape said it’s not a shortage of investment opportunities that’s the problem in the Lowcountry.
“There are deals,” said Snape, a senior adviser to the Nexus Group, which has a North Charleston office. “There’s not enough money. There’s not enough intelligent money.”
But that gap could be closing. Taylor, with the venture capital industry group, said recurring interest from regional institutional investors, teaming with national investors, is a good sign.
“That is an increasingly common model, and that is the way a lot of areas get going, frankly,” he said.
Good people working at a community of good companies trumps distance from traditional venture capital centers, according to both Taylor and Blackbaud Senior Vice President Jana Eggers.
“What they really want is access to talent to make sure that you can grow enough and make sure that you’re not stagnant there,” said Eggers, who dealt extensively with venture capitalists in Europe before coming to work for Blackbaud in 2010.
Besides private angel, venture and corporate money, another source of start-up cash for Charleston tech companies is SC Launch, whose annual Industry Partnership Fund, $6 million of tax-credited contributions, has invested in the likes of FirstString Research and Immunologix.
Recognizing the need, the South Carolina Research Authority, which helps administer SC Launch, is raising money for another fund that would provide follow-on capital.
Even more locally, the Charleston Digital Corridor is recruiting contributions for its own seed fund.
“Things might be coming together here. Feels like it anyway,” said Ghatnekar of FirstString Research.
But Charleston won’t reach its aspirations overnight. It’s a work in progress, just like Ghatnekar’s business.
“Takes time and a lot of patience,” he said.
A sampling of notable capital investments in Charleston technology firms:
1999: San Francisco’s Hellman & Friedman and JMI Equity Fund of San Diego pumped $155 million into Daniel Island-based Blackbaud Inc. The firms took a controlling stake in the then-privately held software company, which sold shares to the public in 2004.
2007: Palo Alto, Calif.-based Technology Crossover Ventures bought a $60 million stake in Mount Pleasant-based Automated Trading Desk, which six months later was purchased by Citigroup for $680 million.
2009: Acadia Human Capital Solutions (later renamed PeopleMatter), got its first round of venture money when InterSouth Partners of Durham, N.C., along with C&B Capital of Atlanta put in $5 million.
2011: PeopleMatter landed $7.2 million in a round led by Atlanta-based Noro-Moseley Partners. Innocutis received a $6.5 million investment from Florida-based Ballast Point Ventures.
2012: PeopleMatter received $14 million in a funding round led by Menlo Park, Calif.-based Morgenthaler Ventures.
Aug 15, 2011
Sep 8, 2010
CHARLESTON, S.C. – September 8, 2010 – FirstString Research, a biotechnology company focused on the development and commercialization of its therapeutic platform for wound healing, scar reduction and tissue regeneration, announced a major milestone in its development with the completion of Phase I clinical trials. “This trial, in 48 human subjects, has successfully demonstrated the safety and early efficacy of our compound,” said FirstString Chief Executive, Dr. Gautam Ghatnekar.
FirstString Research has raised additional capital needed to complete the successive Phase II trial. “We expect to get a larger Phase II trial underway shortly to further demonstrate the efficacy and safety of our lead compound,” said Dr. Ghatnekar.
FirstString has also received patents from the United States Patent and Trademark Office (USPTO) on its composition of matter claims – effectively giving the company a concrete asset by validating their core technology platform. On the subject of its technology, Dr Ghatnekar stated that the company’s patent portfolio is broad and extensive, and the company expects to have additional patents issued shortly.
About FirstString Research, Inc.
FirstString is a biotechnology company focused on the development and commercialization of therapeutics for wound healing, scar reduction and tissue regeneration. FirstString’s proprietary therapies modulate intercellular communication to significantly reduce the development of scar tissue and, at the same time, improve the body’s ability to produce healthy, functional cells.
Nov 4, 2009
A Charleston-based biotech startup is receiving a $200,000 investment today at the Small Business Innovation Summit & Expo at Trident Technical College in North Charleston.
SC Launch, a collaboration with the S.C. Research Authority, is making the investment in FirstString Research Inc., which is developing a product for scar prevention and tissue regeneration.
“The support we’ve received from SC Launch even goes beyond the equity investment we’ve received from them,” said Gautam Ghatnekar, FirstString’s president and CEO. “SC Launch has brought the right resources to the table. This investment, along with SCRA, SC Launch and additional in-state angel support, has helped us double in size over the last six months, enabling continued growth for us here in South Carolina.”
FirstString Research was one of the first recipients of funds from SC Launch as part of the Small Business Innovation Research/Small Business Technology Transfer Phase I Matching Grant Program. SC Launch client companies have secured more than $71 million to date in follow-on venture capital and private equity funding.
“With peptide-based intellectual property licensed from the Medical University of South Carolina, FirstString is the exact archetype of how we want to support bio-related commercialization in partnership with our in-state research universities,” said Bill Mahoney, chief executive of SCRA.
FirstString’s first commercial product will be a topical gel for the prevention of scarring. Based on early results, FirstString has already received regulatory approval in Europe to enter clinical phase testing.
SC Launch assists startup companies with upfront counseling, seed funding and access to a resource network. SCRA is an applied research and commercialization services company with offices in South Carolina, Ohio and the Washington, D.C. area.