From the CRXM CEO:
The Opportunity: We are a San Diego-based, late-stage biotechnology company focused on the clinical development
and commercialization of angiogenic gene therapy biotherapeutics for strategic niche markets primarily for the
treatment of cardiovascular disease.
Our lead Generx [Ad5FGF-4], is an innovative single, one-time dose, catheter-based, treatment for patients with
refractory angina due to late-stage coronary artery disease, for use by interventional cardiology physiciansimmediately
following a standard angiogram procedure. There are an estimated 1.0 million cardiac angiograms are performed in
the U.S. each year, and an estimated 500,000 of these patients could potentially benefit from Generx therapy, at a
projected $8,000 to $10,000 per dose, representing a $4.0 to $5.0 billion total potential market opportunity for this
patient subgroup alone. There is no special training or medical devices required for physicians to integrate the use of
Generx into their daily practice. These patients have an unmet medical need, and Generx offers a new business with a
billion-dollar market opportunity for physicians.
Investors: We are a majority-owned operating subsidiary of Nostrum Pharmaceuticals LLC, a U.S.-based, privately
owned, fully integrated, pharma company, engaged in the formulation and commercialization of specialty products
and controlled-release, orally administrated, branded, and generic drug products. Nostrum operates multiple
manufacturing facilities, supported by sales, marketing and distribution activities, that sells products to hospitals and
retail drug stores, by Rx prescription only, throughout the United States. Nostrum currently owns approximately 75%
of Gene Biotherapeutics and approximately 25% is owned by public shareholders. Gene Bio investors currently own an
85% interest in Generx, and Huapont Life Sciences, a Chinese-based, publicly traded, pharmaceutical company, owns a
15% interest in Generx, and has an exclusive right to market and sell Generx in mainland China following FDA approval
of Generx in the United States
Business Strategy: Gene Bio has a cost-conserved and outsourced-service provider operating structure, supported by
Nostrum’s corporate infrastructure and capital. We have a highly experienced team thatis currently singularity focused
on (1) the manufacture of our lead product Generx [Ad5FGF-4] by
Fujifilm Diosynth Biotechnologies, located in College Park, Texas; (2) the
conduct of our final 160-patient, FDA-cleared, Phase 3 clinical study,
and (3) FDA approval. The FDA has now cleared the Generx cGMP
manufacturing specifications and Phase 3 clinical study. With the
successful completion of this final study, Gene Bio plans submit a
biologics license application to the FDA and secure FDA approval to
market and sell Generx in the United States.
Proposed Financing: We are seeking to raise up to $6.0 million in
a short-term (24 month) debt financing to support our going
forward operations as a C-Round bridge financing which will be
followed (within 12-16 months) by a planned S-1 Registration
Statement initial public financing, that is anticipated to fully fund
the Generx clinical development program. The attached term sheet
provides the terms and conditions covering this proposed financing.
Investors will have a right to receive repayment of principal and
interest at maturity or participate in the planned IPO at a significant
discountto market. Nostrum has agreed to guarantee repayment of
principal at maturity.
Fact Sheet &
Proposed IPO
Bridge C-Round
Debt Financing
The Mechanism of Action: Generx has been designed to improve cardiac perfusion by promoting the formation of
functional coronary collateral blood vessels within the heart through the enlargement of existing arterioles
(arteriogenesis) and the formation on new capillary vessels (angiogenesis). This process, termed “medical
revascularization,” represents a fundamentally new mechanism of action that involves the stimulation and formation
of new biological structures in the heart, as opposed to currently available traditional symptomatic pharmacologic
therapies, which only address the symptoms of angina, or mechanical revascularization, through procedures involving
angioplasty and stent placement or coronary artery bypass graft surgery.
Clinical Findings: The Generx regulatory dossier represents one of the most extensive and advanced gene-based clinical
data platforms ever compiled. In multiple prior clinical studies, the Generx product candidate has been evaluated in
over 650 patients (450 receiving Generx) and appears safe and well-tolerated with findings of efficacy in men and
women, in measures of cardiac perfusion, exercise capacity, and angina status. Specifically, Generx has been evaluated
as a treatment for patients with refractory angina in four prior FDA-cleared, multi-center, randomized and placebocontrolled clinical studies (AGENT 1-4, covering Phase 1|2, Phase 2 and two Phase 2b/3 clinical studies), and one small
Phase 2 international study (ASPIRE). These studies have generated over 2,500 patient years of safety data. Generx has
now been cleared by the FDA for our final Phase 3 clinical study to further evaluate safety and definitive efficacy.
U.S. Market Strategy: To support our go to market strategy, we conducted a survey of U.S. interventional cardiologists to
gauge their experience-based assessment of the prevalence of refractory angina patients, and their openness to integrate
the use of the Generx angiogenic gene therapy product candidate, upon FDA approval, into their clinical practice. The
survey confirmed that all survey physician responders see patients with long-term refractory angina as an unmet medical
need, and all were strongly positive and without reservation about the adoption of Generx following FDA approval. All
cardiologists surveyed felt there is a current need for Generx to treat refractory angina and they would consider using
Generx in their daily practice.
Competitive Factors: We believe that we have a significant clinical lead over even our nearest competitor, and that our
knowledge base, technology platform, a $200 million investment research and development spend by larger pharma
players prior to Nostrum’s acquisition, our FDA cleared treatment algorithm and business model, all represent significant
barriers to entry. Following FDA approval, we believe that Generx will potentially be considered superior in the
competitive marketplace. We believe that Generx therapy will represent a better, faster, and cheaper solution than other
therapies now in development. Our FDA-cleared Ad5FGF-4 manufacturing process offers significant gross margin
opportunities, scalable campaign manufacturing, generates a ready to use plug-n-play product, and has a well-researched
and clinically supported MOA, and receptor-based, cardiac cell targeting. We see little or no direct competition in our
space until 2030.
Potential for Exceptional ROI: With public access to capital markets and sufficient financial resources, an accelerating
Phase 3 clinical study, a NASDAQ listing with stock trading liquidity, on a post-IPO basis, we believe, that there would be
support for an intermediate market capitalization ranging from $200 million to $500 million. Based on the Company’s
business model, market opportunity, overall economics and competitive exclusivity, with the successful completion of the
Phase 3 clinical study, Gene Bio believes that there would be ample evidence to support a Generx terminal value range
of $1.0 billion to $3.0 billion, that could potentially be offered by key strategic players from the pharmaceutical and
biotechnology companies focused in the gene therapy, cardiovascular drugs and biologics and vascular access sectors
operating in the U.S. and internationally.
For additional information, please reference our Annual Report on Form 10-K, dated April 21, 2022, as filed with the SEC,
the IPO Bridge C-Round Debt financing, dated June 22, 2022, and Gene Investor Presentation, dated July 27, 2021, that is
available on the Company’s website below. In addition, please also visit Gene Bio’s website
www.GeneBiotherapeutics.com and our Phase 3 patient recruitment website at www.MyRefractoryAngina.com
The Opportunity: We are a San Diego-based, late-stage biotechnology company focused on the clinical development
and commercialization of angiogenic gene therapy biotherapeutics for strategic niche markets primarily for the
treatment of cardiovascular disease.
Our lead Generx [Ad5FGF-4], is an innovative single, one-time dose, catheter-based, treatment for patients with
refractory angina due to late-stage coronary artery disease, for use by interventional cardiology physiciansimmediately
following a standard angiogram procedure. There are an estimated 1.0 million cardiac angiograms are performed in
the U.S. each year, and an estimated 500,000 of these patients could potentially benefit from Generx therapy, at a
projected $8,000 to $10,000 per dose, representing a $4.0 to $5.0 billion total potential market opportunity for this
patient subgroup alone. There is no special training or medical devices required for physicians to integrate the use of
Generx into their daily practice. These patients have an unmet medical need, and Generx offers a new business with a
billion-dollar market opportunity for physicians.
Investors: We are a majority-owned operating subsidiary of Nostrum Pharmaceuticals LLC, a U.S.-based, privately
owned, fully integrated, pharma company, engaged in the formulation and commercialization of specialty products
and controlled-release, orally administrated, branded, and generic drug products. Nostrum operates multiple
manufacturing facilities, supported by sales, marketing and distribution activities, that sells products to hospitals and
retail drug stores, by Rx prescription only, throughout the United States. Nostrum currently owns approximately 75%
of Gene Biotherapeutics and approximately 25% is owned by public shareholders. Gene Bio investors currently own an
85% interest in Generx, and Huapont Life Sciences, a Chinese-based, publicly traded, pharmaceutical company, owns a
15% interest in Generx, and has an exclusive right to market and sell Generx in mainland China following FDA approval
of Generx in the United States
Business Strategy: Gene Bio has a cost-conserved and outsourced-service provider operating structure, supported by
Nostrum’s corporate infrastructure and capital. We have a highly experienced team thatis currently singularity focused
on (1) the manufacture of our lead product Generx [Ad5FGF-4] by
Fujifilm Diosynth Biotechnologies, located in College Park, Texas; (2) the
conduct of our final 160-patient, FDA-cleared, Phase 3 clinical study,
and (3) FDA approval. The FDA has now cleared the Generx cGMP
manufacturing specifications and Phase 3 clinical study. With the
successful completion of this final study, Gene Bio plans submit a
biologics license application to the FDA and secure FDA approval to
market and sell Generx in the United States.
Proposed Financing: We are seeking to raise up to $6.0 million in
a short-term (24 month) debt financing to support our going
forward operations as a C-Round bridge financing which will be
followed (within 12-16 months) by a planned S-1 Registration
Statement initial public financing, that is anticipated to fully fund
the Generx clinical development program. The attached term sheet
provides the terms and conditions covering this proposed financing.
Investors will have a right to receive repayment of principal and
interest at maturity or participate in the planned IPO at a significant
discountto market. Nostrum has agreed to guarantee repayment of
principal at maturity.
Fact Sheet &
Proposed IPO
Bridge C-Round
Debt Financing
The Mechanism of Action: Generx has been designed to improve cardiac perfusion by promoting the formation of
functional coronary collateral blood vessels within the heart through the enlargement of existing arterioles
(arteriogenesis) and the formation on new capillary vessels (angiogenesis). This process, termed “medical
revascularization,” represents a fundamentally new mechanism of action that involves the stimulation and formation
of new biological structures in the heart, as opposed to currently available traditional symptomatic pharmacologic
therapies, which only address the symptoms of angina, or mechanical revascularization, through procedures involving
angioplasty and stent placement or coronary artery bypass graft surgery.
Clinical Findings: The Generx regulatory dossier represents one of the most extensive and advanced gene-based clinical
data platforms ever compiled. In multiple prior clinical studies, the Generx product candidate has been evaluated in
over 650 patients (450 receiving Generx) and appears safe and well-tolerated with findings of efficacy in men and
women, in measures of cardiac perfusion, exercise capacity, and angina status. Specifically, Generx has been evaluated
as a treatment for patients with refractory angina in four prior FDA-cleared, multi-center, randomized and placebocontrolled clinical studies (AGENT 1-4, covering Phase 1|2, Phase 2 and two Phase 2b/3 clinical studies), and one small
Phase 2 international study (ASPIRE). These studies have generated over 2,500 patient years of safety data. Generx has
now been cleared by the FDA for our final Phase 3 clinical study to further evaluate safety and definitive efficacy.
U.S. Market Strategy: To support our go to market strategy, we conducted a survey of U.S. interventional cardiologists to
gauge their experience-based assessment of the prevalence of refractory angina patients, and their openness to integrate
the use of the Generx angiogenic gene therapy product candidate, upon FDA approval, into their clinical practice. The
survey confirmed that all survey physician responders see patients with long-term refractory angina as an unmet medical
need, and all were strongly positive and without reservation about the adoption of Generx following FDA approval. All
cardiologists surveyed felt there is a current need for Generx to treat refractory angina and they would consider using
Generx in their daily practice.
Competitive Factors: We believe that we have a significant clinical lead over even our nearest competitor, and that our
knowledge base, technology platform, a $200 million investment research and development spend by larger pharma
players prior to Nostrum’s acquisition, our FDA cleared treatment algorithm and business model, all represent significant
barriers to entry. Following FDA approval, we believe that Generx will potentially be considered superior in the
competitive marketplace. We believe that Generx therapy will represent a better, faster, and cheaper solution than other
therapies now in development. Our FDA-cleared Ad5FGF-4 manufacturing process offers significant gross margin
opportunities, scalable campaign manufacturing, generates a ready to use plug-n-play product, and has a well-researched
and clinically supported MOA, and receptor-based, cardiac cell targeting. We see little or no direct competition in our
space until 2030.
Potential for Exceptional ROI: With public access to capital markets and sufficient financial resources, an accelerating
Phase 3 clinical study, a NASDAQ listing with stock trading liquidity, on a post-IPO basis, we believe, that there would be
support for an intermediate market capitalization ranging from $200 million to $500 million. Based on the Company’s
business model, market opportunity, overall economics and competitive exclusivity, with the successful completion of the
Phase 3 clinical study, Gene Bio believes that there would be ample evidence to support a Generx terminal value range
of $1.0 billion to $3.0 billion, that could potentially be offered by key strategic players from the pharmaceutical and
biotechnology companies focused in the gene therapy, cardiovascular drugs and biologics and vascular access sectors
operating in the U.S. and internationally.
For additional information, please reference our Annual Report on Form 10-K, dated April 21, 2022, as filed with the SEC,
the IPO Bridge C-Round Debt financing, dated June 22, 2022, and Gene Investor Presentation, dated July 27, 2021, that is
available on the Company’s website below. In addition, please also visit Gene Bio’s website
www.GeneBiotherapeutics.com and our Phase 3 patient recruitment website at www.MyRefractoryAngina.com
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