Sarepta CEO Doug Ingram on gene therapy for DMD

Sarepta CEO Doug Ingram on gene therapy for DMD
Image source: CNBC Video Screenshot

The following are excerpts from CNBC’s Meg Tirrell’s live interviews from the J.P. Morgan Healthcare Conference, today Monday, January 13th, including: Cigna CEO David Cordani; Sarepta Therapeutics CEO Doug Ingram; Pfizer CEO Albert Bourla; and Novartis CEO Vasant Narasimhan. The interviews aired across CNBC’s Business Day programming. Links to video of the interviews on are provided below.

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All references must be sourced to CNBC.

Cigna CEO David Cordani Speaks with CNBC’s Meg Tirrell on CNBC’s “Squawk on the Street” Today

Watch CNBC’s full interview with Cigna CEO David Cordani

David Cordani on Global Health Services Company:

We think at the core of our business, we're a health services company, we’re global health services company. And the most important thing we do every day is to try to help our customers – all 165 million of them around the world -- live healthier, vibrant, more productive lives. A big part of that is making health care affordable and predictable. So, we think with the outcome of any dimension of the election, affordability, improved predictability at the individual level and then working to simplify programs, is going to be mission critical under any dimensional that manifests itself politically. And we've positioned our company to continue to drive towards that.

David Cordani on Durability:

We've delivered in excess of 40% TSR over the last ten years, on average. So, when you look at the longevity of that over an environment, coming out of the financial recession, going through the ACA, dealing with geopolitical environments, dealing with changes in the environments, 40% TSR on average over an elongated period of time. The conversation—you have the short-term dynamic of ‘What might a legislative disruptive environment do to business models?’ As a company, we've built the business to actually be quite durable.

David Cordani on Pointing Fingers:

There are tens of millions of individuals who are pre-diabetic in the United States today. They will become diabetic in the next few years. How do we help them avoid that in this first place? Through lifestyle management, through diet, through physical activity and supporting through medications. How do we align incentives around that? There is no one party in a $3.5 trillion industry that is the cause of all good or all bad. And we do not think that actually pointing a finger is a constructive way.

CNBC Exclusive: Sarepta Therapeutics CEO Doug Ingram Speaks with CNBC’s Meg Tirrell on CNBC’s “Fast Money Halftime Report” Today

Watch CNBC’s full interview with Sarepta CEO Doug Ingram

SCOTT WAPNER: The biggest health care investing event is taking place in San Francisco right now. And Our Meg Tirrell is live at the JP Morgan Health Care Conference with the CEO of high-flying Sarepta Therapeutics. Meg, it is all you.

MEG TIRRELL: Well, Scott, thank you so much. And Doug Ingram, thank you for being here with us.

DOUG INGRAM: Thank you for having us. We appreciate it.

MEG TIRRELL: Of course, the program that people are looking at closely with you is your gene therapy for Duchenne Muscular Dystrophy, which could just be transformative for this devastating disease. Tell us about the timelines from an investor standpoint when you see this being de-risked, and for a patient standpoint when this could potentially get on the market.

DOUG INGRAM: Well, I think, in one sense every day we are de-risking along the way. And we still have a lot to do at the same time. Just to remind us, so we have a gene therapy for Duchenne Muscular Dystrophy. It relates to this disease that results in a lack of a protein called dystrophin, which is a shock absorber in the muscles.

We are trying to literally insert into the muscles of these children a significantly truncated but still functional version of that same structural protein to try to keep them alive and keep them healthy.

And we just – we had a really fascinating and very significantly impressive data on first four children, both in 2018 and 2019. And we started a study we call Study 102, which is a placebo-controlled trial. We just completed the dosing of all the children in that trial.

It’s about 41 children in that trial. We are going to start another trial if all goes well by around the middle of this year. And we could be in a position, if we are successful, to have a readout on those studies and apply for approval in the U.S. and around the world in 2021 or so.

MEG TIRRELL: And you are not the only company working in this space. Actually, in the next hour we’re going to have the CEO of Pfizer joining us. And they’re also working on gene therapy for DMD. How do you compete with a behemoth like that?

DOUG INGRAM: Well, a couple things. First of all, we are really immodestly the experts in the natural history and the understanding of Duchenne Muscular Dystrophy. So, I do believe we can do a very good job serving the community. And we have done that already. We have two therapies right now treating patients who have Duchenne Muscular Dystrophy. I think it is a very positive signal when we see companies like Pfizer taking an interest in trying to advance therapies that might treat Duchenne Muscular Dystrophy.

It is one of those things that will give parents and patient a lot of hope that we are going to find solutions for them. With that said we are razor-focused on our therapies and our approach, and we are very excited about the results we’ve seen to-date. And we are going to stay essentially head down executing getting our therapy out to those patients if it works as soon as possible.

MEG TIRRELL: And you’ve already got two drugs on the market for this disease. Which, you know, not as transformational as the gene-therapy in your pipeline. But you have to do a confirmatory study of your first drug because you got conditional approval from it from the FDA. And some folks have said the timelines are more delayed than expected. When can we see the readout?

DOUG INGRAM: So, that read out will probably occur somewhere in the 2023, 2024 range. We do have a confirmatory trial for Exondys ongoing right now. Essentially it is looking at our current therapy Exondys at 30 milligrams per kilogram, versus a much higher dose of the same therapy to see which of those doses would be more beneficial to patients. And we are aggressively pursuing that. That is Exondys. That treats about 13% of the children with Muscular Dystrophy.

We received an approval in December of last year for a therapy called Vyondys. That treats another 8% of children with Duchenne Muscular Dystrophy. And we’re already serving the community with Vyondys now.

And then, we’ve just started a rolling submission, and our goal is to get it approval as soon as possible, hopefully this year, for what’s called Casimersen, another 8% of children with Duchenne Muscular Dystrophy. So, if we are successful, by the end of this year we could be in a place where we have more than doubled the number of patients who are benefiting from our Morpholino technology, this so called PMO that we have.

CNBC Exclusive: Pfizer CEO Albert Bourla Speaks with CNBC’s Meg Tirrell on CNBC’s “The Exchange” Today

Watch Pfizer CEO Albert Bourla’s first TV interview with CNBC

Albert Bourla on New Pfizer:

Our new Pfizer, the remaining Pfizer, that will be 20% smaller in revenue, retains this new base, all the growth drivers and the entire pipeline. So, now the company is becoming best in class revenue growth story, sustainable for the long-term and with a balance sheet that is relatively unlevered. So, I think we can do miracles.

Albert Bourla on Acquisitions:

We never say never to any opportunities. But strategically, we are very interested in acquiring or in licensing phase two, phase three projects. Projects that could get products in 2004, 5, 6, 7. And in six therapeutic areas we are mastering. This is where we have the best scientists that they can select rightly, make fewer mistakes in selection. But also, they are the scientists that can develop the best products.

Albert Bourla on Policies:

Well, I think our role, it is not to focus on the politics, but to focus on the policies. So, whoever is in the administration and with both sides of the aisle, we try to promote policies that are pro-innovation. Means that all this bio-tech crown of the jewel industry of the United States, or the bio-pharmaceutical companies, will be able to find medicines that the people need. And pro-patient policies, which means policies that would make those medicines accessible for all.

Novartis CEO Vasant Narasimhan Speaks with CNBC’s Meg Tirrell on CNBC’s “Closing Bell” Today

Vasant Narasimhan on UK Deal / New Cholesterol Vaccine:

The idea here is with the UK government we signed a partnership that allows them to really work to drive a utilization of this medicine. It’s really hundreds of thousands of patients. It’s a pretty revolutionary agreement. It kind of takes population health of vaccine-based approach to cardiovascular disease. So, we’re pretty excited.

Vasant Narasimhan on Cost Effective for UK / New Cholesterol Vaccine:

The UK government believes this will be a highly cost-effective intervention. I think that’s why they’re very excited about it. No numbers yet. But I think they believe this will be an important contributor to their ability to hit their targets to reduce cardiovascular disease.

Vasant Narasimhan on Drug Pricing:

We really commit to pricing our medicines in a value-based way. And, being thoughtful about how we think about any pricing increases.

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Jacob Wolinsky is the founder of, a popular value investing and hedge fund focused investment website. Jacob worked as an equity analyst first at a micro-cap focused private equity firm, followed by a stint at a smid cap focused research shop. Jacob lives with his wife and four kids in Passaic NJ. - Email: jacob(at) - Twitter username: JacobWolinsky - Full Disclosure: I do not purchase any equities anymore to avoid even the appearance of a conflict of interest and because at times I may receive grey areas of insider information. I have a few existing holdings from years ago, but I have sold off most of the equities and now only purchase mutual funds and some ETFs. I also own a few grams of Gold and Silver
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