BridgeBio: Expected November 2024 PDUFA Date Could Change Momentum (NASDAQ:BBIO)

Last time we talked about BridgeBio Pharma, Inc. (NASDAQ:BBIO), was in a Seeking Alpha article titled “BridgeBio Pharma: Acromidis FDA submission and top results on deck.“The principal thing to note about this article is that I mentioned that the new drug application (NDA) of this drug for the treatment of patients with amyloid transthyretin cardiomyopathy (ATTR-CM) has been submitted to the FDA. However, at that time there was to be a review period during which the agency would decide whether to accept the submission of this application.

I am pleased to report that not only has the FDA accepted Acoramidis’ NDA submission for the treatment of this patient population, but it has set a PDUFA date of November 29, 2024. Even better, the Marketing Authorization Application (MAA) of Acoramidis for this patient population was also accepted setting for success on European territory. Speaking of which, another big event that has happened since we last talked about it is that it has entered into a licensing agreement with Bayer (OTCPK: BAYRY). This big pharma would have licensing rights to sell this drug to this patient population in Europe.

This brought in a lot of money, along with the fact that BridgeBio now has a huge pharmaceutical partner to market the drug in that territory. Regarding the use of BBP-631 for the treatment of patients with congenital adrenal hyperplasia (CAH), there is a go/no go update planned in August 2024 for this. That is, at that time a program update will be provided stating whether they intend to continue development with this specific program.

The Acoramidis review date is set with the potential to target a large ATTR-CM market

As I said above, things are looking very good for BridgeBio because The FDA has accepted its NDA for Acoramidis for the treatment of patients with ATTR-CM. That being said, the set PDUFA date by which the FDA will review whether this drug should be approved for the treatment of this patient population has been set for November 29, 2024. What’s even better about this set review date is that at the time acceptance, it was announced that the FDA would not hold an advisory committee to discuss it.

What’s even better is that there might be an opportunity for this drug to be approved for European territory as well. This is because the European Medicines Agency (EMA) has accepted Acoramidis’ Marketing Authorization Application (MAA) for this patient population as well. Both review acceptance applications for this drug bode well for this company. There is even the possibility of expanding this drug to other territories through other regulatory filings that are planned in the coming years.

In terms of being able to commercialize Acoramidis for ATTR-CM, BridgeBio is not going it alone. Instead, it chose to license European rights to a partner named Bayer. This large pharmacy will be responsible for marketing this drug in this territory. I think this is a good deal for BridgeBio and that’s because it gets a few things done. The first element being that it brought in a large amount of cash. In fact, he received $300 million up front and in the short term payment of stakes because of her. From there, he is eligible to receive additional undisclosed payments based on sales and tiered royalties of at least thirty percent. The second aspect is that it allows Bayer to be responsible for marketing this drug in European territories. This allows BridgeBio to focus on commercialization in the US and other markets.

In order for the company to be in a position to submit regulatory applications for Acoramidis, it had to meet the primary objective of phase 3 study ATTRIbute-CM. This late-stage study recruited up to 632 ATTR-CM patients. Patients were randomized to receive either Acoramidis or placebo twice daily. The primary endpoint of this study was all-cause mortality, cumulative frequency of CV hospitalization, change from baseline in NT-proBNP, and change from baseline in the 6-minute walk test (6MWT). This the primary objective was met with statistical significance with an earnings ratio of 1.8 with a p-value of p

The key question is whether this will be enough to counter other competitors in the market. For example, Pfizer (PFE) has the drug approved Vyndamax (Tafamidis)which has been approved to treat these ATTR-CM patients. The thing is, it’s not clear whether cardiologists will switch patients from entrenched Vyndamax to Acoramidis. However, such a change can happen if BridgeBio can continue to highlight the key differences as stated above, which is a greater separation between ACM and CVH in the 3rd month. Not only that, but where it could compete with Pfizer is if it can. get a lower price for Acoramidis.

One downside to BridgeBio is that this treatment must be taken orally twice a day, while Vyndamax only needs to be taken once a day. Another competitor would be on his name Alnylam Pharmaceuticals (ALNY) with its drug Amvuttra (vutrisiran), which has already received FDA approval for this drug for the treatment of patients with transthyretin amyloidosis (ATTR). However, it is in the process of advancing this drug also for the treatment of patients with ATTR-CM.

Speaking of which, just a few months ago on June 24, 2024, announced positive results from its phase 3 HELIIOS-B trial using this drug to treat this patient population. Where this company might have an advantage is that its drug is given as a subcutaneous injection once every three months. This will be a highly competitive area, but if the company can price its drug lower compared to the others, plus show an efficacy advantage over these other drugs, then it will still be poised to gain market share. Transthyretin Amyloidosis Treatment Market Size is it is projected to reach $11.2 billion by 2032. This market continues to grow and currently Pfizer’s Vyndamax dominates it. However, I believe that if some competitive advantages could be established with Acoramidis, then the company should be able to hold a market share in this space. It believes that, as an estimate, it may be able to capture between 25% and 40% market share for this ATTR-CM market.

financial

right 10-Q Filing SECBridgeBio Pharma had cash, cash equivalents, marketable securities and short-term restricted cash of $587.2 million as of June 30, 2024. The reason for the cash on hand is because of several financial transactions it made to obtain all of these additional funds. . Such financial transactions established to obtain financing were the following:

• Net proceeds from the term loan under the credit facility with Blue Owl of $434 million
• Net proceeds from various equity financing offerings of $314.8 million
• Proceeds from the sale of equity investments of $63.2 million
• Special cash dividends received on equity investments of $25.7 million.

The fact is that this biotech will continue to make financial transactions, which could affect the stock price in some way. Why am I saying this? That’s because in its 10-Q SEC filing, it says it believes it has one cash trackor sufficient cash on hand for at least the next 12 months.

Business risks

There are several risks that investors should be aware of before investing in BridgeBio Pharma. The the first risk to consider would be regarding the development of Acoramidis for the treatment of patients with ATTR-CM. The PDUFA date for this drug for the treatment of this patient population has been set for November 29, 2024. The risk here is that there is no assurance that the FDA will approve this drug for marketing in the United States. In addition, there is currently no advisory committee planned to review this drug. However, while it is not guaranteed to happen, it is possible that one will be summoned later. Ultimately, rejection of the drug for approval by the FDA would mean a loss of an estimated $500 million in payout. This would indeed be a considerable loss in terms of funding.

The the second risk to be considered would be with the MAA submission of Acoramidis for ATTR-CM to the European Medicines Agency. There is no assurance that this medicine will be approved for marketing in European territories. If approved, the expected commercial launch of the drug would take place in 2025. Even if this drug is marketed in Europe, there is no guarantee that sales of the drug will do well.

The the third risk to consider would be regarding competing drugs in the ATTR-CM market space. As stated above, Pfizer is the main competitor to Vyndamax for these patients. For BridgeBio to compete with it and other companies for market share, it will need to find competitive advantages. A key area where they could establish such an advantage would be in terms of lower prices. If it can price its drug lower than all the others, then it will definitely be in a good position to gain market share. The risk is that there is no assurance that it will be able to price its drug lower compared to Vyndamax or other competing ATTR-CM treatments.

The the fourth and final risk to consider would be with respect to the development of BBP-631 for the treatment of patients with congenital adrenal hyperplasia or CAH. There is a program update for this candidate planned for sometime during August 2024. Such a program update will dictate whether they intend to move forward with this candidate or remove it from the pipeline. The risk here is that removing this program will result in the loss of a significant program. One of these is a considerable market opportunity.

Conclusion

I think it was important to update the Bridge Bio perspectives. This is because not only was Acoramidies’ NDA regulatory submission for ATTR-CM accepted by the FDA, but a PDUFA date of November 29, 2024 was set for it.

The global transthyretin amyloidosis treatment market is a billion dollar market opportunity. If this company can somehow get regulatory approval for it in the United States and a few other territories, then they might be able to capture a good chunk of it. Despite the competition, the conservative estimate is that it could capture between 25% and 40% of the ATTR-CM market share. Even at the low end of the spectrum getting just 25% market share would still be a win in my eyes.

Editor’s Note: This article discusses one or more securities that are not traded on a major US exchange. Please be aware of the risks associated with these stocks.