Q&A with David Nierengarten, Laura Chico, and Liana Moussatos, Wendbush Securities analysts

Med Ad News talked to Wendbush Securities analysts David Nierengarten, Laura Chico, and Liana Moussatos about various new drug approvals and other recent news updates in the industry. 

Med Ad News: Please discuss the current multiple myeloma drug market and the potential impact of Bristol Myers Squibb/bluebird bio’s recently approved Abecma.

David Nierengarten

David Nierengarten: That is a broad question. Generally speaking, there are several drug classes currently used to treat multiple myeloma–the “imids” (e.g. Revlimid, Thalomid, Pomalyst), proteasome inhibitors (Velcade, carfilzomib, ixazomib), anti-CD38 (Darzalex), and other treatments like Xpovio (selinexor). Most patients try a stem cell transplant. 

Abecma is a CAR-T (engineered T-cell from the patient) targeted to BCMA (B-cell Maturation Antigen). BCMA is found on the plasma cells that make up myeloma, and so targeting the T-cell to it, directs the T-cell to kill that cell. Abecma is the first CAR-T approved in multiple myeloma and we expect it will be incorporated by oncologists at least as readily as the CD19-directed CAR-T therapies approved for DLBCL (diffuse large B-cell lymphoma, another hematological cancer). 

There are many late-stage BCMA targeted drug candidates however, and so we expect a lot of competition from them, not least of which is another CAR-T being developed by Legend Biotech and Janssen. 

Med Ad News: What other promising medicines are on track to emerge from the bluebird bio pipeline and reach the marketplace?

David Nierengarten: Bluebird is also working on removing the clinical and commercial holds from Zynteglo/LentiGlobin, its gene therapy for beta-thalassemia and sickle cell disease. It has another gene therapy for cerebral adrenoleukodystrophy in late stage studies. In oncology, the company is developing another BCMA-directed CAR-T, bb21217, and has brought a TCR therapy for Merkel Cell Carcinoma into the clinic recently. It has other cell and TCR based therapies in preclinical development. 

Med Ad News: What is the market potential of mavacamten, and will the drug’s anticipated regulatory approval justify Bristol Myers Squibb’s $13.1 billion acquisition of MyoKardia?

David Nierengarten: We think so. The market potential for mavacamten, our last published estimates for mavacamten sales were for $3.3B by 2026; MyoKardia was also developing danicamtiv, among other earlier stage drug candidates that could have large market potentials. 

Med Ad News: Please discuss the market expectations for recently launched Cosela as the first FDA-approved therapy that helps proactively deliver multilineage myeloprotection to patients with extensive-stage small cell lung cancer being treated with chemotherapy.

David Nierengarten: We think market expectations could turn out to be conservative for Cosela, as they exceeded our first quarter estimate out of the gate. We think the company still has a lot of prescribers to reach as Cosela was available for just four weeks as of the end of Q1, and so sales could surprise to the upside next quarter too.  

Med Ad News: How is MacroGenics’ recently approved Margenzain being differentiated in the breast cancer marketplace?

David Nierengarten: It could provide a survival advantage in patients, with benefits more pronounced in the majority of patients who carry a certain genotype that responds better to the immune cell activation by the antibody. 

Med Ad News: What are some of the most anticipated new medicines for rare disease expected to emerge from the industry’s product pipeline?

David Nierengarten: I think investors are excited for the first sets of results for in vivo gene editing studies in patients with rare diseases.  

Med Ad News: How is the “TD Spotlight” direct-to-consumer advertising campaign educating patients about tardive dyskinesia and the benefits of Ingrezza? What type of impact could a successful DTC campaign have for Ingrezza’s marketplace success? 

Laura Chico

Laura Chico: The TD Spotlight DTC campaign represents a newer campaign so it would seem to be early days to gauge its true impact thus far. That being said, much of the TD market remains undiagnosed and with VMAT2 inhibitors like Ingrezza being recently incorporated into treatment guidance, we do see it as a good tool to encourage dialog between physicians and patients.

Med Ad News: What is the market potential for newly approved Empaveli as the first targeted C3 therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)? 

Laura Chico: For Soliris- or Ultomiris-refractory patients, we think Empaveli may be a relevant treatment option. However, given the limited size of the treated PNH population (~1,500 patients in the US) and the introduction of newer treatments like ALXN’s Ultomiris, we do think broader conversion to Empaveli may prove challenging.

Med Ad News: What are some of the most anticipated new medicines for rare disease expected to emerge from the industry’s product pipeline? 

Laura Chico: One of the most exciting aspects about the biotech sector’s maturation has been the expansion of the therapeutic “toolbox” and development of novel treatment modalities. Gene therapy efforts in the CNS and metabolic rare disease spaces from companies like PassageBio (PASG) and Ultragenyx Pharmaceutical (RARE) may offer patients one-time treatments. Stoke Therapeutics (STOK) uses unique antisense oligonucleotides to rescue protein expression in orphan disease Dravet Syndrome. Finally, Edgewise Therapeutics (EWTX) holds a small molecule-focused platform attempting to preserve muscle function in rare muscle disorders like Becker Muscular Dystrophy and Duchenne Muscular Dystrophy. 

Med Ad News: What is the current pipeline/regulatory status and market potential for BioMarin’s achondroplasia treatment vosoritide and hemophilia A product valoctocogene roxaparvovec?

Liana Moussatos: Vosoritide/Achondroplasia: PDUFA November 20, 2021 (Deadline for FDA to decide on approval) $680MM gross peak sales in 2025

Roctavian/Hemophilia A: Q1:22 (our est) Two year Ph3 data; Q2:22 resubmit BLA; H2:22 potential approval; $1.5 billion gross peak sales in 2027.

Med Ad News: How is Global Blood Therapeutics transforming the SCD market, where therapeutic innovation and access to care has significantly lagged?

Liana Moussatos: Global Blood has developed a pipeline of sickle cell products starting with OXBRYTA which is the first disease modifying treatment which is efficacious and has a tolerable side effect profile. 

Med Ad News: How is the launch progressing for Tyvaso for the treatment of pulmonary hypertension associated with interstitial lung disease? What other indications is the medicine being studied for?

Liana Moussatos: United Tx does not separate Tyvaso sales by indication so we don’t have specific numbers for PH-ILD and PAH. Recall on April 1, the company announced FDA

approval and launch of Tyvaso in patients with PH-ILD (Please refer to our April 19, 2021 research report for additional details). The Company anticipates a quick Tyvaso PH-ILD uptake (Management expects to double the number of patients within two years of launch). We currently forecast WW net revenue of $547 million in 2026. The company initiated commercial efforts shortly after approval and we look forward to updates in the coming months.

Tyvaso is also being tested in pulmonary hypertension with COPD and IPF

Med Ad News: What is the status and potential outlook for Sangamo’s hemophilia A medicine SB-525/giroctocogene fitelparvovec in partnership with Pfizer? 

Liana Moussatos: SB-525/Hemophilia A Phase I/II two year followup data from high dose cohort in Q1:22 (our estimate), PFE guidance for 2022 for topline data from trial and potential U.S. approval in 2023 (PFE). Our peak sales are $1.5 billion in U.S. in 2029.

Med Ad News: What are some of the most anticipated new medicines for rare disease expected to emerge from the industry’s product pipeline?

Liana Moussatos: There are multiple gene therapy cures being developed for rare diseases. BioMarin has Roctavian for hemophilia A and one for PKU.