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Jonathan Poyer

Light M&A But Favorable Regulatory Activity Highlights Innovation and Advancements in Biotech

Updated: Apr 11




The S&P Select Biotech Index retraced back down to the 50-day moving average as investors digested the initial recovery move.



Hotter than expected inflation data has tempered expectations for near term rate cuts. Additional clues into the direction for monetary policy may come at Fed’s upcoming policy meeting (March 19-20). In the broader market, the weight of high momentum stocks in US equity markets reached all time high. The momentum factor has led so far in 2024, lifting the S&P to a 17th record high for the year. M&A has been quiet in recent weeks on the biotech front, but favorable regulatory activity continues to showcase innovative new medicines that are advancing forward.


M&A:


  • AstraZeneca (AZN) announced the acquisition of Amolyt Pharma (private) for $800 million upfront and a potential milestone payment of $250 million. The lead asset eneboparatide is in a Phase 3 trial for hypoparathyroidism

  • Bristol Myers (BMY) notified CytomX (CTMX) that it does not intend to continue the development of partnered program BMS-986288, a next generation anti-CTLA-4 antibody, beyond the current Phase 2 study

  • The HSR waiting period for BMY's pending acquisition of Karuna therapeutics (KRTX) expired

  • The HSR waiting period for Sanofi's (SNY) acquisition of Inhibrx (INBX) expired


Regulatory:


  • FDA's Oncologic Drugs Advisory Committee (ODAC) unanimously voted (11-0) that Legend Biotech's (LEGN) Carvykti had a favorable risk-benefit profile in 2L lenalidomide-refractory multiple myeloma

  • ODAC also voted (8-3) that Bristol Myers Squibb (BMY) and 2-seventy bio's (TSVT) Abecma had favorable risk-benefit in 3L+ multiple myeloma. The early imbalance of deaths in KarMMa-3 trial was attributed to suboptimal bridging, not direct shortcomings from the therapy

  • Geron (GERN) traded up over 70% on a positive FDA Adcom Vote (12-2-0) for imetelstat

  • FDA granted accelerated approval for Madrigal Pharmaceuticals (MDGL) Rezdiffra (resmetirom) for the treatment of adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis). This is a landmark approval for the first drug in this indication. Rezdiffra will be priced at $47K WAC

  • Mirum Pharmaceuticals (MIRM) announced FDA approval of Livmarli for cholestatic pruritus secondary to progressive familial intrahepatic cholestasis in patients ≥5 years old

  • Cybin (CYBN) announced FDA granted Breakthrough Therapy Designation (BTD) to psuchedelic candidate CYB003 for depression and a private placement of $150 million

  • Pacira Biosciences (PCRX) announced PCRX-201 was granted Regenerative Medicine Advance Therapy (RMAT) designation for the treatment of osteoarthritis of the knee

  • Pfizer (PFE) the EC granted market authorization for Pfizer's PREVENAR 20 vaccine against pneumococcal disease for prevention of invasive disease, pneumonia and acute otitis media caused by Streptococcus pneumoniae in infants, children and adolescents from 6 weeks to less than 18


Clinical:


  • Imuneering (IMRX) was cut in half as IMM-1-104 failed to show any RECIST responses in a P1 trial in RAS-mutant solid tumors

  • Spruce Biosciences (SPRB) fell ~75% after announcing negative topline results from the CAHmelia 203 study of Tildacerfont in CAH. SPRB will discontinue tildacerfont and cut workforce by ~21%

  • Acadia Pharmaceuticals (ACAD) announced the Phase 2 ADVANCE-2 trial of Pimavanserin did not meet its primary endpoint of negative symptoms in schizophrenia. Negative Symptom Assessment-16 (NSA-16) total score (-11.8 vs. -11.1; p=0.4825; effect size=0.07) was not significant, but safety and tolerability were consistent with previous clinical trials with a low rate of adverse events


Corporate Updates & Earnings:


  • Urogen Pharma (URGN) reported 4Q23 Jelmyto sales of $23.5 million (+28% Y/Y) vs street $22.6 million estimates despite 340B drug pricing headwinds on gross-to-net (~$4M across 2023). URGN provided 2024 sales guidance of $95-102 million below the street at $108 million


Culling the Herd:


  • Coherus Biosciences (CHRS) announced a 30% RIF

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