medcitynews.com Open in urlscan Pro
2606:4700:20::ac43:45a1  Public Scan

Submitted URL: http://go.pardot.com/e/710433/tential-edge-over-sarepta-med-/f5m6g/512790016?h=Fe5kCOS2UnAXhCo7kNb7KOS6YJWEXhZxrrZ0TH...
Effective URL: https://medcitynews.com/2022/05/pepgen-ipo-nabs-108m-for-muscular-dystrophy-drug-with-potential-edge-over-sarepta-med/
Submission: On May 09 via api from US — Scanned from DE

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Pharma, BioPharma


PEPGEN IPO NABS $108M FOR MUSCULAR DYSTROPHY DRUG WITH POTENTIAL EDGE OVER
SAREPTA MED


PEPGEN’S TECHNOLOGY IMPROVES THE DELIVERY OF A THERAPY TO MORE TISSUE TYPES AND
THE CLINICAL-STAGE BIOTECH PLANS TO USE ITS IPO CASH TO CONTINUE DEVELOPING ITS
LEAD PROGRAM FOR DUCHENNE MUSCULAR DYSTROPHY. MEANWHILE, EYE PRODUCTS GIANT
BAUSCH + LOMB RETURNED TO THE PUBLIC MARKETS AS A STANDALONE COMPANY. BOTH THE
PEPGEN AND BAUSCH + LOMB IPOS PRICED BELOW THEIR RESPECTIVE PRICE RANGES.

By Frank Vinluan

Post a comment / May 6, 2022 at 1:46 PM
1Shares

 

The rare disorder Duchenne muscular dystrophy is treated with genetic medicines
that get skeletal muscle cells to produce a version of the key protein that they
lack. Though these therapies slow progression of this disease that robs patients
of the ability to walk, patients typically die from Duchenne’s effects on the
heart and breathing. Biotech company PepGen is developing a drug that delivers
its therapeutic payload not only to skeletal muscle, but also to the diaphragm,
cardiac muscle, and the central nervous system. PepGen’s scientists believe this
broader delivery capability gives it an edge and the clinical-stage company has
joined the public markets as it sets out to prove its approach.



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Cambridge, Massachusetts-based PepGen initially planned to offer 7.2 million
shares in the range of $13 to $15 each, which would have raised $108 at the $14
per share midpoint. The company was able to raise that amount, but it had to
offer more shares do so. Late Thursday, PepGen priced its offering of 9 million
shares at $12 apiece. Those shares began trading on the Nasdaq Friday under the
stock symbol “PEPG.”

For diseases with a known genetic cause, one therapeutic approach is an
antisense oligonucleotide, a type of therapy made from small pieces of synthetic
DNA or RNA. The four FDA-approved therapies for Duchenne—three from Sarepta
Therapeutics and one from NS Pharma—are all antisense oligonucleotides. PepGen
said in its IPO filing that the limiting factor for oligonucleotide-based drugs
is delivery.

“On their own, oligonucleotides therapeutics are not readily distributed to
heart and skeletal muscle, the key tissues affected in neuromuscular diseases,
and are not efficiently taken up into these cells,” the company said. “To
address this challenge, we engineered our proprietary EDO technology to optimize
tissue penetration, cellular uptake and nuclear delivery, which we believe may
enhance the therapeutic activity of oligonucleotides and improve the
tolerability of these genetic medicines.”

EDO stands for Enhanced Delivery Oligonucleotide. This technology platform
engineers peptides so that they can better carry oligonucleotide cargos to
desired tissue destinations. In preclinical research, PepGen has shown its
technology can transport oligonucleotides into tissue types that include smooth,
skeletal, and cardiac muscle, as well as the central nervous system.

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PepGen’s lead program, called PGN-EDO51, is intended to treat Duchenne patients
whose disease can be addressed by getting the cell’s protein-making machinery to
skip exon 51, one of the segments of a gene containing the code that is
translated into a protein. This approach leads to a shortened version of the key
muscle protein that Duchenne patients lack.

Exon skipping is the same approach taken by Sarepta’s first FDA-approved
Duchenne therapy, Exondys 51. Sarepta is developing a next-generation exon
51-skipping drug that uses cell-penetrating peptides to improve delivery. That
therapeutic candidate, SRP-5051, is currently in Phase 2 testing. PepGen said in
its IPO filing that it conducted a monkey study compared its experimental drug
against a molecule that’s equivalent to Sarepta’s next-generation exon-skipper.
According to PepGen, results showed a greater than 70% increase in exon 51
skipping in skeletal muscle, including the diaphragm. The PepGen therapy also
showed greater activity.

Last month, PepGen began a Phase 1 single-ascending dose study enrolling healthy
volunteers in Canada. Preliminary data from the clinical trial are expected by
the end of this year. The EDO platform has yielded a second program, PGN-EDODM1,
which the biotech is developing as a treatment for myotonic dystrophy type 1
(DM1), a rare muscle disorder that currently has no FDA-approved treatments.
PepGen said in its prospectus that it expects to submit an investigational new
drug application for a DM1 therapy in the first half of next year.

A second Duchenne therapy, PGN-ENDO53, is being developed to address patients
who can be treated by skipping exon 53. PepGen said it expects to begin a test
of that drug in monkeys in the second half of 2022. Besides further Duchenne
research, PepGen said it plans to study potential applications of its technology
in additional indications that include neuromuscular diseases and neurological
disorders.

The science behind PepGen’s drugs is based on more than a decade of research
from the labs of Matthew Wood, a professor of neuroscience at the University of
Oxford, and Mike Gait, emeritus scientist at the Medical Research Council of
United Kingdom Research and Innovation. PepGen was founded in 2018; in 2020, the
biotech closed a $45 million Series A round of funding. Prior to the IPO, PepGen
had raised $163.9 million, the company said in its prospectus. Its most recent
financing was a $112.5 million crossover round that closed last August.

RA Capital is PepGen’s largest shareholder with a 23.7% stake after the IPO,
according to the prospectus. Oxford Sciences Enterprises, a firm that takes a
stake in Oxford University science spinouts, owns 18.3% of the company. As of
the end of 2021, PepGen had $132.8 million in cash, the prospectus shows.
Combined with the IPO proceeds, PepGen plans to spend about $70 million on
completing Phase 1 testing of PGN-EDO51 and continuing on to a planned Phase 2a
study. Another $45 million is set aside for completing a Phase 1/2 clinical
trial for PGN-EDODM1, while about $30 million is earmarked for further
development of its drug pipeline and platform. Following the IPO, PepGen
estimates its cash holdings will carry the company into the first half of 2025.

Bausch + Lomb raises $630M in return to the public markets

Bausch + Lomb is trading on the New York Stock Exchange again after completing
an IPO that comes 15 after the iconic eye products company was taken private. On
Thursday evening, the company priced its offering of 35 million shares at $18
apiece, well below the $21 to $24 price range the company had previously set. In
addition to its debut on the NYSE, Vaughan, Ontario-based Bausch + Lomb also
listed shares on the Toronto Stock Exchange. The stock trades on both exchanges
under the stock symbol “BLCO.”

The Bausch + Lomb IPO marks the company’s separation from Bausch Health
Companies (formerly Valeant Pharmaceuticals), the company that acquired it in
2013. Bausch + Lomb was initially taken private in 2007 with its acquisition by
private equity firms for $4.7 billion. Bausch + Lomb won’t receive any of the
IPO proceeds, which will go to Bausch Health. In advance of the IPO filing in
January, Bausch Health CEO Joe Papa said that his firm will use the cash from
the IPO to pay down debt.

Public domain image by the Centers for Disease Control and Prevention



1Shares

TOPICS

biotech IPO, Cambridge, Clinical Trials, duchenne muscular dystrophy,
Massachusetts, oligonucleotide, PepGen, rare diseases




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patients and providers and the potential for hybrid care models to improve the
patient experience, health outcomes, and deliver cost savings.

Wheel and MedCity News





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TSX Venture Exchange has a strong history of helping early stage health and life
sciences companies raise patient capital for research and development purposes.

Daniel Lubienietzky, Toronto Stock Exchange




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