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IPO Report: Prosensa Holding B.V.

By  +Follow June 27, 2013 11:50AM
Tickers Mentioned:

Based in Leiden, NL, Prosensa Holding B.V. (RNA) scheduled a $60 million IPO with a market capitalization of $408 million, at a price range mid-point of $12 for Friday, June 28, 2013.

  • F1-A filed June 18, 2013
  • Manager, Joint Managers: J.P. Morgan; Citi
  • Co-Managers: Leerink Swann; Wedbush PacGrow; KBC Securities; Trout Capital


RNA is working on a medical solution to Duchenne muscular dystrophy (DMD), a genetic disease. DMD occurs mostly in boys. Symptoms typically appear between ages 1-4 years old.

The results of clinical trials may indicate that drisapersen may lead to stabilization of the disease. But stabilization is not a molecular cure, which is what some of the recent stem cell IPOs seem to offer as a possiblity.

Another company, PTCT, focuses on a different subset of DMD and was up 10% on its IPO on Thursday, June 20, 2013.

RNA reports in Eurodollars, so to do any financial analysis in the U.S. dollars it's necessary to convert to dollars.


100% of revenue comes from licensing and collaboration agreements with GlaxoSmithKline (GSK), and RNA works closely with Leiden University Medical Center (LUMC)


Post-IPO RNA will have $107 million in cash. The ratio of cash to market capitalization will be 26%, at the price point mid-point of $12. RNA's accumulated deficit is $56 million.

Valuation Ratios

IPO Mrkt

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annualizing Q1 '13

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Prosensa Holding B.V. (RNS)









Relative to price-to-book ratios, RNA is priced at about double PTCT, which IPO'd June 20. Although some IPO watchers believe RNA will go up on the IPO, it seems product to avoid RNA based on the comparison with PTCT.

To put the above conclusions and observations in context, the following is reorganized, edited and summarized from the full S-1 referenced earlier:


RNA is a biotechnology company engaged in the discovery and development of ribonucleic acid-modulating, or RNA-modulating, therapeutics for the treatment of genetic disorders.

RNA's primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease.

RNA"s clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy, or DMD. Each of RNA's DMD compounds has been granted orphan drug status in the United States and the European Union.

About DMD

RNA is working on a medical solution to Duchenne muscular dystrophy , a genetic disease. DMD occurs mostly in boys. Symptoms typically appear between ages 1-4 years old. The main sign of DMD is muscle weakness that worsens over time. Before age five, the muscles in the legs, arms, and trunk begin to weaken. Later in the disease the heart and respiratory muscles weaken.

DMD is a progressive, serious disease and is caused by a genetic mutation. The mutation causes the gene to make inadequate amounts of a protein called dystrophin. This protein is needed to keep muscles intact.

Collaboration with GlaxoSmithKline

In 2009, RNA entered into an exclusive worldwide collaboration with GSK for the development and commercialization of RNA-based therapeutics for DMD, with GSK exclusively licensing worldwide rights to develop and commercialize drisapersen and obtaining an option to exclusively license PRO044.

In addition, GSK has the option to exclusively license either PRO045 or PRO053 and the further option to exclusively license either PRO052 or PRO055. PRO045 is paired with PRO053, and PRO052 is paired with PRO055, because each product candidate in the respective pairings addresses a similar-sized patient sub-population and is at a comparable stage of development.

RNA will retain the full rights to the product candidates that are not licensed by GSK, and for each product candidate licensed by GSK from each of the pairings of product candidates described above, RNA will retain the option to certain commercial rights in a selected European territory. All of other DMD compounds fall outside of the scope of the agreement, and RNA intends to develop and commercialize them or consider partnerships.

GSK financial terms

Upon entering into the agreement with GSK, RNA received a $23 million nonrefundable upfront payment from GSK and RNA has received $63.5 million in total under the agreement. Including amounts already paid, RNA is eligible for up to $677 million in total milestone payments under the agreement.

RNA is also entitled to receive percentage royalties in the low teens on future global sales of drisapersen and each of the other compounds that GSK licenses and successfully commercializes.

More about DMD

DMD is one of the most prevalent rare genetic diseases globally affecting up to 1 in 3,500 boys and is invariably fatal. There is currently no approved disease-modifying therapy for DMD. The progressive muscle-wasting that characterizes this disease is caused by inadequate production of dystrophin, a protein necessary for muscle function, as a result of mutations in the dystrophin gene. The different mutations, which are mostly deletions of one or more exons, found in the dystrophin gene result in distinct sub-populations of DMD patients.

RMA is designing product candidates to address several sub-populations using RNA's platform technology. The first product candidate, drisapersen, can address a variety of mutations in the dystrophin gene, such as a deletion of exon 50 or exons 48 to 50.

RNA financing

Since 2002, RNA raised $75.6 million from private placements of equity securities, including to a number of venture capital firms. In addition, RNA has received grants and loans from several DMD-focused patient advocacy organizations to support research of therapies for DMD. As of March 31, 2013, RNA had $48 million in cash and cash equivalents.

Clinical development

Drisapersen is being developed in collaboration with GlaxoSmithKline, or GSK. Drisapersen aims to restore dystrophin expression and improve muscle condition and function in the largest known sub-population of DMD patients.

In clinical trials, drisapersen has been shown to produce dystrophin expression and have a beneficial therapeutic effect on DMD patients. A Phase II placebo-controlled study of drisapersen in 53 DMD patients was completed and demonstrated a statistically significant and clinically important difference in the primary endpoint, which was the distance walked in the six minute walk test, or 6MWD, between the placebo group and the continuous active-treatment group at a dose of 6 mg/kg/week after 24 weeks.

This clinically meaningful benefit was maintained after 48 weeks of treatment, and drisapersen was well tolerated throughout the duration of this study. Preliminary results suggest that treatment with drisapersen was in general associated with increased levels of dystrophin expression when compared with pre-treatment levels.

Drisapersen successfully completed a twelve-patient Phase I/II study, and all patients were enrolled in an open-label extension study which has been ongoing since August 2009. The results indicate that drisapersen may lead to stabilization of the disease, as evidenced by an improvement or a slower than expected decline in the 6MWD, and the ongoing study continues to provide safety and tolerability data.

A pivotal Phase III study of drisapersen was initiated in December 2010, and results are expected in the fourth quarter of 2013. This study is a randomized, double-blind and placebo-controlled trial, assessing drisapersen at a dose of 6 mg/kg/week in 186 boys. The primary endpoint is the 6MWD at 48 weeks.

To date, over 300 patients have participated in clinical studies of drisapersen at more than 50 trial sites in 25 countries, and patient retention rates through March 2013 averaged 96% across all drisapersen clinical studies.

Follow-on DMD compounds

PRO044, the next most advanced product candidate, addresses a separate sub-population of DMD patients. RNA developed PRO044 using our exon-skipping technology to generate a product candidate with the same mechanism of action that is used by drisapersen.

PRO044 is currently in a Phase I/II study in Europe, which RNA expects to complete in the second half of 2013. RNA has four additional earlier-stage compounds that address other distinct sub-populations of DMD patients.

Of these, PRO045 entered clinical trials in the first quarter of 2013, and DMD anticipates that PRO053 will enter clinical trials in mid-2013. PRO052 and PRO055 are in advanced preclinical development.

RNA has started a research program, PROSPECT, which includes a new and innovative application of RNA's exon-skipping technology platform to specifically target rarer mutations in the dystrophin gene.

Intellectual property

RNA has entered into a collaboration arrangement with LUMC for the reciprocal licensing of RNA's and LUMC's individual and joint intellectual property rights in relation to certain patent rights and know-how rights, which have been the basis of RNA's research and development of treatment targeting DMD.

5% shareholders pre-IPO

ABV IV Holdings N.V., 21.4%
LSP Prosensa Pooling B.V., 21.4%
New Enterprise Associates 13, L.P., 20.8%
Gimv N.V., 9%
Idinvest Partners, 9%
MedSciences Prosensa Holding B.V., 6.9%

Use of proceeds

RNA expects to net $53 million from its IPO.

RNA currently expects that it will use the net proceeds from this offering, together with cash and cash equivalents on hand, as follows:

• $82 million to fund the current DMD development portfolio for which RNA bears expenses (PRO045, PRO053, PRO052 and PRO055), PROSPECT and DMD-support projects, including the DMD natural history study;

• $11 million to fund non-DMD projects, including DM1 and HD; and

• the remainder for working capital and other general corporate purposes.

DISCLOSURE: The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions.

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Results for RNA
25 Oct 14 04:19:27
RNA 12.01 Stock Charts $RNA Prosensa Holding N.V. (NASDAQ) Last Updated: 10/25/2014 06:19:26 RNA Stock Chart - http://t.co/bwv7qbjYoA
24 Oct 14 13:31:23
$RNA Nasdaq stocks posting largest percentage increases http://t.co/3F6IwXoVBZ #BiotechTraderHandbook--> http://t.co/JDdc1OTfZF
24 Oct 14 07:45:13
RT @BioBreakout: $RMTI $OXGN $RNA $SRPT Biotech Stock Mailbag: Rockwell Medical, Oxigene, Prosensa, Sarepta http://t.co/O9sXVKAgJs via @The…
24 Oct 14 06:26:24
RT @BioBreakout: $RMTI $OXGN $RNA $SRPT Biotech Stock Mailbag: Rockwell Medical, Oxigene, Prosensa, Sarepta http://t.co/O9sXVKAgJs via @The…
24 Oct 14 05:42:15
$RMTI $OXGN $RNA $SRPT Biotech Stock Mailbag: Rockwell Medical, Oxigene, Prosensa, Sarepta http://t.co/O9sXVKAgJs via @TheStreet
Penny Stock Secret
23 Oct 14 21:58:49
The Hottest Way to Make Millions From Stock Market.Click HERE http://t.co/SVqoMkrw2z .. $ATOS $HIFS $RNA
John Hall
23 Oct 14 16:18:42
@PinkyG123 you don't walk away from Multi Million investments $GSK $RNA
Dieter Hovekamp
23 Oct 14 08:39:09
RT @AndyBiotech: Milo's #GeneTherapy Improves Walking Ability in BMD; DMD Trial in Planning. http://t.co/vWajgVwxBD http://t.co/KnH0AXTuca…
Andy Biotech
23 Oct 14 08:38:27
Milo's #GeneTherapy Improves Walking Ability in BMD; DMD Trial in Planning. http://t.co/vWajgVwxBD http://t.co/KnH0AXTuca $SRPT $RNA $PTCT
John Hall
22 Oct 14 20:17:39
Condolences to the Peek family of Michigan, Patrick 16 lost his battle to #DMD. It doesn't stop and it's horrible $SRPT $RNA $PTCT
22 Oct 14 17:26:01
22 Oct 14 11:36:36
RT @themicrokid: $SRPT $RNA followers should be listening to hash tag #NORD2014 per Pinneapple on IV and I agree on #NORD2014
micro kid
22 Oct 14 11:18:52
$SRPT $RNA followers should be listening to hash tag #NORD2014 per Pinneapple on IV and I agree on #NORD2014
micro kid
22 Oct 14 11:15:13
@zwerp2000 @bradloncar @Sport234a $SRPT $RNA seems like attorneys doing what they do best! running up billable hours! & Wash DC full of ...
Alexander Morris
22 Oct 14 10:55:27
$RNA: At 3MoHIGH=11.98, hits:3, Rng:35%, ch:+0.3%[1.9%offHi], FltBO+, tf:1110000, LowFlt[4.9M], HiPctSht[44%:7d], PrvClGap-[-2.2%]
Alexander Morris
22 Oct 14 09:46:33
$RNA: At 3MoHIGH=11.98, hits:2, Rng:35%, ch:+0.3%[1.9%offHi], FltBO+, tf:1111000, LowFlt[4.9M], HiPctSht[44%:7d], PrvClGap-[-2.2%]
micro kid
22 Oct 14 09:00:20
@zwerp2000 @bradloncar @Sport234a $SRPT $RNA US patent interference, parties can't agree as to wehther settlement discussions have begun
22 Oct 14 07:54:48
$RNA short interest 19% $SRPT Short interest 33%. What's wrong with this picture?
22 Oct 14 06:44:58
Prosensa Holding NV Upgraded by JPMorgan Chase & Co. to “Overweight” $RNA http://t.co/PHuE8a9POR
Anthony DePaola
21 Oct 14 18:58:50
RT @dougheuringaria: $rna $srpt news flow data points http://t.co/NNSjLPbN3q
21 Oct 14 18:09:28
@CaptainFuture__ $RNA was abandoned by GSK.(wonder why a big pharma did that). RNA side effects are horrendous. Do ur research peeps.go SRPT
21 Oct 14 17:58:59
@BioTecIsFuture well, they certainly won't appr. $RNA due to severe side effects- why do u think GSK pulled out of partnering with. Go $SRPT
21 Oct 14 17:54:16
@dougheuringaria $Rna is laughable- u think GSK would end partnership if they were legit. dah... GSK abandoned cause of severe side effects
21 Oct 14 17:29:20
Jp $rna swing factors http://t.co/zvIk2RblXu
21 Oct 14 15:03:26
RT @dougheuringaria: $rna $srpt news flow data points http://t.co/NNSjLPbN3q
Seung Yoo
21 Oct 14 14:46:25
@dougheuringaria is $RNA really holding a placebo controlled trial for drisa? Can't be true. Who would enroll in that at this point?
21 Oct 14 13:52:25
$rna $srpt news flow data points http://t.co/NNSjLPbN3q
Abby Nachtomi
20 Oct 14 22:38:26
@sububoston if I remember correctly $RNA didn't track lung function data in their trial.
20 Oct 14 22:18:33
@AndyBiotech @adamfeuerstein Does $RNA have pulmonary data like $SRPT.
20 Oct 14 15:55:25
$RNA - Prosensa Holding B.V. http://t.co/aKgysE3w5W
Stephen P Kendal
20 Oct 14 13:26:18
US #HighVolume #BullishBuy Signals: $FLXT $DPW $BSET $FUJIY $SNFCA $BFDI $NDEV $NLNK $RNA $UUU $XNCR $PROW $PFSW http://t.co/dYT29xMagm
Shiraz Lakhi
20 Oct 14 12:34:32
Final Hour: HEALTHCARE #stocks trading UP On STRONG 150%+ Avg-Volume: $AMGN $IMGN $ANGO $USPH $RNA ... (click "View") http://t.co/pjcuaarzUe
20 Oct 14 12:32:24
Upgrades 10/20: $RNA, $AEM, $CBSO, $MU, $ETFC, $ALGN, $SHPG, $ALSN, $RCI, $SWFT, $PPG, $TUBE, $CNX http://t.co/BlPfIWAdeZ
Stephen P Kendal
20 Oct 14 12:05:10
$RNA Prosensa Holding NV. #HighVolume buying Mon-20-Oct. #Vol=428,241. #52WkH/L=$13.32/$3.43. #Price=$11.91⇑+4.9%. S1=⇗ ST=⇗ MT=⇗ LT=⇗
20 Oct 14 11:24:00
@TomSilver39 Yeah, and??? That news has been known for months and is already baked in. If $RNA gets approved, you can be certain $SRPT will.
20 Oct 14 11:16:04
@TomSilver39 FDA lowered the bar for $SRPT AA because of $RNA failure. Ironically, $RNA back from dead due to lowered bar.
20 Oct 14 11:12:40
@TomSilver39 It has no effect on $SRPT either way. At this point, most have assumed both will be approved. $RNA
Dan Marks
20 Oct 14 10:44:55
@TomSilver39 Hmm, effective subgroup for $PTCT was advanced disease, and for $RNA early disease, would think that might be negative?
Stephen P Kendal
20 Oct 14 10:25:39
$RNA Prosensa Holding NV. #HighVolume buying Mon-20-Oct. #Vol=328,385. #52WkH/L=$13.32/$3.43. #Price=$11.92⇑+5.0%. S1=⇗ ST=⇗ MT=⇗ LT=⇗
Trader Tom
20 Oct 14 09:01:36
$RNA +30% from 10/13 watch list ..... gotta love it when a plan comes together
US Banking News
20 Oct 14 08:18:02
JPMorgan Chase & Co. Upgrades Prosensa Holding NV to Overweight $RNA http://t.co/oKHjVci3xm
Day Trade Alerts
20 Oct 14 08:07:48
via #AlertTrade Price Action Alert $RNA - PROSENSA HOLDING NV ORDINARY #stocks #intraday http://t.co/VS8RO1bzXJ
Media Sentiment
20 Oct 14 07:40:17
$RNA received a new alert. Find out why at http://t.co/5udYd0Ofs6 #stocks #daytrading #trading #mkt #NYSE #NASDAQ #markets 264
Stephen P Kendal
20 Oct 14 07:39:03
$RNA Prosensa Holding NV Stock Rating Upgraded by JPMorgan Chase & Co. $RNA http://t.co/6MtejJnbNz via @RatingsNetwork
Stephen P Kendal
20 Oct 14 07:37:34
$RNA Prosensa Holding NV. #HighVolume buying Mon-20-Oct. #Vol=117,135. #52WkH/L=$13.32/$3.43. #Price=$11.74⇑+3.4%. S1=⇗ ST=⇗ MT=⇗ LT=⇗
20 Oct 14 07:21:49
Prosensa Holding NV Upgraded to “Overweight” by JPMorgan Chase & Co. $RNA http://t.co/3VIP6jnpZX
Sleek Money
20 Oct 14 06:53:56
Prosensa Holding NV Stock Rating Upgraded by JPMorgan Chase & Co. $RNA http://t.co/dT3KEQkrw7
Anmol Singh
20 Oct 14 06:06:52
Media Sentiment
20 Oct 14 04:50:01
$RNA received a new alert. Find out why at http://t.co/5udYd0Ofs6 #stocks #daytrading #trading #mkt #NYSE #NASDAQ #markets 95
US Consumer News
20 Oct 14 04:49:15
Prosensa Holding NV upgraded by JPMorgan Chase & Co. to overweight. http://t.co/Azh2lUhImf $RNA #RNA
By  +Follow June 27, 2013 11:50AM



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